Banner

Welcome to

Community & Population Health

Research

Contact Community & Population Health

We use implementation science and community expertise to develop and deliver interventions to improve community health. Our team of faculty researchers, data analysts, and community experts are engaged with partners across Montefiore Einstein and with national and global collaborators. Across all of our projects and geographies, our shared mission is to deliver high quality health care and reduce health inequity.

Our current research focuses on:

  • Optimizing community health worker models to respond to health-related social needs
  • Addressing chronic diseases like asthma and diabetes and health-related social needs with integrated models
  • Evaluating changes in childhood mortality in West Africa following the implementation of an integrated primary care program
  • Identifying implementation strategies to integrate and scale health-related social needs screening in large health systems
  • Assessing training for residents on the Social Determinants of Health
  • Evaluating implementation outcomes, including cost and sustainability
  • Global learning for health equity

 

Publications 

Haughton, J., Dabla, D., Goin, D.E., Singer, A.W., Faro, E.Z., Miziou, E., Kangni-Zovoin, A., Agoro, S., Ekouevi, D.K., Fiori, K.P., & Hirschhorn, L.R. (2024). A time series analysis of disruptions to maternal and child health care in northern Togo during the COVID-19 pandemic in the context of an integrated primary care program. Advances in Global Health, 3(1). https://doi.org/10.1525/agh.2024.2123937

 

Mahajan, L., Granados, N., Haines, A., Fiori, K., & Jariwala, S. (2024). Evaluating the relationship between health-related social needs, patient demographics, and access to biologics in patients with moderate-to-severe asthma in Bronx, NY. Journal of Asthma, 1–10. https://doi.org/10.1080/02770903.2024.2362865. PMID: 38832891

 

Islam, F., Rinke, M. L., Philips, K., Cabrera, K. I., Luke, M. J., Chandhoke, S., Friedland, S. E., Acholonu, R.G., McKenna, K. J., Braganza, S., & Fiori, K. P. (2024). Implementing Inpatient Social Needs Screening in an Urban Tertiary Care Children’s Hospital. Hospital Pediatrics, 14(6). https://doi.org/10.1542/hpeds.2023-007486. PMID: 38742306

 

Midura, D., Levano, S., Fiori, K. P., & Farber, B. A. (2024). Analysis of Social Determinants of health in the pediatric general surgical population of a Northeastern Urban Health System. Journal of Pediatric Surgery. https://doi.org/10.1016/j.jpedsurg.2024.04.018. PMID: 38760308

 

Fiori, K. P., Levano, S., Haughton, J., Whiskey-LaLanne, R., Telzak, A., Muleta, H., Vani, K., Chambers, E. C., & Racine, A. (2024). Advancing Social Care Integration in health systems with Community Health Workers: An implementation evaluation based in Bronx, New York. BMC Primary Care, 25(1). https://doi.org/10.1186/s12875-024-02376-7. PMCID: PMC11055265

 

Telzak, A., Levano, S., Haughton, J., Chambers, E. C., & Fiori, K. P. (2024). Understanding individual health-related social needs in the context of area-level social determinants of health: The case for granularity. Journal of Clinical and Translational Science, 8(1), e78. https://doi.org/10.1017/cts.2024.519. PMCID: PMC11091925

 

Eligulashvili, A., Darrell, M., Gordon, M., Jerome, W., Fiori, K. P., Congdon, S., & Duong, T. Q. (2024). Patients with unmet social needs are at higher risks of developing severe long COVID-19 symptoms and Neuropsychiatric Sequela. Scientific Reports, 14(1). https://doi.org/10.1038/s41598-024-58430-y. PMCID: PMC10987523

 

McKenna, K. J., Fiori, K. P., & Chambers, E. C. (2024). Children with chronic health conditions and social needs: Investigating outcomes to drive health system improvements. Clinical Pediatrics. https://doi.org/10.1177/00099228241241896. PMID: 38554010

 

Radparvar, A. A., Vani, K., Fiori, K. P., Gupta, S., Chavez, P., Fisher, M., Sharma, G., Wolfe, D., & Bortnick, A. E. (2024). Hypertensive disorders of pregnancy: Innovative management strategies. JACC: Advances, 3(3). https://doi.org/10.1016/j.jacadv.2024.100864

 

Levano, S. R., Kraemer, J., Dabla, D., Miziou, E. A., Haughton, J., Jones, H. E., Teasdale, C., Ekouevi, D., Hirschhorn, L. R., & Fiori, K. P. (2024). Identifying determinants of under-five child mortality in northern Togo. Journal of Global Health, 14, 04019. https://doi.org/10.7189/jogh.14.04019. PMCID: PMC10832555

 

Shin, S. S., Shah, A., North-Kabore, J., Rowthorn, V., Fiori, K. P., Dudding, R., Plum, R. A., Parke, D. M., George, C., Thomas, S., Pinkett, R., Porter, K. M., Sirois, A., Cordeiro, V., & Ogbolu, Y. (2023). Global Learning for Health Equity: A survey of five global learning sites in the United States. Community Health Equity Research & Policy. https://doi.org/10.1177/2752535x231210046. PMID: 37947506 

 

Fiori, K. P., Levano, S., Haughton, J., Whiskey, R., Telzak, A., Hodgson, S., Spurrell-Huss, E., & Stark, A. (2023). Learning in real world practice: Identifying implementation strategies to integrate health-related social needs screening within a large health system. Journal of Clinical and Translational Science, 7(1), 1–25. https://doi.org/10.1017/cts.2023.652. PMCID: PMC10643918

 

Berman, R. S., Nguyen, H.-A. T., Levano, S. R., & Fiori, K. P. (2023). Clinician champions’ influence on social needs screening volumes in pediatric practices. Clinical Pediatrics, 63(7), 936-941. https://doi.org/10.1177/00099228231200404. PMID: 37715698

 

Ballard, M., Olaniran, A., Iberico, M. M., Rogers, A., Thapa, A., Cook, J., Aranda, Z., French, M., Olsen, H. E., Haughton, J., Lassala, D., Carpenter Westgate, C., Malitoni, B., Juma, M., & Perry, H. B. (2023). Labour conditions in dual-cadre community health worker programmes: A systematic review. The Lancet Global Health, 11(10), e1598-e1608. https://doi.org/10.1016/s2214-109x(23)00357-1. PMID: 37734803

 

Telzak, A., Fiori, K. P., Chambers, E. C., Haughton, J., Levano, S., & Reznik, M. (2023). Unmet social needs and pediatric asthma severity in an urban primary care setting. Academic Pediatrics, 23(7), 1361–1367. https://doi.org/10.1016/j.acap.2023.02.009. PMID: 36858248

 

Gutierrez, A. K., Twito, V., Ni, J., Liu, J., Atrio, J., Rolston, R., Menezes, M., Dunbar, J., Talib, H., Nguyen, H.-A., Fiori, K. P., Cassese, T., Cervera-Hernandez, M. E., Zwerling, J., & Jariwala, S. (2023). The impact of a Twitter-supplemented educational intervention to promote training in social determinants of health among medical residents. Current Journal of Applied Science and Technology, 42(32), 1–13. https://doi.org/10.9734/cjast/2023/v42i324216   

 

Luke, M. J., Fernandes, D. M., Leon Rodriguez, F. D., Acholonu, R. G., & Fiori, K. P. (2023). Caregiver perspectives on social needs screening and interventions in an urban children’s Hospital. Hospital Pediatrics, 13(8), 670–681. https://doi.org/10.1542/hpeds.2022-006736. PMID: 37483130

 

Shi, M., Fiori, K. P., Kim, R. S., Gao, Q., Umanski, G., Thomas, I., Telzak, A., & Chambers, E. C. (2023). Social Needs Assessment and linkage to community health workers in a large urban hospital system. Journal of Primary Care & Community Health, 14. https://doi.org/10.1177/21501319231166918. PMCID: PMC10126704    

 

Azan, A., Stephens, J., Xie, X., Fiori, K. P., & Gover, M. (2023). Covid-19 and changes in reported social risk factors at a primary care practice in the South Bronx. Journal of Primary Care & Community Health, 14, 215013192211471. https://doi.org/10.1177/21501319221147136. PMCID: PMC9834620 

 

Fiori, K. P., & Oyeku, S. O. (2022). Addressing racial, ethnic, and socioeconomic differences in real-world practice. Pediatrics, 150(6). https://doi.org/10.1542/peds.2022-058243. PMID: 36336641  

 

Ogbolu, Y., Dudding, R., Fiori, K. P., North-Kabore, J., Parke, D., Plum, Rev. A., Shin, S., & Rowthorn, V. (2022). Global Learning for Health Equity: A literature review. Annals of Global Health, 88(1). https://doi.org/10.5334/aogh.3810. PMCID: PMC9585985   

 

Faro, E. Z., Haughton, J., Lauria, M. E., Miziou, E., Singer, A., Dabla, D., Gbeleou, S., Ekouevi, D. K., Hirschhorn, L. R., & Fiori, K. P. (2022). A pragmatic approach to qualitative formative evaluation of an Integrated Primary Care Program in Togo, West Africa. Global Implementation Research and Applications, 2(3), 209–218. https://doi.org/10.1007/s43477-022-00055-2 

 

Telzak, A., Chambers, E. C., Gutnick, D., Flattau, A., Chaya, J., McAuliff, K., & Rapkin, B. (2022). Health Care Worker Burnout and perceived capacity to address social needs. Population Health Management, 25(3), 352–361. https://doi.org/10.1089/pop.2021.0175. PMCID: PMC9232233

 

Hanna, D. B., Felsen, U. R., Anastos, K., Bauman, L. J., Fiori, K. P., Ginsberg, M. S., Watnick, D., & Chambers, E. C. (2022). Correction to: Association of social needs with uncontrolled viremia in people with HIV. AIDS and Behavior, 26(11), 3746–3746. https://doi.org/10.1007/s10461-022-03732-3. PMID: 35704126

 

Herrera, T., Fiori, K. P., Archer-Dyer, H., Lounsbury, D. W., & Wylie-Rosett, J. (2022). Social Determinants of health screening by preclinical medical students during the COVID-19 pandemic: Service-Based Learning Case Study. JMIR Medical Education, 8(1). https://doi.org/10.2196/32818. PMCID: PMC8804950 

 

Heller, C. G., Rehm, C. D., Parsons, A. H., Chambers, E. C., Hollingsworth, N. H., & Fiori, K. P. (2021). The association between social needs and chronic conditions in a large, urban primary care population. Preventive Medicine, 153, 106752. https://doi.org/10.1016/j.ypmed.2021.106752. PMCID: PMC8595547 

