Boston Children s Hospital

Clinical research site in Boston, Massachusetts · 11 HEREFORD ST, APT 4R, BOSTON, MA 02115-1602 · (617) 602-9730 · Website

Part of Boston Children's Hospital

As of July 2026, 246 paid clinical trials are recruiting at Boston Children s Hospital in Boston, Massachusetts. Active studies at this site cover conditions such as Congenital Heart Disease, Cystic Fibrosis and Sickle Cell Disease. 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 19, 2026.

Recruiting clinical trials at Boston Children s Hospital (246)

Phase 2 Study to Assess the Safety and Efficacy of ANG003

for Exocrine Pancreatic Insufficiency (EPI)

26 locations incl. Long Beach

Sponsor: Anagram Therapeutics, Inc.

Sex: All·Age: 12+

Code: NCT07450547·Phase 2·Recruiting·Listed Mar 2026

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A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)

for Chronic Kidney Disease

30 locations incl. Birmingham

Sponsor: Boehringer Ingelheim

Sex: All·Age: 2 - 17

Code: NCT07107945·Phase 3·Recruiting·Listed Aug 2025

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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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Safety Study of Viaskin® Peanut Patch in Peanut-Allergic Children 1 Through 3 Years of Age (COMFORT Toddlers)

for Allergy

56 locations incl. Birmingham

Sponsor: DBV Technologies

Sex: All·Age: 1 - 3

Code: NCT07003919·Phase 3·Recruiting·Listed Jun 2025

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RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

for Idiopathic Inflammatory Myopathy

31 locations incl. Orange

Sponsor: Cabaletta Bio

Sex: All·Age: 6 - 75

Code: NCT06154252·Phase 2, Phase 3·Recruiting·Listed Dec 2023

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A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

for Sickle Cell Disease

45 locations incl. Birmingham

Sponsor: Novo Nordisk A/S

Sex: All·Age: 12+

Code: NCT06612268·Phase 3·Recruiting·Listed Sep 2024

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A Study Investigating Oral Ozanimod (RPC1063) in Pediatric Participants With Moderate to Severe Active Ulcerative Colitis

for Colitis, Ulcerative

18 locations incl. Loma Linda

Sponsor: Bristol-Myers Squibb

Sex: All·Age: 2 - 17

Code: NCT05076175·Phase 2, Phase 3·Recruiting·Listed Oct 2021

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A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants

for Bronchopulmonary Dysplasia

24 locations incl. Little Rock

Sponsor: OHB Neonatology Ltd.

Sex: All·Age: 0 Hours - 24 Hours

Code: NCT03253263·Phase 2·Recruiting·Listed Aug 2017

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

for Complex Congenital Heart Defect

19 locations incl. Little Rock

Sponsor: Edwards Lifesciences

Sex: All·Age: All ages

Code: NCT02744677·Recruiting·Listed Apr 2016

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Evaluation of SPN-812 (Viloxazine Extended-release Capsule) in Preschool-age Children With ADHD

for Attention-Deficit/Hyperactivity Disorder

47 locations incl. Saraland

Sponsor: Supernus Pharmaceuticals, Inc.

Sex: All·Age: 48 Months - 69 Months

Code: NCT04781140·Phase 4·Recruiting·Listed Mar 2021

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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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Child and Adolescent Registry for Participants With Narcolepsy

for Narcolepsy

25 locations incl. Phoenix

Sponsor: Jazz Pharmaceuticals

Sex: All·Age: Up to 17

Code: NCT04899947·Recruiting·Listed May 2021

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A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy

for Spinal Muscular Atrophy (SMA)

17 locations incl. Little Rock

Sponsor: argenx

Sex: All·Age: 5 - 17

Code: NCT07287982·Phase 2·Recruiting·Listed Dec 2025

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A Long-term Follow-up Study in Patients Who Received BEAM-101

for Hemoglobinopathy

17 locations incl. Birmingham

Sponsor: Beam Therapeutics Inc.

Sex: All·Age: 14 - 37

Code: NCT07373639·Recruiting·Listed Jan 2026

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Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental Disorder

for GRIN-related Neurodevelopmental Disorder

17 locations incl. Los Angeles

Sponsor: GRIN Therapeutics, Inc.

Sex: All·Age: 1 Month - 18

Code: NCT07224581·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

21 locations incl. Phoenix

Sponsor: RAPT Therapeutics, Inc.

Sex: All·Age: 12 - 55

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

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Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

for Fragile X Syndrome

15 locations incl. Phoenix

Sponsor: Mirum Pharmaceuticals, Inc.

Sex: Male·Age: 13 - 45

Code: NCT07209462·Phase 2·Recruiting·Listed Oct 2025

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A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)

for PIK3CA-Related Overgrowth Spectrum (PROS)

20 locations incl. Phoenix

Sponsor: Relay Therapeutics, Inc.

