Research and Clinical Trials

Asthma

A 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

Asthma

A 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

Inflammatory Bowel Diseases, Crohn's Disease, Ulcerative Colitis, Indeterminate Colitis

A 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease

• 9842340268
• ldalfonso@targetpharmasolutions.com

Acute Lymphoblastic Leukemia

A Comprehensive Approach to Improve Medication Adherence in Pediatric ALL

Acute Lymphoblastic Leukemia

A Comprehensive Approach to Improve Medication Adherence in Pediatric ALL

Biliary Atresia

A 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

Biliary Atresia

A 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

Atopic Dermatitis, Alopecia Areata, Hidradenitis Suppurativa, Vitiligo, Psoriasis, Chronic Spontaneous Urticaria

A Longitudinal Observational Study of Patients Undergoing Therapy for Immune-Mediated Inflammatory Skin Conditions

• 9842340268
• ldalfonso@targetrwe.com

Nonalcoholic Fatty Liver, Nonalcoholic Steatohepatitis

A Longitudinal Observational Study of Patients With Nonalcoholic Steatohepatitis (NASH) and Related Conditions Across the Entire Spectrum of Nonalcoholic Fatty Liver Disease (NAFLD)

• emichalski@targetrwe.com

Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex

A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex

A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Hematologic 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

A 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

• 10-CBA@ndmp.org

Infusion Reactions

A 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

• 718-706-5202
• dsung@nybc.org

Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndrome

A 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)

Crohn's Disease, Ulcerative Colitis, Indeterminate Colitis, Inflammatory Bowel Diseases

A Multicenter, Prospective, Long-term, Observational Registry of Pediatric Patients With Inflammatory Bowel Disease

Asthma

A Multi-centre, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety of GSK3511294 (Depemokimab) in Adult and Adolescent Participants With Severe Asthma With an Eosinophilic Phenotype From Studies 206713 or 213744

Pediatric Sarcoma, Refractory, Pediatric Sarcoma, Relapsed

A 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

• 813-745-6250
• jessica.crimella@moffitt.org

Ovarian Cancer, Endometrial Cancer, Germ Cell Tumor, Testicular Germ Cell Tumor, Ovarian Germ Cell Tumor

A Phase 1, First-in-Human, Dose Escalation and Expansion Study to Evaluate the Safety and Tolerability of XmAb541 in Advanced Solid Tumors

• 1-858-828-6286
• cbohac@xencor.com

Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia, Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma

A 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)

Anaplastic Astrocytoma, Anaplastic Astrocytoma, Not Otherwise Specified, Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Glioblastoma, Glioblastoma, Not Otherwise Specified, Malignant Glioma

A 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)

Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Mixed Lineage Acute Leukemia, Mixed Phenotype Acute Leukemia, Acute Leukemia of Ambiguous Lineage

A 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

• 781-419-1400
• clinicaltrials@syndax.com

High Risk Neuroblastoma, Recurrent Neuroblastoma, Refractory Neuroblastoma

A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma

High Risk Neuroblastoma, Recurrent Neuroblastoma, Refractory Neuroblastoma

A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma

Anaplastic Astrocytoma, Anaplastic Astrocytoma, Not Otherwise Specified, Anaplastic Ganglioglioma, Anaplastic Pleomorphic Xanthoastrocytoma, Glioblastoma, Malignant Glioma, WHO Grade 3 Glioma

A Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG)

Recurrent B Acute Lymphoblastic Leukemia, Recurrent B Lymphoblastic Lymphoma, Refractory B Acute Lymphoblastic Leukemia, Refractory B Lymphoblastic Lymphoma

A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518) in Children and Young Adults With Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)

Medulloblastoma

A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients

Central 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

A 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

Sickle Cell Disease (SCD)

A 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

Proteinuric Kidney Disease

A 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

• 617-341-6777
• medicalinfo@vrtx.com

Heart Failure, Left Ventricular Systolic Dysfunction

A 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)