 

Garg, A., Brochier, A., Messmer, E., & Fiori, K. P. (2021). Clinical approaches to reducing material hardship due to poverty: Social risks/needs identification and interventions. Academic Pediatrics, 21(8). https://doi.org/10.1016/j.acap.2021.02.007. PMID: 34740423

 

Fiori, K. P., Lauria, M. E., Singer, A. W., Jones, H. E., Belli, H. M., Aylward, P. T., Agoro, S., Gbeleou, S., Sowu, E., Grunitzky-Bekele, M., Singham Goodwin, A., Morrison, M., Ekouevi, D. K., & Hirschhorn, L. R. (2021). An integrated primary care initiative for child health in Northern Togo. Pediatrics, 148(3). https://doi.org/10.1542/peds.2020-035493. PMID: 34452981

 

Fiori, K. P., Heller, C. G., Flattau, A., Harris-Hollingsworth, N. R., Parsons, A., Rinke, M. L., Chambers, E. C., Hodgson, S., Chodon, T., & Racine, A. D. (2021). Scaling-up social needs screening in practice: A retrospective, cross-sectional analysis of data from electronic health records from Bronx County, New York, USA. BMJ Open, 11(9), e053633. https://doi.org/10.1136/bmjopen-2021-053633. PMCID: PMC8483051 

 

Sanderson, D., Braganza, S., Philips, K., Chodon, T., Whiskey, R., Bernard, P., Rich, A., & Fiori, K. P. (2021). “Increasing warm handoffs: Optimizing community-based referrals in primary care using QI methodology.” Journal of Primary Care & Community Health, 12, 215013272110238. https://doi.org/10.1177/21501327211023883. PMCID: PMC8202298 

 

Chambers, E. C., McAuliff, K. E., Heller, C. G., Fiori, K. P., & Hollingsworth, N. (2021). Toward understanding social needs among primary care patients with uncontrolled diabetes. Journal of Primary Care & Community Health, 12. https://doi.org/10.1177/2150132720985044. PMCID: PMC7960895 

 

Chambers, E. C., Heller, C., Fiori, K. P., McAuliff, K., & Rehm, C. D. (2020). Chronic pediatric health conditions among youth living in public housing and receiving care in a large hospital system in Bronx, NY. Global Pediatric Health, 7. https://doi.org/10.1177/2333794x20971164. PMCID: PMC7672759

 

Fiori, K. P., Heller, C. G., Rehm, C. D., Parsons, A., Flattau, A., Braganza, S., Lue, K., Lauria, M., & Racine, A. (2020). Unmet social needs and no-show visits in primary care in a US northeastern urban health system, 2018–2019. American Journal of Public Health, 110(S2), S242-S250. https://doi.org/10.2105/ajph.2020.305717. PMCID: PMC7362703 

 

Dubois, M., Faro, E. Z., Lee, D. S., Katin, V., Kenkou, K., & Fiori, K. P. (2020). Integrating childhood TB: Applying the care delivery value chain to improve pediatric HIV/TB services in Togo, West Africa. AIDS Care, 32(11), 1445–1450. https://doi.org/10.1080/09540121.2020.1770672. PMCID: PMC7572731

 

Kaplowitz, E. T., Fiori, K. P., Lauria, M. E., Gbeleou, S., Miziou, A., Sowu, E., Schechter, J., & Jones, H. E. (2020). Sexual relationship power and socio-demographic factors predicting contraceptive use, antenatal visits and sick child health service use in northern Togo. Maternal and Child Health Journal, 24(7), 845–855. https://doi.org/10.1007/s10995-020-02948-w. PMCID: PMC7340338

 

Heller, C. G., Parsons, A. S., Chambers, E. C., Fiori, K. P., & Rehm, C. D. (2020). Social risks among primary care patients in a large urban health system. American Journal of Preventive Medicine, 58(4), 514–525. https://doi.org/10.1016/j.amepre.2019.11.011. PMCID: PMC7362999

 

Fiori, K. P., Patel, M., Sanderson, D., Parsons, A., Hodgson, S., Scholnick, J., Bathory, E., White-Davis, T., Wigod, N., Chodon, T., Rich, A., & Braganza, S. (2020). From policy statement to practice: Integrating Social Needs Screening and referral assistance with community health workers in an urban academic health center. Journal of Primary Care & Community Health, 10. https://doi.org/10.1177/2150132719899207. PMCID: PMC6940600 

 

Lauria, M. E., Fiori, K. P., Jones, H. E., Gbeleou, S., Kenkou, K., Agoro, S., Agbèrè, A. D., Lue, K. D., & Hirschhorn, L. R. (2019). Assessing the integrated community-based Health Systems Strengthening Initiative in Northern Togo: A pragmatic effectiveness-implementation study protocol. Implementation Science, 14(1), 92. https://doi.org/10.1186/s13012-019-0921-3. PMCID: PMC6796416

 

Fiori, K. P., Belli, H. M., Lauria, M. E., Hirschhorn, L. R., Schechter, J., Hansman, E., Rajshekhar, N., Katin, V., Gbeleou, S., Grunitsky-Bekele, M., & Pitche, V. P. (2019). Implementing an integrated community based Health Systems Strengthening Approach to improve HIV survival in northern Togo. AIDS Care, 32(6), 705–713. https://doi.org/10.1080/09540121.2019.1626342. PMCID: PMC7357197

 

Fiori, K. P., Schechter, J., Gbeleou, S. C., Braganza, S., Rhatigan, J., Houndenou, S., Lopez, A., Bensen, E., & Hirschhorn, L. (2018). Closing the delivery gap: Operationalizing the care delivery value chain and continuous quality improvement for HIV/AIDS services in Kara, Togo. International Journal of Health Care Quality Assurance, 31(4), 327–336. https://doi.org/10.1108/ijhcqa-02-2017-0024. PMCID: 29790442

 

McCarthy, K. J., Braganza, S., Fiori, K. P., Gbeleou, C., Kpakpo, V., Lopez, A., Schechter, J., Singham Goodwin, A., & Jones, H. E. (2017). Identifying inequities in maternal and child health through risk stratification to inform health systems strengthening in northern Togo. PLOS ONE, 12(3), e0173445. https://doi.org/10.1371/journal.pone.0173445. PMCID: PMC5354273  

 

Arnold, J., Samson, M., Schechter, J., Goodwin, A. S., Braganza, S., Sesso, G. C., Lopez, A., & Fiori, K. P. (2016). Getting there: Overcoming barriers to reproductive and maternal health services access in northern Togo-a qualitative study. World Medical & Health Policy, 8(3), 223–244. https://doi.org/10.1002/wmh3.195 

 

Fiori, K. P., Schechter, J., Dey, M., Braganza, S., Rhatigan, J., Houndenou, S., Gbeleou, C., Palerbo, E., Tchangani, E., Lopez, A., Bensen, E., & Hirschhorn, L. R. (2016). Closing the delivery gaps in pediatric HIV care in Togo, West Africa: Using the care delivery value chain framework to direct quality improvement. AIDS Care, 28(sup2), 29–33. https://doi.org/10.1080/09540121.2016.1176678. PMCID: PMC4991220  

 

Loccoh, E. C., Azouma, D., Fiori, K. P., Schechter, J., Gbeleou, S., & Hirschhorn, L. R. (2014). Patient-reported factors facilitating participation in prevention of mother to child transmission of HIV programs in Kara, Togo, West Africa. World Journal of AIDS, 04(04), 446–457. https://doi.org/10.4236/wja.2014.44053 

 

