Upmc Children'S Hospital of Pittsburgh

Clinical research site in Pittsburgh, Pennsylvania · 3705 5TH AVE, GROUND FLOOR, PITTSBURGH, PA 15213 · (412) 692-5352 · Website

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As of September 2026, 71 paid clinical trials are recruiting at Upmc Children'S Hospital of Pittsburgh in Pittsburgh, Pennsylvania. Active studies at this site cover conditions such as Fabry Disease, Cerebral Palsy and Congenital Bone Marrow Failure Syndromes. 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: September 1, 2026.

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Recruiting clinical trials at Upmc Children'S Hospital of Pittsburgh (71)

BEATRIX: A Study to Learn About a Group B Streptococcus Vaccine in Healthy Pregnant Women and Their Babies

for Healthy

118 locations incl. Birmingham

Sponsor: Pfizer

Sex: All·Age: 1 Day - 49·Accepts healthy volunteers

Code: NCT07160244·Phase 3·Recruiting·Listed Sep 2025

Apply to this trial

A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young Adults With Cancer

for Neoplasms

25 locations incl. Birmingham

Sponsor: Eli Lilly and Company

Sex: All·Age: 1 - 39

Code: NCT05999994·Phase 2·Recruiting·Listed Aug 2023

Apply to this trial

Pompe Disease Registry Protocol

for Glycogen Storage Disease Type II

74 locations incl. Birmingham

Sponsor: Genzyme, a Sanofi Company

Sex: All·Age: All ages

Code: NCT00231400·Recruiting·Listed Oct 2005

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Fabry Disease Registry & Pregnancy Sub-registry

for Fabry Disease

74 locations incl. Birmingham

Sponsor: Genzyme, a Sanofi Company

Sex: All·Age: All ages

Code: NCT00196742·Recruiting·Listed Sep 2005

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COMPASSION S3 - Evaluation of the SAPIEN 3 Transcatheter Heart Valve in Patients With Pulmonary Valve Dysfunction

for Complex Congenital Heart Defect

21 locations incl. Birmingham

Sponsor: Edwards Lifesciences

Sex: All·Age: All ages

Code: NCT02744677·Recruiting·Listed Apr 2016

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International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

for Gaucher Disease

70 locations incl. Phoenix

Sponsor: Genzyme, a Sanofi Company

Sex: All·Age: All ages

Code: NCT00358943·Recruiting·Listed Aug 2006

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Fontan Udenafil Exercise Longitudinal Assessment Trial - 2

for Single Ventricle Heart Disease

37 locations incl. Phoenix

Sponsor: Mezzion Pharma Co. Ltd

Sex: All·Age: 12 - 18

Code: NCT05918211·Phase 3·Recruiting·Listed Jun 2023

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EPIC-Peds: A Study to Learn About the Study Medicine Called PF-07321332 (Nirmatrelvir)/Ritonavir in Patients Under 18 Years of Age With COVID-19 That Are Not Hospitalized But Are at Risk for Severe Disease

for COVID-19

24 locations incl. Los Angeles

Sponsor: Pfizer

Sex: All·Age: 0 - 17

Code: NCT05261139·Phase 3·Recruiting·Listed Mar 2022

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A Gaucher Disease Gene Therapy Trial With FLT201

for Gaucher Disease Type 1

16 locations incl. Los Angeles

Sponsor: Spur Therapeutics

Sex: All·Age: 18+

Code: NCT07223944·Phase 3·Recruiting·Listed Nov 2025

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Phase 2b Study of RPT904 as Monotherapy in Participants With IgE-Mediated Food Allergy

for Ig-E Mediated Food Allergy

22 locations incl. Phoenix

Sponsor: RAPT Therapeutics, Inc.