• 1-888-577-8839
• Trialsites@msd.com

Acquired Immune Deficiency Syndrome (AIDS), HIV Infections

A 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

Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)

A 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

Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)

A 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

Arthritis, Juvenile

A 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)

• 844-434-4210
• Participate-In-This-Study@its.jnj.com

Metastatic Malignant Neoplasm in the Lung, Metastatic Osteosarcoma, Osteosarcoma

A Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma

Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma

A 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)

Low Grade Glioma, Neurofibromatosis Type 1, Visual Pathway Glioma

A Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

Acute Myeloid Leukemia

A 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

Acute Myeloid Leukemia

A 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

B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Central Nervous System Leukemia, Mixed Phenotype Acute Leukemia, Testicular Leukemia

A 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

Central Nervous System Carcinoma

A 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

Atopic Dermatitis

A Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Upadacitinib in Adolescent and Adult Subjects With Moderate to Severe Atopic Dermatitis

Ganglioneuroblastoma, Neuroblastoma

A 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)

Ganglioneuroblastoma, Neuroblastoma

A 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)

Childhood 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

A 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

Pre-Exposure Prophylaxis of HIV Infection

A 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

Pre-Exposure Prophylaxis of HIV Infection

A 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

Areata Alopecia, Alopecia, Hypotrichosis, Hair Diseases, Skin Diseases, Pathological Conditions, Anatomical

A 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

• -317-615-4559
• ClinicalTrials.gov@lilly.com

Contraception

A Phase 3, Multicenter, Open-Label, Single Arm Study of MR-100A-01 in Women of Childbearing Potential to Evaluate Contraceptive Efficacy and Safety

Immune Thrombocytopenia

A 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)

Irritable Bowel Syndrome With Constipation, Functional Constipation

A 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)

Irritable Bowel Syndrome With Constipation, Functional Constipation

A 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)

Contraception

A 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

Contraception

A 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

Atopic Dermatitis

A Phase 3, Randomized, 52-week, Placebo-controlled, Double-blind Study With Rerandomization to Assess the Efficacy, Safety, and Tolerability of Rocatinlimab (AMG 451) in Adolescent Subjects With Moderate-to-severe Atopic Dermatitis (AD) (ROCKET-ASTRO)

Migraine

A 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

• 1-800-718-1021
• ClinicalTrials.gov_Inquiries@pfizer.com

Chronic Kidney Disease; Iron Deficiency Anemia

A 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)

• 1-877-374 -4177
• CTInterest@covispharma.com

Recurrent Classic Hodgkin Lymphoma, Refractory Classic Hodgkin Lymphoma

A 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

Osteosarcoma, Osteosarcoma in Children, Osteosarcoma Recurrent, Sarcoma

A 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

Osteosarcoma, Osteosarcoma in Children, Osteosarcoma Recurrent, Sarcoma

A 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

Refractory and/or Relapsed Metastatic Solid Tumors

A Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors

• 410-502-4997
• hsymons2@jhmi.edu

B Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia, BCR-ABL1-Like, Central Nervous System Leukemia, Testicular Leukemia

A 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

Acute Myeloid Leukemia, Leukemia Cutis, Myeloid Neoplasm, Myeloid Sarcoma

A 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

Anaplastic Medulloblastoma, Medulloblastoma, Supratentorial Embryonal Tumor, Not Otherwise Specified, Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor

A Phase III Randomized Trial for the Treatment of Newly Diagnosed Supratentorial PNET and High Risk Medulloblastoma in Children < 36 Months Old With Intensive Induction Chemotherapy With Methotrexate Followed by Consolidation With Stem Cell Rescue vs. the Same Therapy Without Methotrexate

Anaplastic Medulloblastoma, Medulloblastoma, Supratentorial Embryonal Tumor, Not Otherwise Specified, Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor

A Phase III Randomized Trial for the Treatment of Newly Diagnosed Supratentorial PNET and High Risk Medulloblastoma in Children < 36 Months Old With Intensive Induction Chemotherapy With Methotrexate Followed by Consolidation With Stem Cell Rescue vs. the Same Therapy Without Methotrexate

