Clinical Trials at Boston Children's Hospital

As of June 2026, 269 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.

Recruiting trial data synced daily from ClinicalTrials.gov. Last sync: June 12, 2026.

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269 clinical trials at Boston Children's Hospital

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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

Apply to this trial

OnTrackCF: Engagement, Feasibility, and Acceptability Study

for Cystic Fibrosis (CF)

3 locations incl. Birmingham

Sponsor: Boston Children's Hospital

Sex: All·Age: 18+

Code: NCT07031323·Recruiting

Apply to this trial

I-DECIDE After Bronchiolitis Hospitalization

for Bronchiolitis Acute

56 locations incl. Birmingham

Sponsor: Seattle Children's Hospital

Sex: All·Age: 0 Months - 24 Months

Code: NCT07243652·Recruiting

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A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

for Angelman Syndrome

3 locations incl. Los Angeles

Sponsor: MavriX Bio, LLC

Sex: All·Age: 4 - 50

Code: NCT07181837·Phase 1, Phase 2·Recruiting

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Balloon Inflation Time for Esophageal Strictures (BITES): A Randomized Multi-Center Study

for Esophageal Atresia With Tracheo-esophageal Fistula

2 locations incl. Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: Up to 18

Code: NCT07100379·Recruiting

Apply to this trial

Study of Oral Fluid Testing Approach

for Cannabis Use

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 18 - 30

Code: NCT06891235·Recruiting

Apply to this trial

Promoting Resilience Among Adolescents and Young Adults With Sickle Cell Disease

for Sickle Cell Disease

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 8 - 25

Code: NCT06555939·Recruiting

Apply to this trial

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

for Duchene Muscular Dystrophy

Drug: Givinostat

7 locations incl. Little Rock

Sponsor: ITF Therapeutics LLC

Sex: All·Age: 6+

Code: NCT07127978·Recruiting

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Breaking Silence Through Story: A Narrative Medicine Intervention for Parents of Children With Urogenital Conditions

for DSD

1 location in Boston

Sponsor: Sarah Schlegel

Sex: All·Age: 18+·Accepts healthy volunteers

Code: NCT06989593·Recruiting

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A Safety and Feasibility Trial Protocol of Metformin in Infants After Perinatal Brain Injury

for Hypoxic-Ischemic Encephalopathy

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 3 Months - 6 Months

Code: NCT06429007·Phase 1·Enrolling By Invitation

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

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Optimal Ventilation for Cardiac Arrest

for Cardiac Arrest (CA)

20 locations incl. Orange

Sponsor: Children's Hospital of Philadelphia

Sex: All·Age: 37 Weeks - 18

Code: NCT07114510·Recruiting

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Clinical Course Of Disease In Participants With FA-CM

for Friedreich Ataxia

8 locations incl. La Jolla

Sponsor: Lexeo Therapeutics

Sex: All·Age: 6+

Code: NCT06865482·Recruiting

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

for Hypothalamic Obesity

8 locations incl. Birmingham

Sponsor: Rhythm Pharmaceuticals, Inc.

Sex: All·Age: 4+

Code: NCT06760546·Phase 3·Recruiting

Apply to this trial

A Feasibility Study to Evaluate Safety and Probable Benefit of the Eclipse XL1 System for Distraction Enterogenesis in Adult and Pediatric Patients With Short Bowel Syndrome

for Short Bowel Syndrome

6 locations incl. Palo Alto

Sponsor: Eclipse Regenesis, Inc.

Sex: All·Age: 3 Months - 65

Code: NCT05535361·Recruiting

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Epilepsy Seizure Detection With Innovative Tripolar EEG (tEEG)

for Epilepsy

2 locations incl. Phoenix

Sponsor: CREmedical

Sex: All·Age: 6+

Code: NCT05944692·Recruiting

Apply to this trial

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

for HSP

1 location in Boston

Sponsor: BlackfinBio Ltd

Sex: All·Age: 12 Months - 60 Months

Code: NCT06948019·Phase 1, Phase 2·Not Yet Recruiting

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

for Neurofibromatosis 1

Drug: Selumetinib

15 locations incl. Birmingham

Sponsor: University of Alabama at Birmingham

Sex: All·Age: 1 - 8

Code: NCT06188741·Phase 2·Recruiting

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Postoperative Pain Control in AIS Using Liposomal Bupivacaine vs. 0.25% Bupivacaine With Epinephrine

