Institution Directory

Clinical trials at Children's Hospital of Philadelphia

Sites in Philadelphia, PA

Institution pages show live ClinicalTrials.gov records that list this campus as a study site, whoever sponsors them. They do not rate, rank, or describe the institution.

ClinicalTrials.gov public records Last synced Oct 19, 2026, 12:02 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official matching trials

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Conditions: X-Linked Myotubular Myopathy
Interventions: No Intervention; Other
Lead sponsor: Astellas Gene Therapies; Industry
Eligibility: Up to 17 Years · Male only
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 7
States / cities: Santa Monica, California • Chicago, Illinois • Boston, Massachusetts + 4 more

Conditions: Down Syndrome, Obstructive Sleep Apnea
Interventions: Oxygen; Drug
Lead sponsor: Brigham and Women's Hospital; Other
Eligibility: 5 Years to 18 Years
Enrollment: 230 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 7
States / cities: Los Angeles, California • Ann Arbor, Michigan • Cincinnati, Ohio + 4 more

Conditions: Clostridium Difficile, Clostridium Difficile Infections, Clostridium Difficile Infection Recurrence, Clostridioides Difficile Infection, Clostridioides Difficile Infection Recurrence, CDI, C. Diff Infection, Recurrent Clostridium Difficile Infection, C.Difficile Diarrhea, Diarrhea Infectious
Interventions: VE303, Placebo; Biological
Lead sponsor: Vedanta Biosciences, Inc.; Industry
Eligibility: 12 Years and older
Enrollment: 852 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 84
States / cities: Huntsville, Alabama • Chandler, Arizona • Phoenix, Arizona + 73 more

Conditions: Eosinophilic Esophagitis (EoE)
Interventions: dupilumab; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 6 Months to 6 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 13
States / cities: Phoenix, Arizona • San Francisco, California • Aurora, Colorado + 10 more

Conditions: Friedreich's Ataxia (FA)
Interventions: SGT-212; Drug
Lead sponsor: Solid Biosciences Inc.; Industry
Eligibility: 18 Years to 40 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 3
States / cities: Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania

Conditions: Recurrent Classic Hodgkin Lymphoma, Refractory Classic Hodgkin Lymphoma
Interventions: Biopsy Procedure, Biospecimen Collection, Brentuximab Vedotin, Computed Tomography, Ipilimumab, Nivolumab, Positron Emission Tomography; Procedure · Drug · Biological
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Years and older
Enrollment: 146 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 479
States / cities: Birmingham, Alabama • Anchorage, Alaska • Fairbanks, Alaska + 303 more

Conditions: NGLY1 Deficiency
Interventions: GlcNAc-GlcN, Placebo; Drug · Other
Lead sponsor: Eva Morava-Kozicz; Other
Eligibility: 1 Year to 60 Years
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Seattle, Washington

Conditions: Angelman Syndrome
Interventions: obudanersen, Placebo; Drug
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years to 50 Years
Enrollment: 156 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 18
States / cities: Los Angeles, California • San Diego, California • Aurora, Colorado + 13 more

Conditions: X-linked Retinoschisis
Interventions: ATSN-201; Biological
Lead sponsor: Atsena Therapeutics Inc.; Industry
Eligibility: 5 Years and older
Enrollment: 97 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2033
U.S. locations: 18
States / cities: Phoenix, Arizona • La Jolla, California • Los Angeles, California + 14 more

Conditions: Sensorineural Hearing Loss, Bilateral
Interventions: AAVAnc80-hOTOF via Akouos Delivery Device, AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism; Combination Product
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: Not listed
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 6
States / cities: San Francisco, California • Iowa City, Iowa • Boston, Massachusetts + 3 more

Conditions: T-cell Acute Lymphoblastic Leukemia, Lymphoblastic Lymphoma
Interventions: WU-CART-007; Biological
Lead sponsor: Allotera Therapeutics; Industry
Eligibility: 6 Months and older
Enrollment: 125 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 16
States / cities: Duarte, California • Los Angeles, California • Denver, Colorado + 12 more

Conditions: Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
Interventions: Mitapivat Matched Placebo, Mitapivat; Drug
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 1 Year to 17 Years
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 4
States / cities: Atlanta, Georgia • New York, New York • Philadelphia, Pennsylvania + 1 more

About this Children's Hospital of Philadelphia institution page

What this page lists: ClinicalTrials.gov records that list Children's Hospital of Philadelphia in Philadelphia, PA as a study site, whoever sponsors them, among recruiting, not yet recruiting, and active not recruiting trials.
Site status: A study's overall status can differ from this site's status. Sites marked withdrawn, terminated, or completed are not counted here. Each record shows the status listed for this site when the official record provides one.
Where the data comes from: Records are sourced from the official ClinicalTrials.gov public API. Each result links back to the official study page, which is the source of truth for eligibility, enrollment, and contact information.
What this page does not do: No medical advice, eligibility judgments, ranking of trials, or AI-generated medical summaries. This page does not rank or rate institutions.

Clinical trials at Children's Hospital of Philadelphia

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Live official records

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

Down Syndrome Obstructive Sleep Apnea

VE303 for Prevention of Recurrent Clostridioides Difficile Infection

A Study of Dupilumab in Small Children With an Allergic Condition of the Esophagus (Food Pipe): Eosinophilic Esophagitis

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

Brentuximab Vedotin and Nivolumab With or Without Ipilimumab in Treating Patients With Relapsed or Refractory Hodgkin Lymphoma

A Study of GlcNAc on Tear Production in NGLY1-CDDG

REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome

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

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

A Phase 2 Study of WU-CART-007, an Anti-CD7 Allogeneic CAR-T Cell Therapy in T-Cell Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma (T-RRex)

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

About this Children's Hospital of Philadelphia institution page