Adult 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

A 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)

Ann 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

A 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

Ann 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

A 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

Ganglioneuroblastoma, Nodular, Neuroblastoma

A Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma

Subject Presenting Wheeze, COPD, Asthma

A Pilot Study to Assess Aevice Medical Device for Detection of Wheeze in Pediatric Subjects and Use for Remote Auscultation

Cardiomyopathy

A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations

• 650-209-8092
• mpollman@tenayathera.com

Embryonal Rhabdomyosarcoma, Fusion-Negative Alveolar Rhabdomyosarcoma, Spindle Cell/Sclerosing Rhabdomyosarcoma

A Prospective Phase 3 Study of Patients With Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma

Spinal Muscular Atrophy (SMA)

A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

• 1-888-669-6682
• novartis.email@novartis.com

Bile Acid Synthesis Disorders

A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid)

HIV/AIDS

A Randomized Controlled Trial of Women Involved in Supporting Health (WISH), a Peer-Led Intervention to Improve Postpartum Retention in HIV Care

• 215-746-8555
• florence.momplaisir@pennmedicine.upenn.edu

Lugano Classification Limited Stage Hodgkin Lymphoma AJCC v8

A 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

Ann 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

A Randomized Phase 3 Study of Brentuximab Vedotin (SGN-35) for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma (cHL) in Children and Young Adults

Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Rhabdomyosarcoma, Sclerosing Rhabdomyosarcoma, Spindle Cell Rhabdomyosarcoma

A 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)

Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Rhabdomyosarcoma, Sclerosing Rhabdomyosarcoma, Spindle Cell Rhabdomyosarcoma

A 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)

Primary Mediastinal Large B-Cell Lymphoma

A Randomized Phase 3 Trial of Nivolumab (NSC# 748726) in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma

Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Metastatic Embryonal Rhabdomyosarcoma, Metastatic Rhabdomyosarcoma, Solid Alveolar Rhabdomyosarcoma, Spindle Cell Rhabdomyosarcoma, Spindle Cell/Sclerosing Rhabdomyosarcoma

A 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)

Post-Traumatic Headache

A Randomized Study of Dexamethasone as Adjuvant Therapy for Acute Post-traumatic Headache

• 718-920-6626
• befriedm@montefiore.org

B Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, B Acute Lymphoblastic Leukemia, BCR-ABL1-Like, Lymphoblastic Lymphoma, Mixed Phenotype Acute Leukemia, T Acute Lymphoblastic Leukemia

A Randomized Trial of Levocarnitine Prophylaxis to Prevent Asparaginase-Associated Hepatotoxicity in Adolescents and Young Adults Receiving Acute Lymphoblastic Leukemia Therapy

Bowel Preparation

A 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

• +1 833-548-1402 (US/Canada)
• DK0-Disclosure@ferring.com

Aortic Stenosis, Coronary Artery Disease, Coronary Stenosis

A 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

• 604-875-4111
• brobinson@cci-cic.org

Necrotizing Enterocolitis

A 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

Necrotizing Enterocolitis

A 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

Chronic Hepatitis B

A 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

• GS-US-320-1092@gilead.com

Asthma

A 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

Asthma

A 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

Seizures, Lennox Gastaut Syndrome

A 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

• 201-421-3810
• bremes@sklsi.com

Ulcerative Colitis

A 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

• 9082428287
• sandra.narain@bauschhealth.com

Focal Segmental Glomerulosclerosis

A 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

Focal Segmental Glomerulosclerosis

A 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

Acute Myeloid Leukemia (AML), Cancer

A 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)

Acute Myeloid Leukemia (AML), Cancer

A 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)

Systemic Lupus Erythematosus

A Remotely Delivered CBT Intervention for Youth With cSLE: A Multi-site Patient-engaged Investigation

Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis, Unresectable Desmoid Fibromatosis

A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors

Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis, Unresectable Desmoid Fibromatosis

A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors

Medulloblastoma

A 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

Medulloblastoma

A 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

Aortic Stenosis

ACURATE IDE: Transcatheter Replacement of Stenotic Aortic Valve Through Implantation of ACURATE in Subjects InDicatEd for TAVR

Aortic Stenosis

ACURATE IDE: Transcatheter Replacement of Stenotic Aortic Valve Through Implantation of ACURATE in Subjects InDicatEd for TAVR

Sickle Cell Disease

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)

• (+1) 866-867-7178
• clinicaltrials@novonordisk.com

Juvenile Idiopathic Arthritis

An Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA)

Juvenile Idiopathic Arthritis

An Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA)

Progressive Familial Intrahepatic Cholestasis (PFIC)

An 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)

Progressive Familial Intrahepatic Cholestasis (PFIC)

An 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)

Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia

An 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)

• 800-633-1610
• Contact-US@sanofi.com

Epstein-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

An 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)

• 650-278-8930
• clinicalstudies@atarabio.com

Kidney Diseases, Kidney Failure, Kidney Disease, Chronic

APOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO)

• 336-713-4292
• lrussell@wakehealth.edu

Medical Errors, Electronic Medical Records

Assessing the Effectiveness of Pictographs for Preventing Wrong-Patient Errors in Neonatal Intensive Care Units: A Randomized Controlled Trial

• 646-317-4803
• jsa2163@cumc.columbia.edu

Heart Transplant Infection, Kidney Transplant Infection, Liver Transplant Infection, CMV

Assessment of CMV-specific T Cell Responses by Flow Cytometry With Intracellular Cytokine Staining to Predict CMV Infection Risk in Pediatric Solid Organ Transplant Recipients

Acute 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

Cancer Moonshot Biobank Research Protocol

Autism Spectrum Disorder

Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)

• 914-315-4236
• casara.ferretti@einsteinmed.edu

B Acute Lymphoblastic Leukemia Associated With Down Syndrome, Down Syndrome, Myeloid Leukemia Associated With Down Syndrome

Chronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)

Bronchiolitis

Comparison of Conventional Non-Invasive Ventilation and Neurally-Adjusted Ventilatory Assistance (NAVA) Non-Invasive Ventilation for the Treatment of Bronchiolitis

• 2017459825
• jweingar@montefiore.edu

Asthma

Effect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial

• 857-218-5336
• Wanda.Phipatanakul@childrens.harvard.edu

Lupus Nephritis

Efficacy & Safety of Pharmacokinetically-Driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis- a Double-Blind Placebo Controlled Clinical Trial

• 513-803-2118
• plumm@cchmc.org

Anaplastic Medulloblastoma, Medulloblastoma

Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

Anaplastic Medulloblastoma, Medulloblastoma

Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

Bronchiolitis Acute Viral

Eliminating Monitor Overuse (EMO) Hybrid Effectiveness-Deimplementation Trial

• 267-426-2901
• bonafide@chop.edu

Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Central Nervous System Neoplasm, Ewing Sarcoma, Germ Cell Tumor, Leukemia, Lymphoma, Malignant Neoplasm, Neuroblastoma, Osteosarcoma, Retinoblastoma, Rhabdoid Tumor, Rhabdomyosarcoma, Soft Tissue Sarcoma

Establishing Continuous Cell Lines and Xenografts From Pediatric Cancers for Biological and Pre-Clinical Therapeutic Studies

Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Central Nervous System Neoplasm, Ewing Sarcoma, Germ Cell Tumor, Leukemia, Lymphoma, Malignant Neoplasm, Neuroblastoma, Osteosarcoma, Retinoblastoma, Rhabdoid Tumor, Rhabdomyosarcoma, Soft Tissue Sarcoma

Establishing Continuous Cell Lines and Xenografts From Pediatric Cancers for Biological and Pre-Clinical Therapeutic Studies