Trial Description
Disease / Condition
Principal Investigator
Contact
Trial DescriptionA 52-week, Randomised, Double-blind, Double-dummy, Parallel Group, Multi-centre, Non-inferiority Study Assessing Exacerbation Rate, Additional Measures of Asthma Control and Safety in Adult and Adolescent Severe Asthmatic Participants With an Eosinophilic Phenotype Treated With GSK3511294 Compared With Mepolizumab or Benralizumab
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Contact
Email Contact
Trial DescriptionA 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease
Disease/ConditionInflammatory Bowel Diseases
Crohn's Disease
Ulcerative Colitis
Indeterminate Colitis
Principal InvestigatorGitit Tomer
Contact
9842340268
 Email Contact
Trial DescriptionA Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
Disease/ConditionDuchenne Muscular Dystrophy
Principal InvestigatorVolkan Granit
Contact
Email Contact
Trial DescriptionA Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy
Disease/ConditionBiliary Atresia
Principal InvestigatorDebra Pan
Contact
Email Contact
Trial DescriptionA Longitudinal Observational Study of Patients Undergoing Therapy for Immune-Mediated Inflammatory Skin Conditions
Disease/ConditionAtopic Dermatitis
Alopecia Areata
Hidradenitis Suppurativa
Vitiligo
Psoriasis
Chronic Spontaneous Urticaria
Principal InvestigatorHolly Kanavy
Contact
9842340268
 Email Contact
Trial DescriptionA Longitudinal Observational Study of Patients With Nonalcoholic Steatohepatitis (NASH) and Related Conditions Across the Entire Spectrum of Nonalcoholic Fatty Liver Disease (NAFLD)
Disease/ConditionNonalcoholic Fatty Liver
Nonalcoholic Steatohepatitis
Principal InvestigatorPreeti Viswanathan
Contact
Email Contact
Trial DescriptionA Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
Disease/ConditionLennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Principal InvestigatorPuja Patel
Contact
Email Contact
Trial DescriptionA Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
Disease/ConditionLennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Principal InvestigatorPuja Patel
Contact
Email Contact
Trial DescriptionA LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
Contact
Email Contact
Trial DescriptionA Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications
Disease/ConditionHematologic Malignancies
Inherited Disorders of Metabolism
Inherited Abnormalities of Platelets
Histiocytic Disorders
Acute Myelogenous Leukemia (AML or ANLL)
Acute Lymphoblastic Leukemia (ALL)
Other Acute Leukemia
Chronic Myelogenous Leukemia (CML)
Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases
Other Leukemia
Hodgkin Lymphoma
Non-hodgkin Lymphoma
Multiple Myeloma/ Plasma Cell Disorder (PCD)
Inherited Abnormalities of Erythrocyte Differentiation or Function
Disorders of the Immune System
Autoimmune Diseases
Severe Aplastic Anemia
Principal InvestigatorDavid Loeb
Contact
Email Contact
Trial DescriptionA Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients
Disease/ConditionInfusion Reactions
Principal InvestigatorDavid Loeb
Contact
718-706-5202
 Email Contact
Trial DescriptionA Multicenter, Open-label, Randomized, Study With Single-arm Extension Period to Assess the Pharmacokinetics, Safety and Efficacy of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension
Disease/ConditionPulmonary Arterial Hypertension
Principal InvestigatorNicole Sutton
Contact
Email Contact
Trial DescriptionA Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic Syndrome (MDS)
Disease/ConditionAcute Lymphoblastic Leukemia
Acute Myeloid Leukemia
Mixed Phenotype Acute Leukemia
Myelodysplastic Syndrome
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Multicenter, Prospective, Long-term, Observational Registry of Pediatric Patients With Inflammatory Bowel Disease
Disease/ConditionCrohn's Disease
Ulcerative Colitis
Indeterminate Colitis
Inflammatory Bowel Diseases
Principal InvestigatorGitit Tomer
Contact
Email Contact
Trial DescriptionA Multi-center, Randomized, Placebo-Controlled Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of Belimumab, a Human Monoclonal Anti-BLyS Antibody, Plus Standard Therapy in Pediatric Patients With Systemic Lupus Erythematosus
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorDawn Wahezi
Contact
Email Contact
Trial DescriptionA Multi-Institution Study of TGFβ Imprinted, Ex Vivo Expanded Universal Donor NK Cell Infusions As Adoptive Immunotherapy in Combination with Gemcitabine and Docetaxel in Patients with Relapsed or Refractory Pediatric Bone and Soft Tissue
Disease/ConditionPediatric Sarcoma, Refractory
Pediatric Sarcoma, Relapsed
Principal InvestigatorAlice Lee
Contact
813-745-6250
 Email Contact
Trial DescriptionA Phase 1, First-in-Human, Dose Escalation and Expansion Study to Evaluate the Safety and Tolerability of XmAb541 in Advanced Solid Tumors
Disease/ConditionOvarian Cancer
Endometrial Cancer
Germ Cell Tumor
Testicular Germ Cell Tumor
Ovarian Germ Cell Tumor
Principal InvestigatorEric Feldman
Contact
1-858-828-6286
 Email Contact
Trial DescriptionA Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-X19 in Pediatric and Adolescent Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4)
Disease/ConditionRelapsed/Refractory B-precursor Acute Lymphoblastic Leukemia
Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma
Principal InvestigatorEllen Fraint
Contact
Email Contact
Trial DescriptionA Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Disease/ConditionAnaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Diffuse Intrinsic Pontine Glioma
Diffuse Midline Glioma, H3 K27M-Mutant
Glioblastoma
Glioblastoma, Not Otherwise Specified
Malignant Glioma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients With Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation
Disease/ConditionAcute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Mixed Lineage Acute Leukemia
Mixed Phenotype Acute Leukemia
Acute Leukemia of Ambiguous Lineage
Principal InvestigatorIoannis Mantzaris
Contact
781-419-1400
 Email Contact
Trial DescriptionA Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma
Disease/ConditionHigh Risk Neuroblastoma
Recurrent Neuroblastoma
Refractory Neuroblastoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG)
Disease/ConditionAnaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Anaplastic Ganglioglioma
Anaplastic Pleomorphic Xanthoastrocytoma
Glioblastoma
Malignant Glioma
WHO Grade 3 Glioma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518) in Children and Young Adults With Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Recurrent B Lymphoblastic Lymphoma
Refractory B Acute Lymphoblastic Leukemia
Refractory B Lymphoblastic Lymphoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Disease/ConditionMedulloblastoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway-Mutant Acute Lymphoblastic Leukemia
Disease/ConditionLeukemia
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase 2 Trial of Chemotherapy Followed by Response-Based Whole Ventricular &Amp; Spinal Canal Irradiation (WVSCI) for Patients With Localized Non-Germinomatous Central Nervous System Germ Cell Tumor
Disease/ConditionCentral Nervous System Nongerminomatous Germ Cell Tumor
Choriocarcinoma
Embryonal Carcinoma
Immature Teratoma
Malignant Teratoma
Mixed Germ Cell Tumor
Pineal Region Germ Cell Tumor
Pineal Region Immature Teratoma
Pineal Region Yolk Sac Tumor
Suprasellar Germ Cell Tumor
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase 2,Multicenter,Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab,With or Without Hydroxyurea/Hydroxycarbamide,in Sequential,Descending Age Groups of Pediatric Sickle Cell Disease Patients With Vaso-Occlusive Crisis
Disease/ConditionSickle Cell Disease (SCD)
Principal InvestigatorDeepa Manwani
Contact
Email Contact
Trial DescriptionA Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease
Disease/ConditionProteinuric Kidney Disease
Principal InvestigatorMichael Ross
Contact
617-341-6777
 Email Contact
Trial DescriptionA Phase 2/3 Randomized, Placebo-Controlled, Double-blind, Clinical Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Systemic Left Ventricular Systolic Dysfunction (VALOR)
Disease/ConditionHeart Failure
Left Ventricular Systolic Dysfunction
Principal InvestigatorDaphne Hsu
Contact
1-888-577-8839
 Email Contact
Trial DescriptionA Phase 2/3, Open-Label Study of the Pharmacokinetics, Safety, and Antiviral Activity of the Elvitegravir/Cobicistat/Emtricitabine/Tenofovir Alafenamide (E/C/F/TAF) Single Tablet Regimen (STR) in HIV-1 Infected Antiretroviral Treatment-Naive Adolescents and Virologically Suppressed Children
Disease/ConditionAcquired Immune Deficiency Syndrome (AIDS)
HIV Infections
Principal InvestigatorDonna Futterman
Contact
Email Contact
Trial DescriptionA Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity
Disease/ConditionPlexiform Neurofibroma
Neurofibromatosis Type 1 (NF1)
Principal InvestigatorNagma Dalvi
Contact
Email Contact
Trial DescriptionA Phase 3 Multicenter, Long-Term Extension Study to Evaluate the Safety and Efficacy of Upadacitinib (ABT-494) in Subjects With Ulcerative Colitis
Disease/ConditionUlcerative Colitis (UC)
Principal InvestigatorThomas Ullman
Contact
Email Contact
Trial DescriptionA Phase 3 Multicenter, Open-label Study to Evaluate the Efficacy, Pharmacokinetics, Safety, and Immunogenicity of Subcutaneously Administered Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic Arthritis (PSUMMIT-Jr)
Disease/ConditionArthritis, Juvenile
Principal InvestigatorDawn Wahezi
Contact
844-434-4210
 Email Contact
Trial DescriptionA Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
Disease/ConditionMetastatic Malignant Neoplasm in the Lung
Metastatic Osteosarcoma
Osteosarcoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Disease/ConditionLow Grade Astrocytoma
Low Grade Glioma
Metastatic Low Grade Astrocytoma
Metastatic Low Grade Glioma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Disease/ConditionLow Grade Glioma
Neurofibromatosis Type 1
Visual Pathway Glioma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations
Disease/ConditionAcute Myeloid Leukemia
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3 Randomized Trial of Inotuzumab Ozogamicin (NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Central Nervous System Leukemia
Mixed Phenotype Acute Leukemia
Testicular Leukemia
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3 Randomized, Placebo-Controlled Trial Evaluating Memantine for Neurocognitive Protection in Children Undergoing Cranial Radiotherapy as Part of Treatment for Primary Central Nervous System Tumors
Disease/ConditionCentral Nervous System Carcinoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Upadacitinib in Adolescent and Adult Subjects With Moderate to Severe Atopic Dermatitis
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
Contact
Email Contact
Trial DescriptionA Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the BB305 Lentiviral Vector in Subjects With Sickle Cell Disease
