Skip to main content
OpenTrials

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
Refine in advanced search Browse all conditions

Rare Diseases trial results

Enrolling by invitation

Idiopathic Diseases of Man

NCT01440218

Conditions
Disease Attributes, Idiopathic Disease, Pathologic Processes, +3 more
Locations
  • Scripps Translational Science Institute La Jolla, California, United States
View Trial Details
Active, not recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475

Conditions
Beta-Galactosidase-1 (GLB1) Deficiency, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Children's Hospital at St. Peter's University Hospital New Brunswick, New Jersey, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
View Trial Details
Active, not recruiting

Nivolumab and Ipilimumab in Treating Patients With Rare Tumors

NCT02834013

Conditions
Acinar Cell Carcinoma, Adenocarcinoma, Adenocarcinoma of Lung, +207 more
Locations
  • University of Alabama at Birmingham Cancer Center Birmingham, Alabama, United States
  • University of South Alabama Mitchell Cancer Institute Mobile, Alabama, United States
  • Anchorage Associates in Radiation Medicine Anchorage, Alaska, United States
  • Anchorage Radiation Therapy Center Anchorage, Alaska, United States
View all 1007 locations
View Trial Details
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
View Trial Details
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
View Trial Details

Related conditions