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Clinical trial condition

Rare Diseases

Explore clinical trials studying Rare Diseases. Study availability and eligibility vary by location and protocol.

466 public trials 83 recruiting studies
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Rare Diseases trial results

Recruiting

UW Undiagnosed Genetic Diseases Program

NCT04586075

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genetic Disease, +6 more
Locations
  • University of Wisconsin School of Medicine and Public Health Madison, Wisconsin, United States
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Recruiting

Intact Cord Resuscitation in CDH

NCT04429750

Conditions
Congenital Abnormalities, Congenital Diaphragmatic Hernia, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +9 more
Locations
  • Chu Amiens Picardie Amiens, France
  • Hop Jeanne de Flandre Chu Lille Lille, France
  • Chu de Nantes - Nantes, France
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Recruiting

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

NCT06826612

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +19 more
Locations
  • Beth Israel Deaconess Medical Center Boston, Massachusetts, United States
  • University of Cincinnati/Cincinnati Children's Hospital Cincinnati, Ohio, United States
  • Ohio State University Columbus, Ohio, United States
  • University of Pennsylvania Philadelphia, Pennsylvania, United States
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Recruiting

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) Boston, Massachusetts, United States
  • Weill Cornell Medicine; Division of Pediatric Neurology New York, United States
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Recruiting

BEhavioral and Adherence Model for Improving Quality, Health Outcomes and Cost-Effectiveness of healthcaRe

NCT06856902

Conditions
Behavior, Cardiovascular Diseases, Disease Attributes, +10 more
Locations
  • UDUS - Heinrich-Heine-University Duesseldorf Düsseldorf, Germany
  • FISM - Italian Multiple Sclerosis Foundation Genova, Italy
  • WDO - World Duchenne Organization Veenendaal, Netherlands
  • AHUS - Akershus University Hospital Lørenskog, Akershus, Norway
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Recruiting

International Rare Brain Tumor Registry

NCT05697874

Conditions
Astroblastoma, BCOR ITD Sarcoma, CNS Sarcoma, +12 more
Locations
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California San Francisco San Francisco, California, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
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