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Contact
Email Contact
Trial DescriptionA Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)
Disease/ConditionGanglioneuroblastoma
Neuroblastoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients With Germ Cell Tumors
Disease/ConditionChildhood Extracranial Germ Cell Tumor
Extragonadal Embryonal Carcinoma
Germ Cell Tumor
Malignant Germ Cell Tumor
Malignant Ovarian Teratoma
Stage I Ovarian Choriocarcinoma
Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage I Ovarian Teratoma AJCC v6 and v7
Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage I Testicular Choriocarcinoma AJCC v6 and v7
Stage I Testicular Embryonal Carcinoma AJCC v6 and v7
Stage I Testicular Seminoma AJCC v6 and v7
Stage I Testicular Yolk Sac Tumor AJCC v6 and v7
Stage II Ovarian Choriocarcinoma
Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage II Testicular Choriocarcinoma AJCC v6 and v7
Stage II Testicular Embryonal Carcinoma AJCC v6 and v7
Stage II Testicular Yolk Sac Tumor AJCC v6 and v7
Stage III Ovarian Choriocarcinoma
Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage III Testicular Choriocarcinoma AJCC v6 and v7
Stage III Testicular Embryonal Carcinoma AJCC v6 and v7
Stage III Testicular Yolk Sac Tumor AJCC v6 and v7
Stage IV Ovarian Choriocarcinoma
Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7
Testicular Mixed Choriocarcinoma and Embryonal Carcinoma
Testicular Mixed Choriocarcinoma and Teratoma
Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3 Trial Investigating Blinatumomab (NSC# 765986) in Combination With Chemotherapy in Patients With Newly Diagnosed Standard Risk or Down Syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients With Localized B-Lymphoblastic Lymphoma (B-LLy)
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Down Syndrome
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3 Trial Investigating Blinatumomab (NSC# 765986) in Combination With Chemotherapy in Patients With Newly Diagnosed Standard Risk or Down Syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients With Localized B-Lymphoblastic Lymphoma (B-LLy)
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Down Syndrome
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Phase 3, Double-Blind, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Subcutaneous Twice Yearly Long-Acting Lenacapavir for HIV Pre-Exposure Prophylaxis in Cisgender Men, Transgender Women, Transgender Men, and Gender Nonbinary People ≥ 16 Years of Age Who Have Sex With Male Partners and Are at Risk for HIV Infection
Disease/ConditionPre-Exposure Prophylaxis of HIV Infection
Principal InvestigatorMichelle Collins-Ogle
Contact
Email Contact
Trial DescriptionA Phase 3, Double-Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics (PK) of Baricitinib in Children From 6 Years to Less Than 18 Years of Age With Alopecia Areata
Disease/ConditionAreata Alopecia
Alopecia
Hypotrichosis
Hair Diseases
Skin Diseases
Pathological Conditions, Anatomical
Principal InvestigatorHolly Kanavy
Contact
-317-615-4559
 Email Contact
Trial DescriptionA Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Disease/ConditionImmune Thrombocytopenia
Principal InvestigatorIrina Murakhovskaya
Contact
Email Contact
Trial DescriptionA Phase 3, Open-label, Long-term Safety Study of Oral Linaclotide Administered to Pediatric Participants With Functional Constipation (FC) or Irritable Bowel Syndrome With Constipation (IBS-C)
Disease/ConditionIrritable Bowel Syndrome With Constipation
Functional Constipation
Principal InvestigatorJohn Thompson
Contact
Email Contact
Trial DescriptionA Phase 3, Open-label, Multi-center, Single Arm Study to Assess Contraceptive Efficacy and Safety of the Etonogestrel (MK-8415) Implant During Extended Use From 3 Years After Insertion in Females 35 Years of Age or Younger
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
Contact
Email Contact
Trial DescriptionA Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Rimegepant in Migraine Prevention in Children and Adolescents ≥ 6 to <18 Years of Age
Disease/ConditionMigraine
Principal InvestigatorJelena Pavlovic
Contact
1-800-718-1021
 Email Contact
Trial DescriptionA Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
Disease/ConditionChronic Kidney Disease; Iron Deficiency Anemia
Principal InvestigatorAnna Zolotnitskaya
Contact
1-877-374 -4177
 Email Contact
Trial DescriptionA Phase I Study With an Expansion Cohort/Randomized Phase II Study of the Combinations of Ipilimumab, Nivolumab and Brentuximab Vedotin in Patients With Relapsed/Refractory Hodgkin Lymphoma
Disease/ConditionRecurrent Classic Hodgkin Lymphoma
Refractory Classic Hodgkin Lymphoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination With Azacitidine in Patients With Recurrent, Resectable Osteosarcoma
Disease/ConditionOsteosarcoma
Osteosarcoma in Children
Osteosarcoma Recurrent
Sarcoma
Principal InvestigatorDaniel Weiser
Contact
Email Contact
Trial DescriptionA Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors
Disease/ConditionRefractory and/or Relapsed Metastatic Solid Tumors
Principal InvestigatorDavid Loeb
Contact
410-502-4997
 Email Contact
Trial DescriptionA Phase III Randomized Study of Adjuvant Ipilimumab Anti-CTLA4 Therapy Versus High-Dose Interferon a-2b for Resected High-Risk Melanoma
Disease/ConditionMelanoma of Unknown Primary
Recurrent Melanoma
Stage IIIB Cutaneous Melanoma AJCC v7
Stage IIIC Cutaneous Melanoma AJCC v7
Stage IV Cutaneous Melanoma AJCC v6 and v7
Principal InvestigatorJoseph Sparano
Contact
Email Contact
Trial DescriptionA Phase III Randomized Trial for Newly Diagnosed High Risk B-Lymphoblastic Leukemia (B-ALL) Including a Stratum Evaluating Dasatinib (NSC#732517) in Patients With Ph-like Tyrosine Kinase Inhibitor (TKI) Sensitive Mutations
Disease/ConditionB Acute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia, BCR-ABL1-Like
Central Nervous System Leukemia
Testicular Leukemia
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase III Randomized Trial for Patients With De Novo AML Using Bortezomib and Sorafenib (NSC# 681239, NSC# 724772) for Patients With High Allelic Ratio FLT3/ITD
Disease/ConditionAcute Myeloid Leukemia
Leukemia Cutis
Myeloid Neoplasm
Myeloid Sarcoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase III Randomized Trial Investigating Bortezomib (NSC# 681239) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy)
Disease/ConditionAdult T Acute Lymphoblastic Leukemia
Ann Arbor Stage II Adult Lymphoblastic Lymphoma
Ann Arbor Stage II Childhood Lymphoblastic Lymphoma
Ann Arbor Stage III Adult Lymphoblastic Lymphoma
Ann Arbor Stage III Childhood Lymphoblastic Lymphoma
Ann Arbor Stage IV Adult Lymphoblastic Lymphoma
Ann Arbor Stage IV Childhood Lymphoblastic Lymphoma
Childhood T Acute Lymphoblastic Leukemia
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase III Randomized Trial of Adding Vincristine-Topotecan-Cyclophosphamide to Standard Chemotherapy in Initial Treatment of Non-Metastatic Ewing Sarcoma
Disease/ConditionLocalized Extraskeletal Ewing Sarcoma
Peripheral Primitive Neuroectodermal Tumor of Bone
Peripheral Primitive Neuroectodermal Tumor of Soft Tissues
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase III Study for Patients With Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid
Disease/ConditionAcute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Phase III, Randomized Study of Nivolumab (Opdivo) Plus AVD or Brentuximab Vedotin (Adcetris) Plus AVD in Patients (Age >/= 12 Years) With Newly Diagnosed Advanced Stage Classical Hodgkin Lymphoma
Disease/ConditionAnn Arbor Stage III Hodgkin Lymphoma
Ann Arbor Stage III Lymphocyte-Depleted Classic Hodgkin Lymphoma
Ann Arbor Stage III Mixed Cellularity Classic Hodgkin Lymphoma
Ann Arbor Stage III Nodular Sclerosis Classic Hodgkin Lymphoma
Ann Arbor Stage IV Hodgkin Lymphoma
Ann Arbor Stage IV Lymphocyte-Depleted Classic Hodgkin Lymphoma
Ann Arbor Stage IV Mixed Cellularity Classic Hodgkin Lymphoma
Ann Arbor Stage IV Nodular Sclerosis Classic Hodgkin Lymphoma
Classic Hodgkin Lymphoma
Lymphocyte-Rich Classic Hodgkin Lymphoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma
Disease/ConditionGanglioneuroblastoma, Nodular
Neuroblastoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations
Disease/ConditionCardiomyopathy
Principal InvestigatorDaphne Hsu
Contact
650-209-8092
 Email Contact
Trial DescriptionA Prospective Observational Study of Patients Receiving DUPIXENT® for Atopic Dermatitis
Disease/ConditionDermatitis, Atopic
Principal InvestigatorHolly Kanavy
Contact
Email Contact
Trial DescriptionA Prospective Phase 3 Study of Patients With Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma
Disease/ConditionEmbryonal Rhabdomyosarcoma
Fusion-Negative Alveolar Rhabdomyosarcoma
Spindle Cell/Sclerosing Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Disease/ConditionSpinal Muscular Atrophy (SMA)
Principal InvestigatorLESLIE DELFINER
Contact
1-888-669-6682
 Email Contact
Trial DescriptionA Prospective, Multi-center Registry for Patients With Short Bowel Syndrome
Disease/ConditionShort Bowel Syndrome
Principal InvestigatorJohn Thompson
Contact
Email Contact
Trial DescriptionA Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid)
Disease/ConditionBile Acid Synthesis Disorders
Principal InvestigatorNadia Ovchinsky
Contact
Email Contact
Trial DescriptionA Prospective, Open-label, Randomized, Concurrent Active-controlled, Longitudinal, Multicenter, Phase 3 Clinical Study of the Safety and Efficacy of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee
Disease/ConditionChondral Defect
Osteochondritis Dissecans (OCD)
Articular Cartilage Defect
Articular Cartilage Disorder of Knee
Principal InvestigatorEric Fornari
Contact
484-387-2257
 Email Contact
Trial DescriptionA Randomized Controlled Trial of Women Involved in Supporting Health (WISH), a Peer-Led Intervention to Improve Postpartum Retention in HIV Care
Disease/ConditionHIV/AIDS
Principal InvestigatorRodney Wright
Contact
215-746-8555
 Email Contact
Trial DescriptionA Randomized Phase 3 Interim Response Adapted Trial Comparing Standard Therapy With Immuno-oncology Therapy for Children and Adults With Newly Diagnosed Stage I and II Classic Hodgkin Lymphoma
Disease/ConditionLugano Classification Limited Stage Hodgkin Lymphoma AJCC v8
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Randomized Phase 3 Study of Brentuximab Vedotin (SGN-35) for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma (cHL) in Children and Young Adults
Disease/ConditionAnn Arbor Stage IIB Hodgkin Lymphoma
Ann Arbor Stage IIIB Hodgkin Lymphoma
Ann Arbor Stage IVA Hodgkin Lymphoma
Ann Arbor Stage IVB Hodgkin Lymphoma
Childhood Hodgkin Lymphoma
Classic Hodgkin Lymphoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Rhabdomyosarcoma
Sclerosing Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Rhabdomyosarcoma
Sclerosing Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Randomized Phase 3 Trial of Nivolumab (NSC# 748726) in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma
