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 22, 2026, 12:00 PM EDT

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

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Conditions: Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
Interventions: Apitegromab; Drug
Lead sponsor: Scholar Rock, Inc.; Industry
Eligibility: 2 Years and older
Enrollment: 238 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 25
States / cities: Phoenix, Arizona • La Jolla, California • Los Angeles, California + 22 more

Conditions: Lymphoblastic Lymphoma, Acute Lymphoblastic Leukemia ALL
Interventions: 4A10; Drug
Lead sponsor: Allterum Therapeutics, Inc; Industry
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 7
States / cities: Aurora, Colorado • Indianapolis, Indiana • New York, New York + 4 more

Conditions: Hematopoietic and Lymphatic System Neoplasm, Malignant Solid Neoplasm
Interventions: Best Practice, Biospecimen Collection, Electronic Health Record Review, Survey Administration, Triptorelin Pamoate; Other · Procedure · Drug
Lead sponsor: Children's Oncology Group; Network
Eligibility: Up to 39 Years · Female only
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 201
States / cities: Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 143 more

Conditions: Warm Autoimmune Hemolytic Anemia
Interventions: M281, Placebo; Drug
Lead sponsor: Janssen Research & Development, LLC; Industry
Eligibility: 18 Years and older
Enrollment: 118 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2028
U.S. locations: 29
States / cities: Fountain Valley, California • Los Angeles, California • Riverside, California + 24 more

Conditions: X-linked Hypophosphatemia, Hypophosphatemic Rickets
Interventions: No intervention; Other
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: Not listed
Enrollment: 782 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2032
U.S. locations: 24
States / cities: Birmingham, Alabama • Los Angeles, California • San Diego, California + 18 more

Conditions: Heart Failure, Left Ventricular Systolic Dysfunction
Interventions: Vericiguat tablet, Vericiguat suspension, Placebo tablet, Placebo suspension; Drug
Lead sponsor: Merck Sharp & Dohme LLC; Industry
Eligibility: 29 Days to 17 Years
Enrollment: 342 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2032
U.S. locations: 20
States / cities: Los Angeles, California • Palo Alto, California • San Bernardino, California + 17 more

Conditions: Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue, Clear Cell Sarcoma of Soft Tissue, Ewing Sarcoma, Hepatoblastoma, Hepatocellular Carcinoma, Osteosarcoma, Recurrent Adrenal Cortical Carcinoma, Recurrent Alveolar Soft Part Sarcoma, Recurrent Clear Cell Sarcoma of Soft Tissue, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Kidney Wilms Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Osteosarcoma, Recurrent Primary Malignant Central Nervous System Neoplasm, Recurrent Renal Cell Carcinoma, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Medullary Carcinoma, Refractory Adrenal Cortical Carcinoma, Refractory Alveolar Soft Part Sarcoma, Refractory Clear Cell Sarcoma of Soft Tissue, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Primary Malignant Central Nervous System Neoplasm, Refractory Renal Cell Carcinoma, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Medullary Carcinoma, Refractory Wilms Tumor, Renal Cell Carcinoma, Rhabdomyosarcoma, Soft Tissue Sarcoma, Solid Neoplasm, Thyroid Gland Medullary Carcinoma, Wilms Tumor
Interventions: Cabozantinib, Cabozantinib S-malate, Pharmacological Study; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 2 Years to 30 Years
Enrollment: 109 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2027
U.S. locations: 142
States / cities: Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 104 more

Conditions: Insufficient Sleep, Hypertension
Interventions: Intervention; Behavioral
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 13 Years to 18 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Busulfan Pharmacokinetic Analysis, Busulfan Dosing, Hematopoietic Stem Cell Transplant (HSCT), Busulfan, Glutathione
Interventions: Busulfan (BU); Drug
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: Up to 21 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Interventions: Mitapivat, Mitapivat-matching placebo; Drug
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 1 Year to 17 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2030
U.S. locations: 11
States / cities: Palo Alto, California • Aurora, Colorado • Atlanta, Georgia + 8 more

Conditions: Congenital Hyperinsulinism
Interventions: HM15136; Drug
Lead sponsor: Hanmi Pharmaceutical Company Limited; Industry
Eligibility: 2 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Cardiac Arrest (CA), Pediatric Cardiac Arrest (Simulated), Cardiopulmonary Resuscitation (CPR)
Interventions: AR-CPR; Device
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years and older
Enrollment: 252 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2028
U.S. locations: 7
States / cities: St. Petersburg, Florida • Baltimore, Maryland • New York, New York + 4 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

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

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.

Triptorelin for the Prevention of Ovarian Damage in Adolescents and Young Adults With Cancer

Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia

X-linked Hypophosphatemia Disease Monitoring Program

Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)

Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors

Sleep Promotion and Pediatric Hypertension

26DT052; Scaling Busulfan Dose to Body Surface Area in Children

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)

Augmented Reality-Cardiopulmonary Resuscitation Support for Pediatric Resuscitation

About this Children's Hospital of Philadelphia institution page