Rhabdomyosarcoma

Evolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma

• 813-745-6250
• Jessica.Crimella@moffitt.org

DiGeorge Syndrome, 22q11.2 Deletion Syndrome

Genetic Modifiers of 22q11.2 Deletion Syndrome

• 914-329-4653
• bernice.morrow@einsteinmed.edu

Congenital Heart Disease

Implementing Models for Mechanical Circulatory Support Presurgical Assessment in Congenital Heart Disease Treatment

• 212-305-8509
• kf2549@cumc.columbia.edu

Anatomic 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

Improving Patient-Centered Communication in Breast Cancer: A RCT of a Shared Decision Engagement System (SHARES)

• 734-936-8816
• sarahawl@umich.edu

Aortic Valve Disease, Aortic Valve Insufficiency, Aortic Regurgitation

J-Valve TF Early Feasibility Study

Central Nervous System Neoplasm, Infantile Fibrosarcoma, Recurrent Acute Leukemia, Refractory Acute Leukemia, Solid Neoplasm

Larotrectinib (LOXO-101, NSC# 788607) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias

Central Nervous System Neoplasm, Infantile Fibrosarcoma, Recurrent Acute Leukemia, Refractory Acute Leukemia, Solid Neoplasm

Larotrectinib (LOXO-101, NSC# 788607) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias

Neoplasms

Long-Term Follow-Up (LTFU) of Participants Treated With ADP Adoptive Cell Therapies

• 215-825-9260
• patients@adaptimmune.com

Osteosarcoma, Ewing Sarcoma

Metformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse

• +39(051)6366668
• alessandra.longhi@ior.it

Sickle Cell Disease

Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease

• 202-476-5000
• RNickel@childrensnational.org

Hemophilia A

MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study

• 404-785-1637
• robert.sidonio.jr@emory.edu

Advanced 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

Molecular Analysis for Combination Therapy Choice (ComboMATCH)

Head and Neck Neoplasms

Molecular and Genomic Profiling of Head and Neck Tumors to Explore Biomarkers Associated With Prognosis and Response to Therapy

• 718-920-7054
• staugust@montefiore.org

Childhood Obesity, Epigenetics

Molecular Basis of Early Childhood Obesity Programming by Intrauterine Growth Restriction

• 718-904-4105
• mfuloria@montefiore.org

Inflammatory Bowel Disease

Multi-Center African-American Inflammatory Bowel Disease Study (MAAIS)

• 410-614-1982
• ibd@jhu.edu

Pediatric Solid Tumor

Multicenter 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

Epstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant, Stem Cell Transplant Complications

Multicenter, 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

• 650-278-8930
• clinicalstudies@atarabio.com

Epstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant, Stem Cell Transplant Complications

Multicenter, 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

• 650-278-8930
• clinicalstudies@atarabio.com

Primary Immunodeficiency Diseases (PID)

Multicenter, Prospective, Open-label, Randomized, Crossover Study to Evaluate Pharmacokinetics (PK), Safety, and Tolerability of TAK-881 in Primary Immunodeficiency Diseases (PIDD)

• +1-877-825-3327
• medinfoUS@takeda.com

Neoplasms, Arteriovenous Malformation of Central Nervous System, Trigeminal Neuralgia

Multi-Institution Registry of SRS/SBRT Procedures

• jjenkins@therss.org

Hypoplastic Left Heart Syndrome (HLHS)

National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children With Complex Congenital Heart Disease

• mark.timbers@cchmc.org

Mycobacterium Infections

Natural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections

• (301) 443-2163
• samantha.kreuzburg@nih.gov

Recurrent 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

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations

Advanced 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

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Erdafitinib in Patients With Tumors Harboring FGFR1/2/3/4 Alterations

Advanced 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

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-101 (Larotrectinib) in Patients With Tumors Harboring Actionable NTRK Fusions

Hematopoietic 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

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-292 in Patients With Tumors Harboring RET Gene Alterations