Disease/ConditionPrimary Mediastinal Large B-Cell Lymphoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy With Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) Plus VINO-CPO Maintenance in Patients With High Risk Rhabdomyosarcoma (HR-RMS)
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Metastatic Embryonal Rhabdomyosarcoma
Metastatic Rhabdomyosarcoma
Solid Alveolar Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
Spindle Cell/Sclerosing Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionA Randomized Phase III Trial Comparing Conventional-Dose Chemotherapy Using Paclitaxel, Ifosfamide, and Cisplatin (TIP) With High-Dose Chemotherapy Using Mobilizing Paclitaxel Plus Ifosfamide Followed by High-Dose Carboplatin and Etoposide (TI-CE) as First Salvage Treatment in Relapsed or Refractory Germ Cell Tumors
Disease/ConditionGerm Cell Tumor
Teratoma
Choriocarcinoma
Germinoma
Mixed Germ Cell Tumor
Yolk Sac Tumor
Childhood Teratoma
Malignant Germ Cell Neoplasm
Extragonadal Seminoma
Non-seminomatous Germ Cell Tumor
Seminoma
Principal InvestigatorBenjamin Gartrell
Contact
Email Contact
Trial DescriptionA Randomized Study of Dexamethasone as Adjuvant Therapy for Acute Post-traumatic Headache
Disease/ConditionPost-Traumatic Headache
Principal InvestigatorBenjamin Friedman
Contact
718-920-6626
 Email Contact
Trial DescriptionA Randomized, Assessor-Blind, Parallel-Groups, Multicenter Trial Assessing the Safety and Efficacy, Including Pharmacokinetic Assessments, of CLENPIQ in Children Aged 2 Years to Less Than 9 Years
Disease/ConditionBowel Preparation
Principal InvestigatorInna Novak
Contact
+1 833-548-1402 (US/Canada)
 Email Contact
Trial DescriptionA Randomized, Comparative Effectiveness Study of Staged Complete Revascularization With Percutaneous Coronary Intervention to Treat Coronary Artery Disease vs Medical Management Alone in Patients With Symptomatic Aortic Valve Stenosis Undergoing Elective Transfemoral Transcatheter Aortic Valve Replacement: The COMPLETE TAVR Study
Disease/ConditionAortic Stenosis
Coronary Artery Disease
Coronary Stenosis
Principal InvestigatorMark Menegus
Contact
604-875-4111
 Email Contact
Trial DescriptionA Randomized, Double Blind, Parallel-group, Placebo Controlled Study to Evaluate the Efficacy and Safety of IBP-9414 in Premature Infants 500-1500g Birth Weight in the Prevention of Necrotizing Enterocolitis - The Connection Study
Disease/ConditionNecrotizing Enterocolitis
Principal InvestigatorShantanu Rastogi
Contact
Email Contact
Trial DescriptionA Randomized, Double-Blind Evaluation of the Pharmacokinetics, Safety, and Antiviral Efficacy of Tenofovir Alafenamide (TAF) in Children and Adolescent Subjects With Chronic Hepatitis B Virus Infection
Disease/ConditionChronic Hepatitis B
Principal InvestigatorDebra Pan
Contact
Email Contact
Trial DescriptionA Randomized, Double-Blind, Double Dummy, Parallel Group, Multicenter Variable Length Study to Assess the Efficacy and Safety of PT010 Relative to PT009 and Symbicort® in Adult and Adolescent Participants With Inadequately Controlled Asthma
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Contact
Email Contact
Trial DescriptionA Randomized, Double-Blind, Double Dummy, Parallel Group, Multicenter Variable Length Study to Assess the Efficacy and Safety of PT010 Relative to PT009 and Symbicort® in Adult and Adolescent Participants With Inadequately Controlled Asthma
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Contact
Email Contact
Trial DescriptionA Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Carisbamate (YKP509) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults, With Optional Open-Label Extension
Disease/ConditionSeizures
Lennox Gastaut Syndrome
Principal InvestigatorAlexis Boro
Contact
201-421-3810
 Email Contact
Trial DescriptionA Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Extended-release Tablets n Pediatric Subjects Aged 5 to 17 Years With Active, Mild to Moderate Ulcerative Colitis
Disease/ConditionUlcerative Colitis
Principal InvestigatorGitit Tomer
Contact
9082428287
 Email Contact
Trial DescriptionA Randomized, Multicenter, Double-blind, Parallel, Active-control Study of the Effects of Sparsentan, a Dual Endothelin Receptor and Angiotensin Receptor Blocker, on Renal Outcomes in Patients With Primary FSGS
Disease/ConditionFocal Segmental Glomerulosclerosis
Principal InvestigatorFrederick Kaskel
Contact
Email Contact
Trial DescriptionA Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in Combination With Azacitidine After Allogeneic Stem Cell Transplantation in Subjects With Acute Myeloid Leukemia (AML) (VIALE-T)
Disease/ConditionAcute Myeloid Leukemia (AML)
Cancer
Principal InvestigatorIoannis Mantzaris
Contact
Email Contact
Trial DescriptionA Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in Combination With Azacitidine After Allogeneic Stem Cell Transplantation in Subjects With Acute Myeloid Leukemia (AML) (VIALE-T)
Disease/ConditionAcute Myeloid Leukemia (AML)
Cancer
Principal InvestigatorIoannis Mantzaris
Contact
Email Contact
Trial DescriptionA Remotely Delivered CBT Intervention for Youth With cSLE: A Multi-site Patient-engaged Investigation
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorTamar Rubinstein
Contact
Email Contact
Trial DescriptionA Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors
Disease/ConditionDesmoid Fibromatosis
Recurrent Desmoid Fibromatosis
Unresectable Desmoid Fibromatosis
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionA Study Evaluating Limited Target Volume Boost Irradiation and Reduced Dose Craniospinal Radiotherapy (18.00 Gy) and Chemotherapy in Children With Newly Diagnosed Standard Risk Medulloblastoma: A Phase III Double Randomized Trial
Disease/ConditionMedulloblastoma
Principal InvestigatorPeter Cole
Contact
Email Contact
Trial DescriptionACURATE IDE: Transcatheter Replacement of Stenotic Aortic Valve Through Implantation of ACURATE in Subjects InDicatEd for TAVR
Disease/ConditionAortic Stenosis
Principal InvestigatorMohamed Azeem Latib
Contact
Email Contact
Trial DescriptionAn Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Contact
(+1) 866-867-7178
 Email Contact
Trial DescriptionAn Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA)
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
Contact
Email Contact
Trial DescriptionAn Open-Label Extension Study of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin
Disease/ConditionAutism
Principal InvestigatorEric Hollander
Contact
Email Contact
Trial DescriptionAn Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)
Disease/ConditionProgressive Familial Intrahepatic Cholestasis (PFIC)
Principal InvestigatorNadia Ovchinsky
Contact
Email Contact
Trial DescriptionAn Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)
Disease/ConditionProgressive Familial Intrahepatic Cholestasis (PFIC)
Principal InvestigatorNadia Ovchinsky
Contact
Email Contact
Trial DescriptionAn Open-label, First-in-human, Dose-escalation/Expansion Study of SAR443579 Administered as Single Agent by Intravenous Infusion in Adult and Pediatric Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Disease/ConditionAcute Lymphocytic Leukaemia
Acute Myeloid Leukaemia Refractory
Myelodysplastic Syndromes
Blastic Plasmacytoid Dendritic Cell Neoplasia
Principal InvestigatorIoannis Mantzaris
Contact
800-633-1610
 Email Contact
Trial DescriptionAn Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases (EBVision)
Disease/ConditionEpstein-Barr Virus (EBV)-associated Diseases
EBV+ Lymphoproliferative Disease with Primary Immunodeficiency (EBV+ PID LPD)
EBV+ Lymphoproliferative Disease with Acquired (non-congenital) Immunodeficiency (EBV+ AID LPD)
EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD)
EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
Solid Organ Transplant Complications
Lymphoproliferative Disorders
Allogeneic Hematopoietic Cell Transplant
Stem Cell Transplant Complications
EBV+ Sarcomas
Leiomyosarcoma
Principal InvestigatorDavid Loeb
Contact
650-278-8930
 Email Contact
Trial DescriptionAPOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO)
Disease/ConditionKidney Diseases
Kidney Failure
Kidney Disease, Chronic
Principal InvestigatorEnver Akalin
Contact
336-713-4292
 Email Contact
Trial DescriptionAssessing the Effectiveness of Pictographs for Preventing Wrong-Patient Errors in Neonatal Intensive Care Units: A Randomized Controlled Trial
Disease/ConditionMedical Errors
Electronic Medical Records
Principal InvestigatorKatie Forman
Contact
646-317-4803
 Email Contact
Trial DescriptionAssessment of CMV-specific T Cell Responses by Flow Cytometry With Intracellular Cytokine Staining to Predict CMV Infection Risk in Pediatric Solid Organ Transplant Recipients
Disease/ConditionHeart Transplant Infection
Kidney Transplant Infection
Liver Transplant Infection
CMV
Principal InvestigatorBetsy Herold
Contact
Email Contact
Trial DescriptionCancer Moonshot Biobank Research Protocol
Disease/ConditionAcute Myeloid Leukemia
Anatomic Stage III Breast Cancer AJCC v8
Anatomic Stage IV Breast Cancer AJCC v8
Clinical Stage III Cutaneous Melanoma AJCC v8
Clinical Stage IV Cutaneous Melanoma AJCC v8
Clinical Stage IV Esophageal Adenocarcinoma AJCC v8
Clinical Stage IV Esophageal Squamous Cell Carcinoma AJCC v8
Clinical Stage IV Gastric Cancer AJCC v8
Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8
Lung Non-Small Cell Carcinoma
Lung Small Cell Carcinoma
Malignant Solid Neoplasm
Metastatic Prostate Carcinoma
Multiple Myeloma
Stage III Lung Cancer AJCC v8
Stage III Ovarian Cancer AJCC v8
Stage IV Colorectal Cancer AJCC v8
Stage IV Lung Cancer AJCC v8
Stage IV Ovarian Cancer AJCC v8
Stage IV Prostate Cancer AJCC v8
Stage IVB Prostate Cancer AJCC v8
Principal InvestigatorBalazs Halmos
Contact
Email Contact
Trial DescriptionCannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)
Disease/ConditionAutism Spectrum Disorder
Principal InvestigatorEric Hollander
Contact
914-315-4236
 Email Contact
Trial DescriptionChronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)
Disease/ConditionB Acute Lymphoblastic Leukemia Associated With Down Syndrome
Down Syndrome
Myeloid Leukemia Associated With Down Syndrome
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionChronic Kidney Disease in Children Prospective Cohort Study (CKiD)
Disease/ConditionChronic Kidney Disease
Principal InvestigatorFrederick Kaskel
Contact
Email Contact
Trial DescriptionComparison of Conventional Non-Invasive Ventilation and Neurally-Adjusted Ventilatory Assistance (NAVA) Non-Invasive Ventilation for the Treatment of Bronchiolitis
Disease/ConditionBronchiolitis
Principal InvestigatorJacqueline Weingarten-Arams
Contact
2017459825
 Email Contact
Trial DescriptionDocumentation and Delivery of Guideline-Consistent Treatment in Adolescent and Young Adult (AYA) Acute Lymphoblastic Leukemia (ALL)
Disease/ConditionAcute Lymphoblastic Leukemia
Principal InvestigatorDavid Loeb
Contact