Sex: All·Age: 12 - 55

Code: NCT07220811·Phase 2·Recruiting·Listed Oct 2025

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A Platform Trial for Pediatric Participants With Obesity or Overweight (LY900040)

for Obesity

51 locations incl. Tucson

Sponsor: Eli Lilly and Company

Sex: All·Age: 6 - 17

Code: NCT06672549·Phase 3·Recruiting·Listed Nov 2024

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A Study to Learn More About How Risankizumab Works in Young Participants With Ulcerative Colitis

for Ulcerative Colitis

11 locations incl. Phoenix

Sponsor: AbbVie

Sex: All·Age: 2 - 17

Code: NCT07071519·Phase 3·Recruiting·Listed Jul 2025

Apply to this trial

SAFety and Efficacy of Human Anti-thymocyte ImmunoGlobUlin SAB-142 ARresting Progression of Type 1 Diabetes

for Type 1 Diabetes

22 locations incl. San Francisco

Sponsor: SAb Biotherapeutics, Inc.

Sex: All·Age: 5 - 40

Code: NCT07187531·Phase 2·Recruiting·Listed Sep 2025

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A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

for Chronic Graft-versus-host-disease

11 locations incl. Duarte

Sponsor: Incyte Corporation

Sex: All·Age: 2 - 17

Code: NCT07124078·Phase 2·Recruiting·Listed Aug 2025

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

for Heart Failure

20 locations incl. Los Angeles

Sponsor: Merck Sharp & Dohme LLC

Sex: All·Age: 29 Days - 17

Code: NCT05714085·Phase 2, Phase 3·Recruiting·Listed Feb 2023

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Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

for Primary Mitochondrial Disease

20 locations incl. Orange

Sponsor: Pharming Technologies B.V.

Sex: All·Age: 18+

Code: NCT05650229·Phase 2·Recruiting·Listed Dec 2022

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Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

for Lymphatic Malformations

18 locations incl. Oakland

Sponsor: Novartis Pharmaceuticals

Sex: All·Age: 0 - 100

Code: NCT05948943·Phase 2, Phase 3·Recruiting·Listed Jul 2023

Apply to this trial

A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Crohn's Disease (CD)

for Crohn's Disease (CD)

15 locations incl. Phoenix

Sponsor: Takeda

Sex: All·Age: 2 - 17

Code: NCT04779320·Phase 3·Recruiting·Listed Mar 2021

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A Global Prospective Observational Registry of Patients With Pompe Disease

for Pompe Disease

15 locations incl. Little Rock

Sponsor: Amicus Therapeutics

Sex: All·Age: All ages

Code: NCT06121011·Recruiting·Listed Nov 2023

Apply to this trial

Minima Stent System Post- Approval Study (PAS)

for Pulmonary Artery Stenosis

15 locations incl. Los Angeles

Sponsor: Renata Medical

Sex: All·Age: All ages

Code: NCT06828770·Recruiting·Listed Feb 2025

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Pupillometry in Identifying Risk of Postoperative Opioid-induced Respiratory Depression in Children Undergoing Tonsillectomy

for Opioid-Induced Respiratory Depression

2 locations incl. San Francisco

Sponsor: NeurOptics Inc

Sex: All·Age: 3 - 17·Accepts healthy volunteers

Code: NCT07573150·Recruiting·Listed May 2026

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A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

for Phenylketonuria

3 locations incl. Indianapolis

Sponsor: Agios Pharmaceuticals, Inc.

Sex: All·Age: 18 - 69

Code: NCT07241234·Phase 1·Recruiting·Listed Nov 2025

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

for X-Linked Myotubular Myopathy

7 locations incl. Santa Monica

Sponsor: Astellas Gene Therapies

Sex: Male·Age: Up to 17

Code: NCT06581146·Recruiting·Listed Sep 2024

Apply to this trial

Study to Evaluate the Pharmacokinetics (PK), Safety and Tolerability up to 6 Years of Intravenous (i.v.) Secukinumab in Pediatric Participants With Juvenile Psoriatic Arthritis (JPsA).

for Juvenile Psoriatic Arthritis

11 locations incl. Loma Linda

Sponsor: Novartis Pharmaceuticals

Sex: All·Age: 2 - 17

Code: NCT06751238·Phase 1·Recruiting·Listed Dec 2024

Apply to this trial

EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)

for PIK3CA-related Overgrowth Spectrum (PROS)