Advanced 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

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Ensartinib in Patients With Tumors Harboring ALK or ROS1 Genomic Alterations

Advanced 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

NCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice)- Phase 2 Subprotocol of LY3023414 in Patients With Solid Tumors

Malignant 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

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Tipifarnib in Patients With Tumors Harboring HRAS Genomic Alterations

Advanced 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

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol

Minimal Change Disease (MCD), Membranous Nephropathy, Glomerulosclerosis, Focal Segmental

Nephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network

• 734-615-5021
• NEPTUNE-Study@umich.edu

Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma

Neuroblastoma Biology Studies

Childhood Malignant Neoplasm

Neuropsychological, Social, Emotional, and Behavioral Outcomes in Children With Cancer

Neuroblastoma

NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)

• 7175310003
• BCCEnroll@pennstatehealth.psu.edu

Rheumatic Joint Disease

Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

• (919) 419-5032
• mara.becker@duke.edu

H3 K27M, Glioma

ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study

• 1-919-806-1074
• clinicaltrials@chimerix.com

Pain, Procedural

Optimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure?

• 540 514 4389
• comcnama@montefiore.org

Alveolar 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

Pazopanib 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)

Acute 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

Pediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias

Childhood Hepatocellular Carcinoma, Childhood Malignant Liver Neoplasm, Fibrolamellar Carcinoma, Hepatoblastoma, Hepatocellular Malignant Neoplasm, Not Otherwise Specified

Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

Ewing Sarcoma, Solid Tumors, Rhabdoid Tumor, Rhabdomyosarcoma, Neuroblastoma, Medulloblastoma, Diffuse Intrinsic Pontine Glioma

PHASE 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

Sickle Cell Disease, Pain

Phase 1-2 Trial of Gamunex (intravenous Gammaglobulin) for Sickle Cell Acute Pain

• 718-741-2342
• dmanwani@montefiore.org

Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis

Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis

Pediatric Heart Transplantation, Immunosuppression, Chronic Kidney Diseases, Cardiac Allograft Vasculopathy, Heart Transplant Failure and Rejection, Post-transplant Lymphoproliferative Disorder, Heart Transplant Infection

Phase 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

Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Recurrent Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma

Phase III Randomized Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue

Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Recurrent Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma

Phase III Randomized Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue

Anaplastic Ependymoma, Brain Ependymoma, Cellular Ependymoma, Clear Cell Ependymoma, Ependymoma, Papillary Ependymoma

Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients With Newly Diagnosed Ependymoma Ages 1 to 21 Years

Chronic Kidney Diseases

Phosphate Binder Therapy and Chronic Kidney Disease in Children

• 310-7943144
• jbrook@mednet.ucla.edu

COVID-19 Infection, Hematopoietic and Lymphatic System Neoplasm, Malignant Solid Neoplasm

Prospective Cohort Study to Evaluate Immunologic Response Following COVID-19 Vaccination in Children, Adolescents and Young Adults With Cancer

Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation, Solid Tumors, Blood Cancers, CAR-T, Gene Therapy, Non-malignant Disease

Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

• 763 406 3280
• databaseIRB@nmdp.org

Cmv Congenital, CMV, Congenital Cmv, SNHL, Sensorineural Hearing Loss

Randomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial

Cmv Congenital, CMV, Congenital Cmv, SNHL, Sensorineural Hearing Loss

Randomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial

Acute Lymphoblastic Leukemia, Lymphoblastic Lymphoma

Randomized Study of Intravenous Calaspargase Pegol (SC-PEG Asparaginase) and Intravenous Oncaspar in Children and Adolescents With Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

Acute Ischemic Stroke, Vessel Occlusion, Stroke, Ischemic

REal-World Analyses of Stroke - Thrombus Occlusion REtrieval

• 714.920.3815
• michelle.wetherby@microvention.com

Asthma

Registry of Asthma Patients Initiating DUPIXENT® (RAPID)

Asthma

Registry of Asthma Patients Initiating DUPIXENT® (RAPID)

Adult 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

Renal 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.