Email Contact
Trial DescriptionDocumentation and Delivery of Guideline-Consistent Treatment in Adolescent and Young Adult (AYA) Acute Lymphoblastic Leukemia (ALL)
Disease/ConditionAcute Lymphoblastic Leukemia
Principal InvestigatorDavid Loeb
Contact
Email Contact
Trial DescriptionEffect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial
Disease/ConditionAsthma
Principal InvestigatorSunit Jariwala
Contact
857-218-5336
 Email Contact
Trial DescriptionEfficacy & Safety of Pharmacokinetically-Driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis- a Double-Blind Placebo Controlled Clinical Trial
Disease/ConditionLupus Nephritis
Principal InvestigatorDawn Wahezi
Contact
513-803-2118
 Email Contact
Trial DescriptionEfficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients
Disease/ConditionAnaplastic Medulloblastoma
Medulloblastoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionEliminating Monitor Overuse (EMO) Hybrid Effectiveness-Deimplementation Trial
Disease/ConditionBronchiolitis Acute Viral
Principal InvestigatorAlyssa Silver
Contact
267-426-2901
 Email Contact
Trial DescriptionEROS: Engendering Reproductive Health Within Oncologic Survivorship
Disease/ConditionMalignant Neoplasm
Principal InvestigatorDella Makower
Contact
Email Contact
Trial DescriptionEvaluation of the Asthma Management Program to Promote Activity for Students in Schools (Asthma-PASS)
Disease/ConditionAsthma in Children
Principal InvestigatorMarina Reznik
Contact
Email Contact
Trial DescriptionEvaluation of the Asthma Management Program to Promote Activity for Students in Schools (Asthma-PASS)
Disease/ConditionAsthma in Children
Principal InvestigatorMarina Reznik
Contact
Email Contact
Trial DescriptionEvolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma
Disease/ConditionRhabdomyosarcoma
Principal InvestigatorAlice Lee
Contact
813-745-6250
 Email Contact
Trial DescriptionGenetic Modifiers of 22q11.2 Deletion Syndrome
Disease/ConditionDiGeorge Syndrome
22q11.2 Deletion Syndrome
Principal InvestigatorBernice Morrow
Contact
914-329-4653
 Email Contact
Trial DescriptionImplementing Models for Mechanical Circulatory Support Presurgical Assessment in Congenital Heart Disease Treatment
Disease/ConditionCongenital Heart Disease
Principal InvestigatorOmar Saeed
Contact
212-305-8509
 Email Contact
Trial DescriptionImproving Patient-Centered Communication in Breast Cancer: A RCT of a Shared Decision Engagement System (SHARES)
Disease/ConditionAnatomic Stage 0 Breast Cancer AJCC v8
Anatomic Stage I Breast Cancer AJCC v8
Anatomic Stage IA Breast Cancer AJCC v8
Anatomic Stage IB Breast Cancer AJCC v8
Anatomic Stage II Breast Cancer AJCC v8
Anatomic Stage IIA Breast Cancer AJCC v8
Anatomic Stage IIB Breast Cancer AJCC v8
Anatomic Stage III Breast Cancer AJCC v8
Anatomic Stage IIIA Breast Cancer AJCC v8
Anatomic Stage IIIB Breast Cancer AJCC v8
Anatomic Stage IIIC Breast Cancer AJCC v8
Prognostic Stage 0 Breast Cancer AJCC v8
Prognostic Stage I Breast Cancer AJCC v8
Prognostic Stage IA Breast Cancer AJCC v8
Prognostic Stage IB Breast Cancer AJCC v8
Prognostic Stage II Breast Cancer AJCC v8
Prognostic Stage IIA Breast Cancer AJCC v8
Prognostic Stage IIB Breast Cancer AJCC v8
Prognostic Stage III Breast Cancer AJCC v8
Prognostic Stage IIIA Breast Cancer AJCC v8
Prognostic Stage IIIB Breast Cancer AJCC v8
Prognostic Stage IIIC Breast Cancer AJCC v8
Principal InvestigatorSheldon Feldman
Contact
734-936-8816
 Email Contact
Trial DescriptionInternational Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones
Disease/ConditionAcute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia
Mixed Phenotype Acute Leukemia
T Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionInternational Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones
Disease/ConditionAcute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia
Mixed Phenotype Acute Leukemia
T Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionJ-Valve TF Early Feasibility Study
Disease/ConditionAortic Valve Disease
Aortic Valve Insufficiency
Aortic Regurgitation
Principal InvestigatorMohamed Azeem Latib
Contact
Email Contact
Trial DescriptionLarotrectinib (LOXO-101, NSC# 788607) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
Disease/ConditionCentral Nervous System Neoplasm
Infantile Fibrosarcoma
Recurrent Acute Leukemia
Refractory Acute Leukemia
Solid Neoplasm
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionLong-Term Follow-Up (LTFU) of Participants Treated With ADP Adoptive Cell Therapies
Disease/ConditionNeoplasms
Principal InvestigatorDavid Loeb
Contact
215-825-9260
 Email Contact
Trial DescriptionMaster Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered NY-ESO-1-Specific (c259) T Cells, Alone or in Combination With Other Agents, in HLA-A2+ Participants With NY-ESO-1 and/or LAGE-1a Positive Solid Tumors (IGNYTE-ESO)
Disease/ConditionNeoplasms
Principal InvestigatorDavid Loeb
Contact
Email Contact
Trial DescriptionMaster Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered NY-ESO-1-Specific (c259) T Cells, Alone or in Combination With Other Agents, in HLA-A2+ Participants With NY-ESO-1 and/or LAGE-1a Positive Solid Tumors (IGNYTE-ESO)
Disease/ConditionNeoplasms
Principal InvestigatorDavid Loeb
Contact
Email Contact
Trial DescriptionMatched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome
Disease/ConditionRecurrent, Refractory, or High Risk Leukemias
Matched Targeted Therapy
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionMetformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Principal Investigator
Contact
+39(051)6366668
 Email Contact
Trial DescriptionMinimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease
Disease/ConditionSickle Cell Disease
Principal InvestigatorMichelle Lee
Contact
202-476-5000
 Email Contact
Trial DescriptionMOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study
Disease/ConditionHemophilia A
Principal InvestigatorWilliam Mitchell
Contact
404-785-1637
 Email Contact
Trial DescriptionMolecular Analysis for Combination Therapy Choice (ComboMATCH)
Disease/ConditionAdvanced Malignant Solid Neoplasm
Anatomic Stage III Breast Cancer AJCC v8
Anatomic Stage IV Breast Cancer AJCC v8
Locally Advanced Malignant Solid Neoplasm
Malignant Female Reproductive System Neoplasm
Metastatic HER2-Negative Breast Carcinoma
Metastatic Malignant Solid Neoplasm
Recurrent Endometrial Carcinoma
Recurrent Fallopian Tube Carcinoma
Recurrent Malignant Female Reproductive System Neoplasm
Recurrent Malignant Solid Neoplasm
Recurrent Ovarian Carcinoma
Recurrent Primary Peritoneal Carcinoma
Unresectable HER2-Negative Breast Carcinoma
Unresectable Malignant Solid Neoplasm
Principal InvestigatorBalazs Halmos
Contact
Email Contact
Trial DescriptionMolecular and Genomic Profiling of Head and Neck Tumors to Explore Biomarkers Associated With Prognosis and Response to Therapy
Disease/ConditionHead and Neck Neoplasms
Principal InvestigatorRichard Smith
Contact
718-920-7054
 Email Contact
Trial DescriptionMolecular Basis of Early Childhood Obesity Programming by Intrauterine Growth Restriction
Disease/ConditionChildhood Obesity
Epigenetics
Principal InvestigatorMamta Fuloria
Contact
718-904-4105
 Email Contact
Trial DescriptionMulti-Center African-American Inflammatory Bowel Disease Study (MAAIS)
Disease/ConditionInflammatory Bowel Disease
Principal InvestigatorRuby Greywoode
Contact
410-614-1982
 Email Contact
Trial DescriptionMulticenter Cohort Study To Evaluate Outcomes After Receipt of Targeted Therapy Matched to an Individualized Cancer Therapy (iCat) Recommendations in Children and Young Adults With Solid Tumors: The iCat2, GAIN Consortium Study
Disease/ConditionPediatric Solid Tumor
Principal InvestigatorDaniel Weiser
Contact
Email Contact
Trial DescriptionMulticenter, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Subjects with Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure of Rituximab or Rituximab and Chemotherapy
Disease/ConditionEpstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
Solid Organ Transplant Complications
Lymphoproliferative Disorders
Allogeneic Hematopoietic Cell Transplant
Stem Cell Transplant Complications
Principal InvestigatorIoannis Mantzaris
Contact
650-278-8930
 Email Contact
Trial DescriptionMulticenter, Prospective, Open-label, Randomized, Crossover Study to Evaluate Pharmacokinetics (PK), Safety, and Tolerability of TAK-881 in Primary Immunodeficiency Diseases (PIDD)
Disease/ConditionPrimary Immunodeficiency Diseases (PID)
Principal InvestigatorArye Rubinstein
Contact
+1-877-825-3327
 Email Contact
Trial DescriptionMulti-Institution Registry of SRS/SBRT Procedures
Disease/ConditionNeoplasms
Arteriovenous Malformation of Central Nervous System
Trigeminal Neuralgia
Principal InvestigatorMadhur Garg
Contact
Email Contact
Trial DescriptionNational Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children With Complex Congenital Heart Disease
Disease/ConditionHypoplastic Left Heart Syndrome (HLHS)
Principal InvestigatorNicole Sutton
Contact
Email Contact
Trial DescriptionNatural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections
Disease/ConditionMycobacterium Infections
Principal InvestigatorSteven Porcelli
Contact
(301) 443-2163
 Email Contact
Trial DescriptionNCI COVID-19 in Cancer Patients Study (NCCAPS): A Longitudinal Natural History Study
Disease/ConditionCOVID-19 Infection
Hematopoietic and Lymphatic System Neoplasm
Malignant Solid Neoplasm
Metastatic Malignant Solid Neoplasm
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations
Disease/ConditionRecurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Rhabdoid Tumor
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Recurrent WHO Grade 2 Glioma
Refractory Ependymoma
Refractory Ewing Sarcoma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Refractory WHO Grade 2 Glioma
Wilms Tumor
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Erdafitinib in Patients With Tumors Harboring FGFR1/2/3/4 Alterations
Disease/ConditionAdvanced Malignant Solid Neoplasm
Recurrent Childhood Ependymoma
Recurrent Childhood Malignant Germ Cell Tumor
Recurrent Childhood Medulloblastoma
Recurrent Childhood Non-Hodgkin Lymphoma
Recurrent Childhood Osteosarcoma
Recurrent Childhood Rhabdomyosarcoma
Recurrent Childhood Soft Tissue Sarcoma
Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Neuroblastoma
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdoid Tumor
Refractory Childhood Malignant Germ Cell Tumor
Refractory Childhood Osteosarcoma
Refractory Childhood Rhabdomyosarcoma
Refractory Childhood Soft Tissue Sarcoma
Refractory Ependymoma
Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Wilms Tumor
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-101 (Larotrectinib) in Patients With Tumors Harboring Actionable NTRK Fusions
Disease/ConditionAdvanced Malignant Solid Neoplasm
Recurrent Ependymoma
Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Recurrent Glioma
Recurrent Hepatoblastoma
Recurrent Kidney Wilms Tumor
