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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.

12,619 public trials 2,162 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Kaiser Permanente Los Angeles, California, United States
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • SphinCS Höchheim, Germany
View Trial Details
Completed

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

NCT05819775

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Research Solutions of Arizona Litchfield Park, Arizona, United States
  • Medical Research of Arizona Scottsdale, Arizona, United States
  • Donald S. Levy M.D. Orange, California, United States
  • Raffi Tachdjian MD, Inc. Santa Monica, California, United States
View all 13 locations
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Completed

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

NCT04729751

Conditions
Abnormalities, Multiple, Alagille Syndrome, Bile Duct Diseases, +14 more
Locations
  • Children Hospital LA Los Angeles, California, United States
  • University of California - San Francisco San Francisco, California, United States
  • Medstar Georgetown University Hospital Washington D.C., District of Columbia, United States
  • Ochsner Hospital for Children New Orleans, Louisiana, United States
View all 14 locations
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Completed

A Study to Evaluate Upadacitinib in Adolescents and Adults With Moderate to Severe Atopic Dermatitis (Measure Up 2)

NCT03607422

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Total Skin and Beauty Derm Ctr /ID# 205129 Birmingham, Alabama, United States
  • Medical Dermatology Specialist /ID# 205516 Phoenix, Arizona, United States
  • Arizona Research Center, Inc. /ID# 205795 Phoenix, Arizona, United States
  • The Dermatology Clinic of Arkansas /ID# 218749 Bryant, Arkansas, United States
View all 196 locations
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Completed

Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks

NCT04739059

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Clinical Research Center of Alabama Birmingham, Alabama, United States
  • Research Solutions of Arizona Litchfield Park, Arizona, United States
  • Medical Research of Arizona Scottsdale, Arizona, United States
  • Little Rock Allergy & Asthma Clinic Little Rock, Arkansas, United States
View all 44 locations
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