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Clinical trial condition

Rare Diseases

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

473 public trials 86 recruiting studies
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Rare Diseases trial results

Active, not recruiting

Simulated and Synthetic Health Data: Improving Clinical Research on Rare Diseases. A Real-World Data Simulation of Autosomal Dominant Polycystic Kidney Disease (ADPKD) Trials. A Retrospective, Observational Study

NCT07016282

Conditions
Abnormalities, Multiple, Autosomal Dominant Polycystic Kidney Disease (ADPKD), Ciliopathies, +16 more
Locations
  • Clinical Research Centre for Rare Diseases Aldo e Cele Daccò Ranica, BG, Italy
  • Department of Global Public Health (GPH), Karolinska Institutet Stockholm, Sweden
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Completed

Interleukin-1 Receptor Antagonist (IL-1RA) (ANAKINRA) IN SEVERE SYSTEMIC-ONSET JUVENILE IDIOPATHIC ARTHRITIS

NCT00339157

Conditions
Arthritis, Arthritis, Juvenile, Autoimmune Diseases, +11 more
Locations
  • Bordeaux CHU Bordeaux, France
  • Hopital Lyon Edouard Herriot Lyon, France
  • Nancy Hopital d'Enfants Nancy, France
  • Pediatric Immuno-Hematology and Rheumatology Unit, Necker-Enfants Malades Hospital, 149 rue de Sevres Paris, France
View Trial Details
Completed

A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

NCT05358717

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +17 more
Locations
  • University of California, San Diego La Jolla, California, United States
  • Vanderbilt University Medical Center Nashville, Tennessee, United States
  • The University of Texas Health Science Center at Houston; McGovern Medical School Houston, Texas, United States
  • University of Washington Department of Neurology Seattle, Washington, United States
View all 28 locations
View Trial Details
Completed

Genetic Studies in the Amish and Mennonites

NCT00359580

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genetic Disease, +4 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
  • Clinic for Special Children Strasburg, Pennsylvania, United States
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