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Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,656 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH

NCT04842032

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • Peking Union Medical College Hospital Beijing, China
  • Guangzhou Women and Children Medical Center Guangzhou, China
  • Shanghai 6th Hospital Shanghai, China
  • Shanghai Xinhua Hopsital Shanghai, China
View Trial Details
Completed

Vitamin D3 For CGD Patients With BCGosis/Itis

NCT03984890

Conditions
BCG, Chronic Disease, Chronic-granulomatous Disease, +14 more
Locations
  • Children's Hospital of Fudan University Shanghai, Shanghai Municipality, China
  • Children's Hospital of Fudan University Shanghai, Shanghai Municipality, China
View Trial Details
Completed

Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)

NCT03703310

Conditions
Abnormalities, Multiple, Basal Cell Nevus Syndrome, Bone Cysts, +18 more
Locations
  • Mayo Clinical Cancer Center Phoenix, Arizona, United States
  • Dermatology Center of Newport Newport Beach, California, United States
  • Stanford University, Department of Dermatology Redwood City, California, United States
  • University of California, San Francisco San Francisco, California, United States
View all 49 locations
View Trial Details
Completed

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004704

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Investigational Site Number 840001 New York, United States
  • Investigational Site Number 056001 Leuven, Belgium
  • Investigational Site Number 076001 Porto Alegre, Brazil
  • Investigational Site Number 250002 Bron, France
View Trial Details
Completed

Goal Attainment Scaling (GAS) Sub-Study

NCT06484426

Conditions
Abnormalities, Multiple, Alzheimer Disease, Alzheimer's Disease in Down Syndrome, +16 more
Locations
  • University of California, San Diego San Diego, California, United States
  • Advocate Health Park Ridge, Illinois, United States
  • Kennedy Krieger Institute Baltimore, Maryland, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
View Trial Details

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