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Rhabdoid Tumor
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Refractory Ependymoma
Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Refractory Glioma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-292 in Patients With Tumors Harboring RET Gene Alterations
Disease/ConditionHematopoietic and Lymphatic System Neoplasm
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Hepatoblastoma
Recurrent Histiocytic and Dendritic Cell Neoplasm
Recurrent Langerhans Cell Histiocytosis
Recurrent Lymphoma
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Rhabdoid Tumor
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Recurrent WHO Grade 2 Glioma
Refractory Ependymoma
Refractory Ewing Sarcoma
Refractory Hepatoblastoma
Refractory Histiocytic and Dendritic Cell Neoplasm
Refractory Langerhans Cell Histiocytosis
Refractory Lymphoma
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Refractory WHO Grade 2 Glioma
Wilms Tumor
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Ensartinib in Patients With Tumors Harboring ALK or ROS1 Genomic Alterations
Disease/ConditionAdvanced Malignant Solid Neoplasm
Malignant Solid Neoplasm
Recurrent Ependymoma
Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdoid Tumor
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Refractory Ependymoma
Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Wilms Tumor
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice)- Phase 2 Subprotocol of LY3023414 in Patients With Solid Tumors
Disease/ConditionAdvanced Malignant Solid Neoplasm
Ann Arbor Stage III Non-Hodgkin Lymphoma
Ann Arbor Stage IV Non-Hodgkin Lymphoma
Malignant Glioma
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Glioma
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Solid Neoplasm
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Primary Central Nervous System Neoplasm
Rhabdoid Tumor
Stage III Osteosarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Osteosarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Stage IVA Osteosarcoma AJCC v7
Stage IVB Osteosarcoma AJCC v7
Wilms Tumor
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Tipifarnib in Patients With Tumors Harboring HRAS Genomic Alterations
Disease/ConditionMalignant Solid Neoplasm
Recurrent Adrenal Gland Pheochromocytoma
Recurrent Ectomesenchymoma
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Hepatoblastoma
Recurrent Kidney Wilms Tumor
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Medulloblastoma
Recurrent Melanoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Rhabdoid Tumor
Recurrent Rhabdoid Tumor of the Kidney
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Recurrent Thyroid Gland Carcinoma
Recurrent WHO Grade 2 Glioma
Refractory Adrenal Gland Pheochromocytoma
Refractory Ependymoma
Refractory Ewing Sarcoma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Medulloblastoma
Refractory Melanoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Rhabdoid Tumor
Refractory Rhabdoid Tumor of the Kidney
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Refractory Thyroid Gland Carcinoma
Refractory WHO Grade 2 Glioma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
Disease/ConditionAdvanced Malignant Solid Neoplasm
Ann Arbor Stage III Non-Hodgkin Lymphoma
Ann Arbor Stage IV Non-Hodgkin Lymphoma
Histiocytic Sarcoma
Juvenile Xanthogranuloma
Langerhans Cell Histiocytosis
Malignant Glioma
Recurrent Childhood Rhabdomyosarcoma
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Glioma
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdoid Tumor
Recurrent Soft Tissue Sarcoma
Refractory Ewing Sarcoma
Refractory Glioma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Rhabdoid Tumor
Stage III Osteosarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Osteosarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Stage IVA Osteosarcoma AJCC v7
Stage IVB Osteosarcoma AJCC v7
Wilms Tumor
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionNephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network
Disease/ConditionMinimal Change Disease (MCD)
Membranous Nephropathy
Glomerulosclerosis, Focal Segmental
Principal InvestigatorFrederick Kaskel
Contact
734-615-5021
 Email Contact
Trial DescriptionNeuroblastoma Biology Studies
Disease/ConditionGanglioneuroblastoma
Localized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Regional Neuroblastoma
Stage 4 Neuroblastoma
Stage 4S Neuroblastoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNeuropsychological, Social, Emotional, and Behavioral Outcomes in Children With Cancer
Disease/ConditionChildhood Malignant Neoplasm
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionNMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Disease/ConditionNeuroblastoma
Principal InvestigatorDaniel Weiser
Contact
7175310003
 Email Contact
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry
Disease/ConditionRheumatic Joint Disease
Principal InvestigatorDawn Wahezi
Contact
(919) 419-5032
 Email Contact
Trial DescriptionONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study
Disease/ConditionH3 K27M
Glioma
Principal InvestigatorAdilia Hormigo
Contact
1-919-806-1074
 Email Contact
Trial DescriptionOptimize PRO Transcatheter Aortic Valve Replacement Post Market Study
Disease/ConditionSymptomatic Aortic Stenosis
Principal InvestigatorMohamed Azeem Latib
Contact
Email Contact
Trial DescriptionOptimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure?
Disease/ConditionPain, Procedural
Principal InvestigatorCourtney Mcnamara
Contact
540 514 4389
 Email Contact
Trial DescriptionPazopanib Neoadjuvant Trial in Non-Rhabdomyosarcoma Soft Tissue Sarcomas (PAZNTIS): A Phase II/III Randomized Trial of Preoperative Chemoradiation or Preoperative Radiation Plus or Minus Pazopanib (NSC# 737754)
Disease/ConditionAlveolar Soft Part Sarcoma
Angiomatoid Fibrous Histiocytoma
Atypical Fibroxanthoma
Clear Cell Sarcoma of Soft Tissue
Epithelioid Malignant Peripheral Nerve Sheath Tumor
Epithelioid Sarcoma
Extraskeletal Myxoid Chondrosarcoma
Extraskeletal Osteosarcoma
Fibrohistiocytic Neoplasm
Fibrosarcoma
Inflammatory Myofibroblastic Tumor
Intimal Sarcoma
Leiomyosarcoma
Liposarcoma
Liver Embryonal Sarcoma
Low Grade Fibromyxoid Sarcoma
Low Grade Myofibroblastic Sarcoma
Malignant Peripheral Nerve Sheath Tumor
Malignant Skin Granular Cell Tumor
Malignant Triton Tumor
Mesenchymal Chondrosarcoma
Myxofibrosarcoma
Myxoid Chondrosarcoma
Myxoinflammatory Fibroblastic Sarcoma
Nerve Sheath Neoplasm
PEComa
Pericytic Neoplasm
Plexiform Fibrohistiocytic Tumor
Sclerosing Epithelioid Fibrosarcoma
Skin Glomus Tumor
Stage IB Soft Tissue Sarcoma AJCC v7
Stage IIB Soft Tissue Sarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Synovial Sarcoma
Undifferentiated High Grade Pleomorphic Sarcoma of Bone
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionPediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias
Disease/ConditionAcute Lymphoblastic Leukemia
Acute Myeloid Leukemia
Acute Myeloid Leukemia Post Cytotoxic Therapy
Juvenile Myelomonocytic Leukemia
Mixed Phenotype Acute Leukemia
Myelodysplastic Syndrome
Myelodysplastic Syndrome Post Cytotoxic Therapy
Myeloid Leukemia Associated With Down Syndrome
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionPediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
Disease/ConditionChildhood Hepatocellular Carcinoma
Childhood Malignant Liver Neoplasm
Fibrolamellar Carcinoma
Hepatoblastoma
Hepatocellular Malignant Neoplasm, Not Otherwise Specified
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionPHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS
Disease/ConditionEwing Sarcoma
Solid Tumors
Rhabdoid Tumor
Rhabdomyosarcoma
Neuroblastoma
Medulloblastoma
Diffuse Intrinsic Pontine Glioma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionPhase 1-2 Trial of Gamunex (intravenous Gammaglobulin) for Sickle Cell Acute Pain
Disease/ConditionSickle Cell Disease
Pain
Principal InvestigatorDeepa Manwani
Contact
718-741-2342
 Email Contact
Trial DescriptionPhase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis
Disease/ConditionRecurrent Langerhans Cell Histiocytosis
Refractory Langerhans Cell Histiocytosis
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionPhase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders
Disease/ConditionAcute Lymphoblastic Leukemia
Leukemia, Acute Myeloid (AML), Child
Lymphoma, Non-Hodgkin
Myelodysplastic Syndromes
Primary Immune Deficiency Disorder
Osteopetrosis
Cytopenia
Hemoglobinopathy in Children
Anemia, Aplastic
Principal InvestigatorMichelle Lee
Contact
Email Contact
Trial DescriptionPhase II Study of Nab-Paclitaxel in Combination With Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Rhabdomyosarcoma
Soft Tissue Sarcoma
Principal InvestigatorDaniel Weiser
Contact
Email Contact
Trial DescriptionPhase II Study of Nab-Paclitaxel in Combination With Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Rhabdomyosarcoma
Soft Tissue Sarcoma
Principal InvestigatorDaniel Weiser
Contact
Email Contact
Trial DescriptionPhase III Multicenter Open-label Randomized Clinical Trial Comparing Everolimus and Low Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil at 6 mo Post-Transplant to Prevent Long-term Complications After Pediatric Heart Transplantation
Disease/ConditionPediatric Heart Transplantation
Immunosuppression
Chronic Kidney Diseases
Cardiac Allograft Vasculopathy
Heart Transplant Failure and Rejection
Post-transplant Lymphoproliferative Disorder
Heart Transplant Infection
Principal InvestigatorDaphne Hsu
Contact
Email Contact
Trial DescriptionPhase III Randomized Trial of Post-Radiation Chemotherapy in Patients With Newly Diagnosed Ependymoma Ages 1 to 21 Years
Disease/ConditionAnaplastic Ependymoma
Brain Ependymoma
Cellular Ependymoma
Clear Cell Ependymoma
Ependymoma
Papillary Ependymoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionPhosphate Binder Therapy and Chronic Kidney Disease in Children
Disease/ConditionChronic Kidney Diseases
Principal InvestigatorFrederick Kaskel
Contact
310-7943144
 Email Contact
Trial DescriptionProspective Cohort Study to Evaluate Immunologic Response Following COVID-19 Vaccination in Children, Adolescents and Young Adults With Cancer
Disease/ConditionCOVID-19 Infection
Hematopoietic and Lymphatic System Neoplasm
Malignant Solid Neoplasm
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionProtocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries
Disease/ConditionAutologous Stem Cell Transplantation
Allogeneic Stem Cell Transplantation
Solid Tumors
Blood Cancers
CAR-T
Gene Therapy
Non-malignant Disease
Principal InvestigatorMichelle Lee
Contact
763 406 3280
 Email Contact
Trial DescriptionRandomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial
Disease/ConditionCmv Congenital
CMV
Congenital Cmv
SNHL
Sensorineural Hearing Loss
Principal InvestigatorMichel Nassar
Contact
Email Contact
Trial DescriptionRandomized Phase 3 Trial Evaluating the Addition of the IGF-1R Monoclonal Antibody Ganitumab (AMG 479, NSC# 750008) to Multiagent Chemotherapy for Patients With Newly Diagnosed Metastatic Ewing Sarcoma