for Post Operative Pain

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 10 - 17

Code: NCT06471348·Recruiting

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Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies

for Developmental and Epileptic Encephalopathies

1 location in Boston

Sponsor: Heather Olson

Sex: All·Age: 0 Days - 9 Months

Code: NCT06700811·Phase 1·Recruiting

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

for X-Linked Chronic Granulomatous Disease

8 locations incl. Los Angeles

Sponsor: Ensoma

Sex: Male·Age: 3 Months+

Code: NCT06876363·Phase 1, Phase 2·Recruiting

Apply to this trial

Understanding Vape Marketing Study

for E-cigarette

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 13+·Accepts healthy volunteers

Code: NCT07129265·Enrolling By Invitation

Modeling Blood Management and Hemolytic Risk in Pediatric Heart Surgery

for Heart Surgery

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 0 - 17

Code: NCT06721455·Recruiting

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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 incl. Boston

Sponsor: Steven DuBois, MD

Sex: All·Age: 1 - 30

Code: NCT07027748·Phase 1·Recruiting

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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

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Milrinone for Prevention of Post-ligation Cardiac Syndrome Trial

for Post-ligation Cardiac Syndrome

19 locations incl. Birmingham

Sponsor: NICHD Neonatal Research Network

Sex: All·Age: Up to 3 Months

Code: NCT06679855·Phase 3·Recruiting

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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

for PIK3CA-Related Overgrowth Spectrum (PROS)

19 locations incl. Phoenix

Sponsor: Relay Therapeutics, Inc.

Sex: All·Age: 2+

Code: NCT06789913·Phase 2·Recruiting

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REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

for Angelman Syndrome

15 locations incl. Los Angeles

Sponsor: Ionis Pharmaceuticals, Inc.

Sex: All·Age: 2 - 50

Code: NCT06914609·Phase 3·Recruiting

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A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

for Dravet Syndrome

Drug: zorevunersen

31 locations incl. Phoenix

Sponsor: Stoke Therapeutics, Inc

Sex: All·Age: 2 - 17

Code: NCT06872125·Phase 3·Recruiting

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Registry and Natural History of Epilepsy-Dyskinesia Syndromes

for Epilepsy-Dyskinesia

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 0 - 30

Code: NCT06967727·Recruiting

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Glycemic Regulation as Endometriosis Adjunct Treatment

for Pelvic Pain

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: Female·Age: 15 - 35

Code: NCT06611501·Phase 2·Recruiting

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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

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

for X-Linked Myotubular Myopathy

6 locations incl. Chicago

Sponsor: Astellas Gene Therapies

Sex: Male·Age: Up to 17

Code: NCT06581146·Recruiting

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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

Apply to this trial

Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)

for Cystic Fibrosis

18 locations incl. Birmingham

Sponsor: Boston Children's Hospital

Sex: All·Age: 6+

Code: NCT06683092·Recruiting

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Evaluation of a Supervised Clinical Feeding Intervention

for Peanut Allergies in Children

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 4 Months - 6 Months·Accepts healthy volunteers

Code: NCT06892132·Not Yet Recruiting

GF-NOURISH (Gluten Free Nutrition Optimization Through Ultra-processed Food Reduction and Improved Strategies for Health)

for Celiac Disease in Children

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 2 - 18

Code: NCT06356220·Recruiting

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TransHealthGUIDE: Transforming Health for Gender-Diverse Young Adults

for Transgender Persons

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 18 - 29·Accepts healthy volunteers

Code: NCT06177600·Recruiting

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Precision Medicine in the CICU: Identifying Proteomic Biomarkers

for Congenital Heart Disease

1 location in Boston

Sponsor: Boston Children's Hospital

Sex: All·Age: 2 Days - 5

Code: NCT06642662·Enrolling By Invitation

Proof-of-Concept Testing of the Cardiovascular Health Equity Through Food (CHEF) Intervention in Childhood Cancer Survivors

for Childhood Cancer

2 locations incl. Boston

Sponsor: Dana-Farber Cancer Institute

Sex: All·Age: Up to 18

Code: NCT06609473·Recruiting

Apply to this trial