Clinical Trials at Children's Hospital of Philadelphia

As of July 2026, 273 paid clinical trials are recruiting at Children's Hospital of Philadelphia, located at 3550 MARKET ST, 5TH FLOOR, PHILADELPHIA, PA 19104-3329, phone (215) 590-4862 in Philadelphia, Pennsylvania. Compensation typically covers time, travel, and study visits — most studies also offer study-related medical care at no cost to participants.

Recruiting trial data synced daily from ClinicalTrials.gov. Last sync: July 17, 2026.

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274 clinical trials at Children's Hospital of Philadelphia

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A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

for Low-grade Glioma

Sites in Philadelphia +11 more locations

Sponsor: Day One Biopharmaceuticals, Inc.

Sex: All·Age: 6 Months - 25

Code: NCT04775485·Phase 2·Recruiting·Listed Mar 2021

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Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)

for B-cell Acute Lymphoblastic Leukemia

Sites in Philadelphia +12 more locations

Sponsor: Cellectis S.A.

Sex: All·Age: 15 - 50

Code: NCT04150497·Phase 1, Phase 2·Recruiting·Listed Nov 2019

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First-in-human Study of a New Treatment (4A10) for Patients With Relapsed or Hard-to-treat Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma, Focused on Safety and How the Drug Behaves in the Body and Early Signs of Effect.

for Lymphoblastic Lymphoma

Sites in Philadelphia +3 more locations

Sponsor: Allterum Therapeutics, Inc

Sex: All·Age: 18+

Code: NCT07586618·Phase 1·Recruiting·Listed May 2026

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A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients

for High-Risk Neuroblastoma

Sites in Philadelphia +4 more locations

Sponsor: Renaissance Pharma Ltd.

Sex: All·Age: 18 Months - 18

Code: NCT07549321·Phase 2, Phase 3·Recruiting·Listed Apr 2026

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Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

for Achondroplasia

Drug: Vosoritide

Sites in Philadelphia +7 more locations

Sponsor: BioMarin Pharmaceutical

Sex: All·Age: 2 - 17

Code: NCT07441876·Phase 2, Phase 3·Recruiting·Listed Mar 2026

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A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

for Congenital Adrenal Hyperplasia

Sites in Philadelphia +7 more locations

Sponsor: Crinetics Pharmaceuticals Inc.

Sex: All·Age: 1 - 17

Code: NCT07159841·Phase 2, Phase 3·Recruiting·Listed Sep 2025

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AERA Pediatrics Registry

for Dysfunction of Eustachian Tube

Sites in Philadelphia +8 more locations

Sponsor: Integra LifeSciences Corporation

Sex: All·Age: Up to 18

Code: NCT07071298·Recruiting·Listed Jul 2025

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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

for Glycogen Storage Disease Type Ia

Sites in Philadelphia +8 more locations

Sponsor: Ultragenyx Pharmaceutical Inc

Sex: All·Age: 2+

Code: NCT06636383·Recruiting·Listed Oct 2024

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Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

for Familial Partial Lipodystrophy

Sites in Philadelphia +6 more locations

Sponsor: Chiesi Farmaceutici S.p.A.

Sex: All·Age: 13+

Code: NCT06679270·Phase 3·Recruiting·Listed Nov 2024

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A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition

for X-Linked Myotubular Myopathy

Sites in Philadelphia, Pittsburgh +4 more locations

Sponsor: Astellas Gene Therapies

Sex: Male·Age: Up to 17

Code: NCT06581146·Recruiting·Listed Sep 2024

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An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

for Vanishing White Matter Disease

Sites in Philadelphia +2 more locations

Sponsor: Calico Life Sciences LLC

Sex: All·Age: 6 Months+

Code: NCT05757141·Phase 1, Phase 2·Recruiting·Listed Mar 2023

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Two Dose Levels of Privigen in Pediatric CIDP

for Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Sites in Philadelphia +2 more locations

Sponsor: CSL Behring

Sex: All·Age: 2 - 17

Code: NCT03684018·Phase 4·Recruiting·Listed Sep 2018

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A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

for Danon Disease

Sites in Philadelphia +2 more locations

Sponsor: Rocket Pharmaceuticals Inc.