8 locations incl. St Louis

Sponsor: Novartis Pharmaceuticals

Sex: All·Age: 2 - 100

Code: NCT06997588·Phase 2·Recruiting·Listed May 2025

Apply to this trial

A Study of Patients With Fabry Disease (US Specific)

for Fabry Disease

8 locations incl. Birmingham

Sponsor: Amicus Therapeutics

Sex: All·Age: 18+

Code: NCT06906367·Recruiting·Listed Apr 2025

Apply to this trial

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

for Fabry Disease

6 locations incl. Atlanta

Sponsor: Amicus Therapeutics

Sex: All·Age: 2 - 11

Code: NCT06904261·Phase 3·Recruiting·Listed Apr 2025

Apply to this trial

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

for Fabry Disease

8 locations incl. Birmingham

Sponsor: UniQure Biopharma B.V.

Sex: Male·Age: 18 - 50

Code: NCT06270316·Phase 1, Phase 2·Recruiting·Listed Feb 2024

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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)

14 locations incl. Stanford

Sponsor: Carelon Research

Sex: All·Age: 12 - 17

Code: NCT07525843·Recruiting·Listed Apr 2026

Apply to this trial

Spinal Cord Stimulation for Children With Cerebral Palsy

for Cerebral Palsy

1 location in Pittsburgh

Sponsor: Martin Piazza

Sex: All·Age: 5 - 17·Accepts healthy volunteers

Code: NCT07516067·Recruiting·Listed Apr 2026

Apply to this trial

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

for Common Variable Immunodeficiency (CVID)

1 location in Pittsburgh

Sponsor: Paul Szabolcs

Sex: All·Age: 5 - 40

Code: NCT07284641·Phase 2·Recruiting·Listed Dec 2025

Apply to this trial

Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy

for LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)

14 locations incl. Los Angeles

Sponsor: Nationwide Children's Hospital

Sex: All·Age: Up to 5

Code: NCT06503367·Recruiting·Listed Jul 2024

Apply to this trial

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

for Hematologic Malignancies

113 locations incl. Birmingham

Sponsor: Center for International Blood and Marrow Transplant Research

Sex: All·Age: All ages

Code: NCT01351545·Recruiting·Listed May 2011

Apply to this trial

Clinical and Basic Investigations Into Congenital Disorders of Glycosylation

for Congenital Disorders of Glycosylation

12 locations incl. San Diego

Sponsor: Icahn School of Medicine at Mount Sinai

Sex: All·Age: All ages

Code: NCT04199000·Recruiting·Listed Dec 2019

Apply to this trial

A-LiNK: Improving Outcomes in Autoimmune Liver Disease

for Autoimmune Hepatitis

8 locations incl. Palo Alto

Sponsor: Children's Hospital Medical Center, Cincinnati

Sex: All·Age: All ages

Code: NCT05750498·Recruiting·Listed Mar 2023

Apply to this trial

Clinical and Laboratory Study of Methylmalonic Acidemia

for Organic Acidemia

2 locations incl. Bethesda

Sponsor: National Human Genome Research Institute (NHGRI)

Sex: All·Age: 1 Month - 115·Accepts healthy volunteers

Code: NCT00078078·Recruiting·Listed Feb 2004

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Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia

for Metabolic Disease

2 locations incl. Bethesda

Sponsor: National Human Genome Research Institute (NHGRI)

Sex: All·Age: 1 Month - 100·Accepts healthy volunteers

Code: NCT02890342·Recruiting·Listed Sep 2016

Apply to this trial

Mapping Disease Pathways for Biliary Atresia

for Biliary Atresia

1 location in Pittsburgh

Sponsor: University of Pittsburgh

Sex: All·Age: All ages

Code: NCT03273049·Recruiting·Listed Sep 2017

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Molecular Basis of Pediatric Liver Cancer

for Childhood Liver Cancer

1 location in Pittsburgh

Sponsor: University of Pittsburgh

Sex: All·Age: 0 - 99

Code: NCT03959800·Recruiting·Listed May 2019

Apply to this trial

Childhood Cancer Survivor Study

for Cancer

27 locations incl. Birmingham

Sponsor: St. Jude Children's Research Hospital

Sex: All·Age: All ages·Accepts healthy volunteers

Code: NCT01120353·Recruiting·Listed May 2010

Apply to this trial

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