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Rare Diseases clinical trials in Boston, MA
9 studies for Rare Diseases are looking for participants at sites in and around Boston (within about 30 miles). Open a study to see who can join and how to contact the study team.
Recruiting for a study here? Feature it at the top of this page — $99 for 30 days →
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
RecruitingObservational studyHealthy volunteers welcome
- Massachusetts General Hospital, Boston
- Boston Children s Hospital, Boston
- Brigham and Women's Hospital, Boston
- and 1 more
RAPA-501 Therapy for ALS
RecruitingPhase 2/3
- Massachusetts General Hospital, Boston
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
RecruitingObservational study
- Boston Children's Hospital, Boston
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
RecruitingPhase 1/2
- Beth Israel Deaconess Medical Center, Boston
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
RecruitingPhase 1/2
- Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND), Boston
Gene Discovery Core, The Manton Center
RecruitingObservational study
- Boston Children's Hospital, Boston
Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome
RecruitingObservational study
- Boston Children's Hospital, Boston
The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis
RecruitingPhase 2
- Massachusetts General Hospital, Boston
Epilepsy Learning Healthcare System (ELHS)
RecruitingObservational study
- Partners - Massachusetts General Hospital Epilepsy Service (MGH)/ Partners - Brigham and Women's (BWH), Boston
- Brigham & Women's Hospital, Boston
About Rare Diseases
A rare disease is one that affects fewer than 200,000 people in the United States. There are nearly 7,000, affecting more than 25 million Americans.
More about Rare Diseases on MedlinePlus
Rare Diseases trials in other cities
- Chicago, IL8 studies
- Washington, DC8 studies
- Philadelphia, PA7 studies
- Los Angeles, CA5 studies
- Cincinnati, OH5 studies
- Columbus, OH4 studies
- New York City, NY3 studies
- Durham, NC3 studies
- Seattle, WA3 studies
- Atlanta, GA3 studies
- Houston, TX3 studies
- St. Louis, MO3 studies
Other studies in Boston
- Breast Cancer111 studies
- Non-Small Cell Lung Cancer90 studies
- Cancer (all types)86 studies
- Colorectal Cancer63 studies
- Metastatic Cancer63 studies
- Lung Cancer62 studies
- Ovarian Cancer61 studies
- Prostate Cancer59 studies
- Depression52 studies
- Endometrial Cancer50 studies
- Heart Failure44 studies
- Acute Myeloid Leukemia44 studies
From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.