Clinical Trials at Boston Children's Hospital

As of July 2026, 214 paid clinical trials are recruiting at Boston Children's Hospital, located at 11 HEREFORD ST, APT 4R, BOSTON, MA 02115-1602, phone (617) 602-9730 in Boston, Massachusetts. Compensation typically covers time, travel, and study visits — most studies also offer study-related medical care at no cost to participants.

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Recruiting trial data synced daily from ClinicalTrials.gov. Last sync: July 19, 2026.

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Healthy volunteers only

220 clinical trials at Boston Children's Hospital

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AERA Pediatrics Registry

for Dysfunction of Eustachian Tube

Sites in Boston +8 more locations

Sponsor: Integra LifeSciences Corporation

Sex: All·Age: Up to 18

Code: NCT07071298·Recruiting·Listed Jul 2025

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A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

for Duchene Muscular Dystrophy

Drug: Givinostat

Sites in Boston, Worcester +7 more locations

Sponsor: ITF Therapeutics LLC

Sex: All·Age: 6+

Code: NCT07127978·Recruiting·Listed Aug 2025

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Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators

for X-Linked Myotubular Myopathy

Sites in Boston +2 more locations

Sponsor: Astellas Gene Therapies

Sex: Male·Age: Up to 36 Months

Code: NCT07052929·Phase 1, Phase 2·Recruiting·Listed Jul 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

Sites in Boston +5 more locations

Sponsor: Astellas Gene Therapies

Sex: Male·Age: Up to 17

Code: NCT06581146·Recruiting·Listed Sep 2024

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Clinical Course Of Disease In Participants With FA-CM

for Friedreich Ataxia

Sites in Boston +7 more locations

Sponsor: Lexeo Therapeutics

Sex: All·Age: 6+

Code: NCT06865482·Recruiting·Listed Mar 2025

Apply to this trial

Starlight Cardiovascular Lifeline Ductus Arteriosus Stent IDE Study

for Congenital Heart Disease

Sites in Boston +7 more locations

Sponsor: Starlight Cardiovascular Inc

Sex: All·Age: 1 Minute - 6 Months

Code: NCT07114718·Recruiting·Listed Aug 2025

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A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

for Myotonic Dystrophy Type 1 (DM1)

Sites in Boston +7 more locations

Sponsor: Vertex Pharmaceuticals Incorporated

Sex: All·Age: 18 - 64

Code: NCT06185764·Phase 1, Phase 2·Recruiting·Listed Dec 2023

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KiteLock 4% EDTA Lock Solution for the Prevention of Occlusions in Children With Intestinal Failure

for Pediatric Intestinal Failure

Sites in Boston +7 more locations

Sponsor: SterileCare Inc.

Sex: All·Age: 4 Weeks - 18

Code: NCT05879835·Recruiting·Listed May 2023

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Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis

for Cystic Fibrosis

Sites in Boston +2 more locations

Sponsor: SpliSense Ltd.

Sex: All·Age: 18+

Code: NCT06429176·Phase 2·Recruiting·Listed May 2024

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A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

for Danon Disease

Sites in Boston +2 more locations

Sponsor: Rocket Pharmaceuticals Inc.

Sex: Male·Age: 8+

Code: NCT06092034·Phase 2·Recruiting·Listed Oct 2023

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Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

for X-Linked Chronic Granulomatous Disease

Sites in Boston +7 more locations

Sponsor: Ensoma

Sex: Male·Age: 3 Months+

Code: NCT06876363·Phase 1, Phase 2·Recruiting·Listed Mar 2025

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ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

for Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Sites in Boston +7 more locations

Sponsor: Ionis Pharmaceuticals, Inc.

Sex: Male·Age: 2 - 65

Code: NCT06430385·Phase 1, Phase 2·Recruiting·Listed May 2024

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A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

for Muscular Atrophy, Spinal

Sites in Boston +2 more locations

Sponsor: Biogen

Sex: Female·Age: 18 - 54

Code: NCT05789758·Recruiting·Listed Mar 2023

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A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

for Hypothalamic Obesity

Sites in Boston +7 more locations

Sponsor: Rhythm Pharmaceuticals, Inc.

Sex: All·Age: 4+

Code: NCT06760546·Phase 3·Recruiting·Listed Jan 2025

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PKP2-ACM Natural History Study

for Cardiomyopathies

Sites in Boston +1 more location

Sponsor: Rocket Pharmaceuticals Inc.

Sex: All·Age: 12+

Code: NCT06644742·Recruiting·Listed Oct 2024

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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

for Sensorineural Hearing Loss, Bilateral

Sites in Boston +5 more locations

Sponsor: Akouos, Inc.

Sex: All·Age: All ages

Code: NCT05821959·Phase 1, Phase 2·Recruiting·Listed Apr 2023

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JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency

for SHANK3 Haploinsufficiency

Sites in Boston +2 more locations

Sponsor: Jaguar Gene Therapy, LLC

Sex: All·Age: 2 - 9

Code: NCT06662188·Phase 1, Phase 2·Recruiting·Listed Oct 2024

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PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency

for Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency

Sites in Boston +4 more locations

Sponsor: Inozyme Pharma

Sex: All·Age: All ages

Code: NCT06302439·Recruiting·Listed Mar 2024

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Epilepsy Seizure Detection With Innovative Tripolar EEG (tEEG)

for Epilepsy

Sites in Boston +1 more location

Sponsor: CREmedical

Sex: All·Age: 6+

Code: NCT05944692·Recruiting·Listed Jul 2023

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HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

for Angelman Syndrome

Sites in Boston +4 more locations

Sponsor: Ionis Pharmaceuticals, Inc.

