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

4.1on Google· View on Google Maps

As of September 2026, 247 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: September 1, 2026.

New trial at Boston Children s Hospital? We'll email you when one opens.

Recruiting clinical trials at Boston Children s Hospital (247)

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

Apply to this trial

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

Apply to this trial

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

for Sickle Cell Disease

44 locations incl. Birmingham

Sponsor: Novo Nordisk A/S

Sex: All·Age: 12+

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

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

A Study to Evaluate the Long-term Safety and Tolerability of KarXT and KarX-EC for the Treatment of Schizophrenia and Autism-Related Irritability in Adolescents, Respectively

for Schizophrenia

49 locations incl. Dothan

Sponsor: Karuna Therapeutics, Inc., a Bristol Myers Squibb company

Sex: All·Age: 5 - 17

Code: NCT07424404·Phase 3·Recruiting·Listed Feb 2026

Apply to this trial

A Study to Evaluate the Efficacy and Safety of KarXT for the Treatment of Schizophrenia in Adolescents (EMERGENT TEEN)

for Schizophrenia

21 locations incl. Anaheim

Sponsor: Bristol-Myers Squibb

Sex: All·Age: 13 - 17

Code: NCT07288567·Phase 3·Recruiting·Listed Dec 2025

Apply to this trial

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)

15 locations incl. Little Rock

Sponsor: argenx

Sex: All·Age: 5 - 17

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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)

22 locations incl. Phoenix

Sponsor: Relay Therapeutics, Inc.

Sex: All·Age: 2+

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

Apply to this trial

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

Apply to this trial

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

for Angelman Syndrome

18 locations incl. Los Angeles

Sponsor: Ionis Pharmaceuticals, Inc.

Sex: All·Age: 2 - 50

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

Apply to this trial

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

Apply to this trial

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

18 locations incl. Orange

Sponsor: Cabaletta Bio

Sex: All·Age: 18 - 65

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment

for Parenteral Nutrition Associated Liver Disease (PNALD)

10 locations incl. Fountain Valley

Sponsor: Fresenius Kabi

Sex: All·Age: 1 Day - 17

Code: NCT06274788·Recruiting·Listed Feb 2024

Apply to this trial

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

Apply to this trial

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

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

Apply to this trial

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

Apply to this trial

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

Apply to this trial

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

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

Apply to this trial

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 247 recruiting trials at this site. Browse all with filters →

Get new-trial alerts for Boston Children s Hospital

We'll email you whenever a new clinical trial starts recruiting at this research site in Boston, MA. Unsubscribe anytime.

More research sites in Boston

Massachusetts General Hospital (693 trials)
Brigham and Women's Hospital (352 trials)
Dana-Farber Cancer Institute (346 trials)
Beth Israel Deaconess Medical Center (263 trials)
Boston Children's Hospital (258 trials)
Dana Farber Cancer Institute (218 trials)
Tufts Medical Center (113 trials)
Boston Medical Center (95 trials)
Massachusetts General Hospital Cancer Center (51 trials)
VA Boston Healthcare System Jamaica Plain Campus, Jamaica Plain, MA (30 trials)

Is this your research site?

This page is built from public ClinicalTrials.gov records. If you work here, we can correct it, send you the patients who apply, and build you a free website from your real studies — verify it with your work email, or read what we do for research sites.

Explore more

Paid clinical trials in Boston, MA · Massachusetts clinical trials