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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.

12,537 public trials 2,139 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT06623890

Conditions
Brain Diseases, Central Nervous System Diseases, Cerebellar Diseases, +11 more
Locations
  • UCLA Neurology Los Angeles, California, United States
  • University of Colorado Aurora, Colorado, United States
  • University of Florida Gainesville, Florida, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
View all 14 locations
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Recruiting

A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders

NCT06904027

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Peking University First Hospital Beijing, Beijing Municipality, China
  • Guangzhou Women and Children's Medical Center Guangzhou, Guangdong, China
  • Tongji Hosipital Wuhan, Hubei, China
  • Xinhua Hospital, Shanghai Jiao Tong University School of Medicine Shanghai, Shanghai Municipality, China
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Recruiting

HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease

NCT02855476

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +14 more
Locations
  • Cenexel Englewood, Colorado, United States
  • Georgetown University Washington D.C., District of Columbia, United States
  • John Hopkins University Baltimore, Maryland, United States
  • Wake Forest University Winston-Salem, North Carolina, United States
View all 40 locations
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Recruiting

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

NCT04883905

Conditions
Acute Hepatic Porphyria, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Coproporphyria, Hereditary, +10 more
Locations
  • Clinical Trial Site Birmingham, Alabama, United States
  • Clinical Trial Site Gilbert, Arizona, United States
  • Clinical Trial Site San Francisco, California, United States
  • Clinical Trial Site Baltimore, Maryland, United States
View all 30 locations
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Recruiting

Early PKD Observational Cohort Study

NCT02936791

Conditions
Abnormalities, Multiple, Ciliopathies, Congenital Abnormalities, +12 more
Locations
  • University of Chicago Chicago, Illinois, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • Children's Mercy Hospital Kansas City, Missouri, United States
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Recruiting

Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

NCT06876363

Conditions
Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, +12 more
Locations
  • University of California, Los Angeles Los Angeles, California, United States
  • University of California, San Francisco San Francisco, California, United States
  • Johns Hopkins All Children's Hospital St. Petersburg, Florida, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
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Recruiting

Lentiviral FIX Gene Therapy

NCT03961243

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Shenzhen Geno-immune Medical Institute Shenzhen, Guangdong, China
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