Disease/ConditionMetastatic Ewing Sarcoma
Metastatic Malignant Neoplasm in the Bone
Metastatic Malignant Neoplasm in the Bone Marrow
Metastatic Malignant Neoplasm in the Lung
Metastatic Peripheral Primitive Neuroectodermal Tumor of Bone
Peripheral Primitive Neuroectodermal Tumor of Soft Tissues
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionRandomized Study of Intravenous Calaspargase Pegol (SC-PEG Asparaginase) and Intravenous Oncaspar in Children and Adolescents With Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma
Disease/ConditionAcute Lymphoblastic Leukemia
Lymphoblastic Lymphoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionREal-World Analyses of Stroke - Thrombus Occlusion REtrieval
Disease/ConditionAcute Ischemic Stroke
Vessel Occlusion
Stroke
Ischemic
Principal InvestigatorNeil Haranhalli
Contact
714.920.3815
 Email Contact
Trial DescriptionRegistry of Asthma Patients Initiating DUPIXENT® (RAPID)
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Contact
Email Contact
Trial DescriptionRenal Tumors Classification, Biology, and Banking Study - PRIMARY OBJECTIVES: I. Classify patients with renal tumors by histological categorization, surgico-pathological stage, presence of metastases, age at diagnosis, tumor weight, and loss of heterozygosity for chromosomes 1p and 16q, to define eligibility for a series of therapeutic studies. (As of Amendment 8, this aim is limited to patients with institutionally classified Stage I to IV focal or diffuse anaplasia identified at initial biopsy or primary nephrectomy OR at delayed nephrectomy/second biopsy.) II. To maintain a biological samples bank to make specimens available to scientists to evaluate additional potential biological prognostic variables and for the conduct of other research by scientists. SECONDARY OBJECTIVES: I. To monitor outcome for those patients who are not eligible for a subsequent therapeutic study. II. To describe whether the pulmonary tumor burden correlates with outcome in Stage IV patients.(Completed as of Amendment 7) III. To describe the sensitivity and specificity of abdominal computed tomography (CT) by comparison with surgical and pathologic findings for identification of local tumor spread beyond the renal capsule to adjacent muscle and organs, lymph node involvement at the renal hilum and in the retroperitoneum, preoperative tumor rupture and metastases to the liver. (Completed as of Amendment 7) IV. To compare the sensitivity and specificity of preoperative abdominal CT and MRI for the identification and differentiation of nephrogenic rests and Wilms tumor in children with multiple renal lesions. (Completed as of Amendment 7) V. To correlate the method of conception (natural versus assisted reproductive technology) with the development of Wilms tumor. (Completed as of Amendment 7) VI. To evaluate the frequency of integrase interactor 1 (INI1) mutations in renal and extrarenal malignant rhabdoid tumor of the kidney and to determine the incidence of germline and inherited versus somatic mutations to facilitate clinical correlations on the companion study AREN0321. (Completed as of Amendment 7) OUTLINE: Tumor tissue, blood, and urine samples are collected for research studies, including immunohistochemistry. CT scans and MRIs are also performed. Loss of heterozygosity analyses (chromosome 1p and 16q) are performed by extraction of DNA. DNA polymorphisms are assayed by polymerase chain reaction using standard methodology. Leftover specimens are archived for future studies. (LOH and INI1 testing discontinued as of April 2014) Patients are followed up periodically for 5 years.
Disease/ConditionAdult Cystic Nephroma
Anaplastic Kidney Wilms Tumor
Angiolipoma
Cellular Congenital Mesoblastic Nephroma
Classic Congenital Mesoblastic Nephroma
Clear Cell Sarcoma of the Kidney
Congenital Mesoblastic Nephroma
Cystic Partially Differentiated Kidney Nephroblastoma
Diffuse Hyperplastic Perilobar Nephroblastomatosis
Extrarenal Rhabdoid Tumor
Kidney Medullary Carcinoma
Kidney Neoplasm
Kidney Oncocytoma
Kidney Wilms Tumor
Metanephric Adenofibroma
Metanephric Adenoma
Metanephric Stromal Tumor
Metanephric Tumor
Mixed Congenital Mesoblastic Nephroma
Ossifying Renal Tumor of Infancy
Papillary Renal Cell Carcinoma
Renal Cell Carcinoma
Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions
Rhabdoid Tumor of the Kidney
Wilms Tumor
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionRisk-Stratified Randomized Phase III Testing of Blinatumomab (NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionRisk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
Disease/ConditionAcute Myeloid Leukemia
Down Syndrome
Myelodysplastic Syndrome
Myeloid Leukemia Associated With Down Syndrome
Myeloproliferative Neoplasm
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionRisk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
Disease/ConditionAcute Myeloid Leukemia
Down Syndrome
Myelodysplastic Syndrome
Myeloid Leukemia Associated With Down Syndrome
Myeloproliferative Neoplasm
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionSickle Cell Disease (SCD) Biochip': Towards a Simple and Reliable Way to Monitor Sickle Cell Disease
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Contact
(216) 368-6447
 Email Contact
Trial DescriptionSimple Bone Cysts in Kids (SBoCK)
Disease/ConditionBone Cyst
Principal InvestigatorJacob Schulz
Contact
Email Contact
Trial DescriptionSingle-arm, Open-label, Phase 1b/2 Trial of Nivolumab Therapy Following Partially HLA Mismatched (Haploidentical) Bone Marrow Transplant in Children and Young Adults With High Risk, Recurrent or Refractory Sarcomas
Disease/ConditionSarcoma
Solid Tumor, Adult
Solid Tumor, Childhood
Principal InvestigatorDavid Loeb
Contact
410-955-0432
 Email Contact
Trial DescriptionStopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP)
Disease/ConditionChronic Phase Chronic Myeloid Leukemia, BCR-ABL1 Positive
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionStudy for the Prediction of Active Rejection in Organs Using Donor-derived Cell-free DNA Detection
Disease/ConditionTransplant Rejection
Allograft Rejection
Complications
Principal InvestigatorNicole Hayde
Contact
6502499090
 Email Contact
Trial DescriptionSurveillance HeartCare® Outcomes Registry
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Contact
Email Contact
Trial DescriptionTesting the Feasibility of a Teletherapy Plus Parent Coaching Intervention for Children With Autism
Disease/ConditionAutism Spectrum Disorder
Principal InvestigatorElizabeth Ridgway
Contact
6102912179
 Email Contact
Trial DescriptionThe Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study
Disease/ConditionAdrenal Gland Pheochromocytoma
Carcinoma In Situ
Central Nervous System Neoplasm
Childhood Immature Teratoma
Childhood Langerhans Cell Histiocytosis
Childhood Mature Teratoma
Congenital Mesoblastic Nephroma
Desmoid Fibromatosis
Ganglioneuroma
Lymphoproliferative Disorder
Malignant Neoplasm
Malignant Solid Neoplasm
Melanocytic Neoplasm
Myeloproliferative Neoplasm
Neoplasm of Uncertain Malignant Potential
Neuroendocrine Neoplasm
Stromal Neoplasm
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionThe Tether™ - Vertebral Body Tethering System Post Approval Study
Disease/ConditionScoliosis Idiopathic
Principal InvestigatorJaime Gomez
Contact
1-303-465-8960
 Email Contact
Trial DescriptionTransvenous Approach for the Treatment of Cerebral Arteriovenous Malformations (TATAM): A Randomized Controlled Trial and Registry
Disease/ConditionArteriovenous Malformations, Cerebral
Unruptured Brain Arteriovenous Malformation
Ruptured Brain Arteriovenous Malformation
Principal InvestigatorDavid Altschul
Contact
Email Contact
Trial DescriptionTransvenous Approach for the Treatment of Cerebral Arteriovenous Malformations (TATAM): A Randomized Controlled Trial and Registry
Disease/ConditionArteriovenous Malformations, Cerebral
Unruptured Brain Arteriovenous Malformation
Ruptured Brain Arteriovenous Malformation
Principal InvestigatorDavid Altschul
Contact
Email Contact
Trial DescriptionTreatment of Children With All Stages of Hepatoblastoma With Temsirolimus (NSC#683864) Added to High Risk Stratum Treatment
Disease/ConditionPRETEXT I Hepatoblastoma
PRETEXT II Hepatoblastoma
PRETEXT III Hepatoblastoma
PRETEXT IV Hepatoblastoma
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionTreatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents
Disease/ConditionAcute Lymphoblastic Leukemia, Pediatric
Principal InvestigatorLisa Gennarini
Contact
Email Contact
Trial DescriptionTreatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
Disease/ConditionAnaplastic Kidney Wilms Tumor
Recurrent Kidney Wilms Tumor
Stage II Kidney Wilms Tumor
Stage III Kidney Wilms Tumor
Stage IV Kidney Wilms Tumor
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionTreatment of Patients With Newly Diagnosed Standard Risk B-Lymphoblastic Leukemia (B-ALL) or Localized B-Lineage Lymphoblastic Lymphoma (B-LLy)
Disease/ConditionAcute Lymphoblastic Leukemia
Adult B Lymphoblastic Lymphoma
Ann Arbor Stage I B Lymphoblastic Lymphoma
Ann Arbor Stage II B Lymphoblastic Lymphoma
Childhood B Acute Lymphoblastic Leukemia
Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1
Childhood B Lymphoblastic Lymphoma
Down Syndrome
Hypodiploid B Acute Lymphoblastic Leukemia
Philadelphia Chromosome Positive
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionTrialNet Pathway to Prevention of T1D
Disease/ConditionDiabetes Mellitus, Type 1
Principal InvestigatorLisa Underland
Contact
1-800-425-8361
 Email Contact
Trial DescriptionTrifecta-Heart cfDNA-MMDX Study: Comparing the DD-cfDNA Test to MMDx Microarray Test and Central HLA Antibody Test
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Contact
1 780 782 9463
 Email Contact
Trial DescriptionTumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial
Disease/ConditionSolid Tumors
Principal InvestigatorHaiying Cheng
Contact
888-662-6728 (U.S. and Canada)
 Email Contact
Trial DescriptionUmbrella Long-Term Follow-Up Protocol
Disease/ConditionHematopoietic Cell Transplantation Recipient
Leukemia
Solid Tumor
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionUS Selumetinib Registry
Disease/ConditionNeurofibromatosis Type 1
Plexiform Neurofibromas
Principal InvestigatorNagma Dalvi
Contact
1-855-752-2356
 Email Contact
Trial DescriptionUtilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients With Non-High-Risk Neuroblastoma
Disease/ConditionGanglioneuroblastoma
Localized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Neuroblastoma
Principal InvestigatorAlice Lee
Contact
Email Contact
Trial DescriptionViral Infection and Respiratory Illness Universal Study[VIRUS]: COVID-19 Registry and Validation of C2D2 (Critical Care Data Dictionary)
Disease/ConditionCoronavirus
Principal InvestigatorJen-Ting Chen
Contact
5072557196
 Email Contact
View all Clinical Trials