Recurrent B Acute Lymphoblastic Leukemia

Risk-Stratified Randomized Phase III Testing of Blinatumomab (NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)

Aortic Stenosis, Severe

Safety and Effectiveness of Balloon-Expandable Bioprosthetic SAPIEN X4 Transcatheter Heart Valve

Sickle Cell Disease

Sickle Cell Disease (SCD) Biochip': Towards a Simple and Reliable Way to Monitor Sickle Cell Disease

• (216) 368-6447
• umut@case.edu

Bone Cyst

Simple Bone Cysts in Kids (SBoCK)

• sbock.study@sickkids.ca

Sarcoma, Solid Tumor, Adult, Solid Tumor, Childhood

Single-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

• 410-955-0432
• mpetryc1@jhmi.edu

Chronic Phase Chronic Myeloid Leukemia, BCR-ABL1 Positive

Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP)

Transplant Rejection, Allograft Rejection, Complications

Study for the Prediction of Active Rejection in Organs Using Donor-derived Cell-free DNA Detection

• 6502499090
• zdemko@natera.com

Heart Transplant Rejection

Surveillance HeartCare® Outcomes Registry

Heart Transplant Rejection

Surveillance HeartCare® Outcomes Registry

Autism Spectrum Disorder

Testing the Feasibility of a Teletherapy Plus Parent Coaching Intervention for Children With Autism

• 6102912179
• roseann.schaaf@jefferson.edu

Adrenal 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

The Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study

Scoliosis Idiopathic

The Tether™ - Vertebral Body Tethering System Post Approval Study

• 1-303-465-8960
• Monica.Barascout@zimvie.com

Adult Kidney Wilms Tumor, Beckwith-Wiedemann Syndrome, Childhood Kidney Wilms Tumor, Diffuse Hyperplastic Perilobar Nephroblastomatosis, Rhabdoid Tumor of the Kidney, Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor, Stage V Kidney Wilms Tumor

Treatment for Patients With Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor

Adult Kidney Wilms Tumor, Beckwith-Wiedemann Syndrome, Childhood Kidney Wilms Tumor, Diffuse Hyperplastic Perilobar Nephroblastomatosis, Rhabdoid Tumor of the Kidney, Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor, Stage V Kidney Wilms Tumor

Treatment for Patients With Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor

PRETEXT I Hepatoblastoma, PRETEXT II Hepatoblastoma, PRETEXT III Hepatoblastoma, PRETEXT IV Hepatoblastoma

Treatment of Children With All Stages of Hepatoblastoma With Temsirolimus (NSC#683864) Added to High Risk Stratum Treatment

Acute Lymphoblastic Leukemia, Pediatric

Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents

Anaplastic Kidney Wilms Tumor, Recurrent Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor

Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)

Diabetes Mellitus, Type 1

TrialNet Pathway to Prevention of T1D

• 1-800-425-8361

Heart Transplant Rejection

Trifecta-Heart cfDNA-MMDX Study: Comparing the DD-cfDNA Test to MMDx Microarray Test and Central HLA Antibody Test

• 1 780 782 9463
• konrad@ualberta.ca

Solid Tumors

Tumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial

• 888-662-6728 (U.S. and Canada)
• Global-Roche-Genentech-Trials@gene.com

Hematopoietic Cell Transplantation Recipient, Leukemia, Solid Tumor

Umbrella Long-Term Follow-Up Protocol

Neurofibromatosis Type 1, Plexiform Neurofibromas

US Selumetinib Registry

• 1-855-752-2356
• clinicaltrials@alexion.com

Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Neuroblastoma

Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients With Non-High-Risk Neuroblastoma

Coronavirus

Viral Infection and Respiratory Illness Universal Study[VIRUS]: COVID-19 Registry and Validation of C2D2 (Critical Care Data Dictionary)

• 5072557196
• Kashyap.Rahul@mayo.edu