Sex: Male·Age: 8+

Code: NCT06092034·Phase 2·Recruiting·Listed Oct 2023

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To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

for Fibrodysplasia Ossificans Progressiva (FOP)

Sites in Philadelphia +1 more location

Sponsor: Incyte Corporation

Sex: All·Age: 2 - 99

Code: NCT05090891·Phase 2·Recruiting·Listed Oct 2021

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A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

for Friedreich Ataxia

Sites in Philadelphia +3 more locations

Sponsor: Biogen

Sex: All·Age: 16+

Code: NCT06623890·Recruiting·Listed Oct 2024

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ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

for X-linked Retinoschisis

Sites in Philadelphia +3 more locations

Sponsor: Atsena Therapeutics Inc.

Sex: All·Age: 6+

Code: NCT05878860·Phase 3·Recruiting·Listed May 2023

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ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

for Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Sites in Philadelphia +7 more locations

Sponsor: Ionis Pharmaceuticals, Inc.

Sex: Male·Age: 2 - 65

Code: NCT06430385·Phase 1, Phase 2·Recruiting·Listed May 2024

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A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

for Hypothalamic Obesity

Sites in Philadelphia +7 more locations

Sponsor: Rhythm Pharmaceuticals, Inc.

Sex: All·Age: 4+

Code: NCT06760546·Phase 3·Recruiting·Listed Jan 2025

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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

for Sensorineural Hearing Loss, Bilateral

Sites in Philadelphia +5 more locations

Sponsor: Akouos, Inc.

Sex: All·Age: All ages

Code: NCT05821959·Phase 1, Phase 2·Recruiting·Listed Apr 2023

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A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL)

for Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia

Sites in Philadelphia +2 more locations

Sponsor: Autolus Limited

Sex: All·Age: 0 - 18

Code: NCT06173518·Phase 1·Recruiting·Listed Dec 2023

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Otoferlin Gene-mediated Hearing Loss Natural History Study

for Sensorineural Hearing Loss, Bilateral

Sites in Philadelphia +3 more locations

Sponsor: Akouos, Inc.

Sex: All·Age: Up to 44

Code: NCT05572073·Recruiting·Listed Oct 2022

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A Retrospective Study to Characterize Participants With Propionic Acidemia

for Propionic Acidemia

Sites in Philadelphia +3 more locations

Sponsor: ModernaTX, Inc.

Sex: All·Age: 2+

Code: NCT05769621·Recruiting·Listed Mar 2023

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A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

for Mucopolysaccharidosis II

Sites in Philadelphia +7 more locations

Sponsor: Denali Therapeutics Inc.

Sex: All·Age: 2 - 25

Code: NCT05371613·Phase 2, Phase 3·Recruiting·Listed May 2022

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A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

for Cerebral Adrenoleukodystrophy (CALD)

Sites in Philadelphia +2 more locations

Sponsor: Genetix Biotherapeutics Inc.

Sex: Male·Age: All ages

Code: NCT06224413·Recruiting·Listed Jan 2024

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Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

for Alagille Syndrome

Sites in Philadelphia, Pittsburgh +6 more locations

Sponsor: Mirum Pharmaceuticals, Inc.

Sex: All·Age: All ages

Code: NCT06193928·Recruiting·Listed Jan 2024

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A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

for Beta-Thalassemia

Sites in Philadelphia +5 more locations

Sponsor: Genetix Biotherapeutics Inc.

Sex: All·Age: All ages

Code: NCT06271512·Recruiting·Listed Feb 2024

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Pediatric Post-Approval Registry

for Obstructive Sleep Apnea

Sites in Philadelphia, Sewickley +5 more locations

Sponsor: Inspire Medical Systems, Inc.

