Skip to main content
OpenTrials

Clinical trial condition

Genetic Diseases, Inborn

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

12,584 public trials 2,158 recruiting studies
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Active, not recruiting

Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY

NCT05485831

Conditions
Brain Diseases, Central Nervous System Diseases, Congenital Abnormalities, +25 more
Locations
  • Policlinico Sant'Orsola di Bologna Bologna, Italy
  • NPI AOU Mater Domini Università Magna Graecia Catanzaro, Italy
  • Università degli studi "G. D'annunzio" Chieti Chieti, Italy
  • Azienda Ospedaliera Universitaria Meyer Neurologia Pediatrica Florence, Italy
View all 18 locations
View Trial Details
Active, not recruiting

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • University of California Los Angeles, California, United States
  • University of Colorado Aurora, Colorado, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University Hospitals Cleveland Medical Center Cleveland, Ohio, United States
View all 16 locations
View Trial Details
Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

NCT03861273

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • UCSF IDS Pharmacy San Francisco, California, United States
  • University of California, San Francisco - Clinical Research Center San Francisco, California, United States
  • University of California, San Francisco - Outpatient Hematology Clinic San Francisco, California, United States
  • The Regents of the University of California, San Francisco campus San Francisco, California, United States
View all 67 locations
View Trial Details
Active, not recruiting

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • The Children's Hospital Colorado Aurora, Colorado, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Icahn School of Medicine New York, United States
  • University Hospital Cleveland Medical Center/Case Western Reserve University Cleveland, Ohio, United States
View Trial Details
Active, not recruiting

NF-1, Nutraceutical Intervention

NCT05363267

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +13 more
Locations
  • Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
View Trial Details
Active, not recruiting

A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD

NCT05281328

Conditions
Abnormalities, Multiple, Ciliopathies, Congenital Abnormalities, +13 more
Locations
  • Mayo Clinic Rochester, Minnesota, United States
  • Mount Sinai Hospital New York, United States
  • The New York Presbyterian Hospital New York, United States
  • Hospital of the University of Pennsylvania Philadelphia, Pennsylvania, United States
View Trial Details

Related conditions