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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

Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Study

NCT03461211

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations
  • Macro, Llc Beverly Hills, California, United States
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • Bascom Palmer Eye Institute Miami, Florida, United States
  • Kellogg Eye Center at University of Michigan Ann Arbor, Michigan, United States
View all 13 locations
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Completed

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +11 more
Locations
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
  • University of California, Davis Sacramento, California, United States
  • University of California, San Diego San Diego, California, United States
  • University of Michigan Ann Arbor, Michigan, United States
View Trial Details
Completed

Pegloticase and Methotrexate Co-administered in Participants With Uncontrolled Gout Who Previously Failed Pegloticase Monotherapy

NCT04772313

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +10 more
Locations
  • University of Alabama at Birmingham (UAB) - Center for Education & Research on Therapeutics of Musculoskeletal Disorders Birmingham, Alabama, United States
  • Arizona Arthritis and Rheumatology Associates Flagstaff, Arizona, United States
  • Arizona Arthritis and Rheumatology Associates Glendale, Arizona, United States
  • Arizona Arthritis and Rheumatology Associates Mesa, Arizona, United States
View all 15 locations
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Completed

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

NCT03860935

Conditions
Alzheimer Disease, Amyloid Cardiomyopathy, Amyloid Neuropathies, +25 more
Locations
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • Pacific Heart Institute Santa Monica, California, United States
  • University of Colorado Hospital - Anschutz Medical Campus Aurora, Colorado, United States
  • Yale School of Medicine New Haven, Connecticut, United States
View all 104 locations
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Completed

Study of Pegloticase (KRYSTEXXA®) Plus Methotrexate in Patients With Uncontrolled Gout

NCT03635957

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +9 more
Locations
  • Orthopedic Physicians Alaska Anchorage, Alaska, United States
  • Arizona Arthritis & Rheumatology -West Valley Glendale, Arizona, United States
  • Arizona Arthritis & Rheumatology -East Valley Mesa, Arizona, United States
  • Avail Clinical Research DeLand, Florida, United States
View Trial Details
Completed

Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial

NCT03462511

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +13 more
Locations
  • Feinstein Institute for Medical Research Manhasset, New York, United States
  • Columbia University Irving Medical Center New York, United States
  • Albert Einstein College of Medicine The Bronx, New York, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
View Trial Details

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