Sex: All·Age: 18 - 21

Code: NCT04457154·Recruiting·Listed Jul 2020

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MSC EVs in Dystrophic Epidermolysis Bullosa

for Dystrophic Epidermolysis Bullosa

Sites in Philadelphia +2 more locations

Sponsor: Aegle Therapeutics

Sex: All·Age: 6 Months+

Code: NCT04173650·Phase 1, Phase 2·Recruiting·Listed Nov 2019

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Early Signals of the Transition From Immune Quiescence to Activation in the Liver Allograft Microenvironment and in the Circulation

for Liver Transplant

Sites in Philadelphia +9 more locations

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Sex: All·Age: 3 - 13

Code: NCT07549503·Phase 2·Recruiting·Listed Apr 2026

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A Pragmatic Clinical Trial of the WE BEAT Well-Being Education Program in Adolescent Congenital Heart Disease: WE BEAT CHD Study

for Patient-Reported Outcome Measures (PROMs)

Sites in Philadelphia, Pittsburgh +12 more locations

Sponsor: Carelon Research

Sex: All·Age: 12 - 17

Code: NCT07525843·Recruiting·Listed Apr 2026

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Nutrition Intervention for Pancreatic Cancer

for Pancreatic Ductal Adenocarcinoma (PDAC)

2 locations in Philadelphia

Sponsor: Children's Hospital of Philadelphia

Sex: All·Age: 18+

Code: NCT07554560·Recruiting·Listed Apr 2026

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CART123 Cells With or Without Ruxolitinib in Relapsed/Refractory Acute Myeloid Leukemia

for Acute Myeloid Leukemia (AML)

Drug: Ruxolitinib

1 location in Philadelphia

Sponsor: Stephan Grupp MD PhD

Sex: All·Age: 0 - 29

Code: NCT07464951·Phase 1·Recruiting·Listed Mar 2026

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Bowel Continence Across the Lifespan in People With Spina Bifida

for Spina Bifida

Sites in Philadelphia, Pittsburgh +22 more locations

Sponsor: David Chu

Sex: All·Age: 5+

Code: NCT07390318·Recruiting·Listed Feb 2026

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Urinary Titin Biomarker in DMD

for Duchenne Muscular Dystrophy (DMD)

1 location in Philadelphia

Sponsor: Children's Hospital of Philadelphia

Sex: Male·Age: 2 - 10·Accepts healthy volunteers

Code: NCT07332013·Recruiting·Listed Jan 2026

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Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease

for Sickle Cell Disease

Drug: Ruxolitinib

Sites in Philadelphia +3 more locations

Sponsor: Arkansas Children's Hospital Research Institute

Sex: All·Age: 12 - 45

Code: NCT07252050·Phase 1, Phase 2·Recruiting·Listed Nov 2025

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Endotype DIrected Treatment for OSA in Down Syndrome

for Obstructive Sleep Apnea (OSA)

Sites in Philadelphia +4 more locations

Sponsor: University of Arizona

Sex: All·Age: 6+

Code: NCT07280468·Phase 4·Recruiting·Listed Dec 2025

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SPACE for Youth With Chronic Pain

for Chronic Pain

Sites in Philadelphia +1 more location

Sponsor: Children's Hospital of Philadelphia

Sex: All·Age: 10 - 17

Code: NCT07425691·Recruiting·Listed Feb 2026

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Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors

for Childhood Extracranial Germ Cell Tumor

Sites in Philadelphia, Allentown +385 more locations

Sponsor: Children's Oncology Group

Sex: All·Age: All ages

Code: NCT03067181·Phase 3·Recruiting·Listed Mar 2017

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A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab

for Lugano Classification Limited Stage Hodgkin Lymphoma AJCC v8

Drug: Nivolumab

Sites in Philadelphia, Allentown +380 more locations

Sponsor: National Cancer Institute (NCI)

Sex: All·Age: 5 - 60

Code: NCT05675410·Phase 3·Recruiting·Listed Jan 2023

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Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy

for B Acute Lymphoblastic Leukemia

Sites in Philadelphia, Allentown +186 more locations

Sponsor: Children's Oncology Group

Sex: All·Age: 365 Days - 25

Code: NCT03959085·Phase 3·Recruiting·Listed May 2019

Apply to this trial