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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

Active, Not Recruiting

Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)

NCT06721871

Conditions
Congenital Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, +5 more
Locations
  • Boston Children's Hospital Boston, Massachusetts, United States
  • UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4 Rome, Italy
  • Al Jalila Children's Hospital Dubai, United Arab Emirates
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Active, Not Recruiting

An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)

NCT06254482

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +17 more
Locations
  • Novartis Investigative Site Clayton, Australia
  • Novartis Investigative Site Westmead, Australia
  • Novartis Investigative Site Innsbruck, Austria
  • Novartis Investigative Site Ottawa, Canada
View all 24 locations
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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
View Trial Details

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