Sex: All·Age: 2+

Code: NCT06789913·Phase 2·Recruiting·Listed Jan 2025

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4D-710 in Adult Patients With Cystic Fibrosis

for Cystic Fibrosis Lung

18 locations incl. Birmingham

Sponsor: 4D Molecular Therapeutics

Sex: All·Age: 18+

Code: NCT05248230·Phase 2·Recruiting·Listed Feb 2022

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Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects With Moderately to Severely Active Crohn's Disease

for Crohn's Disease

12 locations incl. Oakland

Sponsor: AbbVie

Sex: All·Age: 2 - 17

Code: NCT06332534·Phase 3·Recruiting·Listed Mar 2024

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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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REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

for Angelman Syndrome

17 locations incl. Los Angeles

Sponsor: Ionis Pharmaceuticals, Inc.

Sex: All·Age: 2 - 50

Code: NCT06914609·Phase 3·Recruiting·Listed Apr 2025

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A Study to Evaluate Etrasimod Treatment in Adolescents With Ulcerative Colitis

for Ulcerative Colitis

20 locations incl. Little Rock

Sponsor: Pfizer

Sex: All·Age: 12 - 17

Code: NCT05287126·Phase 2·Recruiting·Listed Mar 2022

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A Master Protocol (AMAZ): A Study of Mirikizumab (LY3074828) in Pediatric Participants With Ulcerative Colitis or Crohn's Disease (SHINE-ON)

for Ulcerative Colitis

11 locations incl. San Francisco

Sponsor: Eli Lilly and Company

Sex: All·Age: 2 - 19

Code: NCT04844606·Phase 3·Recruiting·Listed Apr 2021

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Patiromer for Treatment of Hyperkalaemia in Children Under 12 Years of Age

for Hyperkalemia

11 locations incl. Aurora

Sponsor: Vifor Pharma, Inc.

Sex: All·Age: 0 - 11

Code: NCT05766839·Phase 2·Recruiting·Listed Mar 2023

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A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

for Dravet Syndrome

11 locations incl. San Francisco

Sponsor: Encoded Therapeutics

Sex: All·Age: 6 Months - 17

Code: NCT05419492·Phase 1, Phase 2·Recruiting·Listed Jun 2022

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A Study of Mirikizumab (LY3074828) in Pediatric Participants With Crohn's Disease

for Crohn's Disease

15 locations incl. Los Angeles

Sponsor: Eli Lilly and Company

Sex: All·Age: 2 - 17

Code: NCT05509777·Phase 3·Recruiting·Listed Aug 2022

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Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis

for Cystic Fibrosis

12 locations incl. Tucson

Sponsor: Arcturus Therapeutics, Inc.

Sex: All·Age: 18+

Code: NCT06747858·Phase 2·Recruiting·Listed Dec 2024

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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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Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

for Spinal Muscular Atrophy

15 locations incl. Phoenix

Sponsor: Alcyone Therapeutics, Inc

Sex: All·Age: 3+

Code: NCT05866419·Recruiting·Listed May 2023

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RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus

for Systemic Lupus Erythematosus

19 locations incl. Orange

Sponsor: Cabaletta Bio

Sex: All·Age: 18 - 65

Code: NCT06121297·Phase 1, Phase 2·Recruiting·Listed Nov 2023

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A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

for Phenylketonuria (PKU)

12 locations incl. Aurora

Sponsor: BioMarin Pharmaceutical

Sex: All·Age: All ages

Code: NCT05813678·Recruiting·Listed Apr 2023

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A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

for Phenylketonuria (PKU)

11 locations incl. Aurora

Sponsor: BioMarin Pharmaceutical

Sex: All·Age: All ages

Code: NCT06305234·Recruiting·Listed Mar 2024

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A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)

for Muscular Atrophy, Spinal

10 locations incl. Orange

Sponsor: Biogen

Sex: All·Age: 3+

Code: NCT06555419·Phase 1·Recruiting·Listed Aug 2024

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52 Week Study + 24-Month Long-Term Extension of Safety, PK, & Efficacy of XYOSTED® for Testosterone Replacement in Male Adolescents With Hypogonadism

for Hypogonadism, Male

20 locations incl. Los Angeles

Sponsor: Halozyme Therapeutics

Sex: Male·Age: 12 - 17

Code: NCT06689085·Phase 3·Recruiting·Listed Nov 2024

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A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

for Cystic Fibrosis

13 locations incl. Birmingham

Sponsor: ReCode Therapeutics

Sex: All·Age: 18 - 60·Accepts healthy volunteers

Code: NCT06237335·Phase 2·Recruiting·Listed Feb 2024

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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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A Multicenter Multinational Observational Study of Children With Hypochondroplasia

for Hypochondroplasia

16 locations incl. Phoenix

Sponsor: BioMarin Pharmaceutical

Sex: All·Age: Up to 15

Code: NCT06212947·Recruiting·Listed Jan 2024

Apply to this trial

Showing the 40 most relevant of 246 recruiting trials at this site. Browse all with filters →

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Paid clinical trials in Boston, MA · Massachusetts clinical trials