Sex: All·Age: 0 - 50

Code: NCT05127226·Phase 1, Phase 2·Recruiting·Listed Nov 2021

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Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

for Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency

Sites in Boston +4 more locations

Sponsor: Medicure

Sex: All·Age: All ages

Code: NCT04706013·Phase 3·Recruiting·Listed Jan 2021

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A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

for Cerebral Adrenoleukodystrophy (CALD)

Sites in Boston +2 more locations

Sponsor: Genetix Biotherapeutics Inc.

Sex: Male·Age: All ages

Code: NCT06224413·Recruiting·Listed Jan 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)

Sites in Boston +13 more locations

Sponsor: Carelon Research

Sex: All·Age: 12 - 17

Code: NCT07525843·Recruiting·Listed Apr 2026

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Use of Indocyanine Green (ICG) for the Diagnosis of Biliary Atresia

for Biliary Atresia

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 0 Days - 3 Months

Code: NCT07513038·Phase 1, Phase 2·Recruiting·Listed Apr 2026

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Bowel Continence Across the Lifespan in People With Spina Bifida

for Spina Bifida

Sites in Boston +23 more locations

Sponsor: David Chu

Sex: All·Age: 5+

Code: NCT07390318·Recruiting·Listed Feb 2026

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Propofol-Only Versus Dexmedetomidine-Propofol in Children Undergoing Magnetic Resonance Imaging

for MRI Sedation

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 1 - 12

Code: NCT07369128·Phase 4·Recruiting·Listed Jan 2026

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A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy

for Epileptic Encephalopathy

Sites in Boston +5 more locations

Sponsor: Institut de Recherches Internationales Servier

Sex: All·Age: 2 - 12

Code: NCT07227857·Phase 1, Phase 2·Recruiting·Listed Nov 2025

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Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

for Ataxia Telangiectasia

1 location in Boston

Sponsor: Timothy Yu

Sex: All·Age: 0 - 17

Code: NCT07215416·Phase 1, Phase 2·Recruiting·Listed Oct 2025

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Assessment of Microvascular Circulation in the Pediatric Cardiac Surgery Patient

for Tetrology of Fallot

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 1 Day - 17

Code: NCT07184476·Recruiting·Listed Sep 2025

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I-DECIDE After Bronchiolitis Hospitalization

for Bronchiolitis Acute

Sites in Boston, Springfield +54 more locations

Sponsor: Seattle Children's Hospital

Sex: All·Age: 0 Months - 24 Months

Code: NCT07243652·Recruiting·Listed Nov 2025

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Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

for Cystic Fibrosis

Sites in Boston +34 more locations

Sponsor: Sonya Heltshe

Sex: All·Age: Up to 10

Code: NCT04509050·Recruiting·Listed Aug 2020

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Milrinone for Prevention of Post-ligation Cardiac Syndrome Trial

for Post-ligation Cardiac Syndrome

Sites in Boston +18 more locations

Sponsor: NICHD Neonatal Research Network

Sex: All·Age: Up to 3 Months

Code: NCT06679855·Phase 3·Recruiting·Listed Nov 2024

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Distal Radius Interventions for Fracture Treatment

for Fracture Distal Radius

Sites in Boston +23 more locations

Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

Sex: All·Age: 4 - 10

Code: NCT05131685·Recruiting·Listed Nov 2021

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Rett Syndrome Registry

for Rett Syndrome

Sites in Boston +18 more locations

Sponsor: International Rett Syndrome Foundation

Sex: All·Age: 0 - 99

Code: NCT05432349·Recruiting·Listed Jun 2022

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Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma

for Neuroblastoma

Drug: Irinotecan

2 locations in Boston

Sponsor: Steven DuBois, MD

Sex: All·Age: 1 - 30

Code: NCT07027748·Phase 1·Recruiting·Listed Jun 2025

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Clinical and Basic Investigations Into Congenital Disorders of Glycosylation

for Congenital Disorders of Glycosylation

Sites in Boston +11 more locations

Sponsor: Icahn School of Medicine at Mount Sinai

Sex: All·Age: All ages

Code: NCT04199000·Recruiting·Listed Dec 2019

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Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis

for Hemophilia A

Sites in Boston +9 more locations

Sponsor: Wayne State University

Sex: All·Age: 6 - 19

Code: NCT05022459·Recruiting·Listed Aug 2021

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Striae Distensae Treatment Using Deep Skin Abrasion

for Striae Distensae

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 18 - 70

Code: NCT07087405·Recruiting·Listed Jul 2025

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Advanced SPinal Innovations With Robotics and Enabling Technology Registry

for Spine Deformity

Sites in Boston +9 more locations

Sponsor: Boston Children's Hospital

Sex: All·Age: 0 - 21·Accepts healthy volunteers

Code: NCT05071144·Recruiting·Listed Oct 2021

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The Use of High Bile-binding Foods to Reduce Upper Gastrointestinal Bile Acid Concentrations (Aim 1)

for Feeding Difficulties

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 5 - 21

Code: NCT07303738·Phase 4·Recruiting·Listed Dec 2025

Apply to this trial