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

NCT03994731

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +9 more
Locations
  • University of Alabama Birmingham, Alabama, United States
  • Orthopedic Physicians Alaska Anchorage, Alaska, United States
  • Arizona Arthritis and Rheumatology Research, PLLC - Flagstaff Flagstaff, Arizona, United States
  • Arizona Arthritis and Rheumatology Research, PLLC-West Glendale, Arizona, United States
View all 60 locations
View Trial Details
Completed

Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT®

NCT05904210

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Luskin Orthopaedic Institute for Children Los Angeles, California, United States
  • Valley Children's Hospital Madera, California, United States
  • Children's healthcare of Atlanta Atlanta, Georgia, United States
  • Mayo Clinic Rochester, Minnesota, United States
View Trial Details
Completed

A Study of TEPEZZA® Treatment in Participants With Thyroid Eye Disease

NCT06389578

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations
  • Bascom Palmer Eye Institute - University of Miami Miami, Florida, United States
  • Barnes Jewish Hospital Washington University St Louis, Missouri, United States
  • Neuro-Eye Clinical Trials Bellaire, Texas, United States
View Trial Details
Completed

Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)

NCT03349242

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations
  • Shiley Eye Institute - UCSD La Jolla, California, United States
  • Stanford University, Spencer Center for Vision Research Stanford, California, United States
  • Emory Eye Centre Atlanta, Georgia, United States
  • Massachusetts Eye and Ear Infirmary Boston, Massachusetts, United States
View Trial Details
Completed

A Study to Assess the Efficacy and Safety of CMK389 in Patients With Moderate to Severe Atopic Dermatitis.

NCT04836858

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • Novartis Investigative Site Pardubice, Czech Republic, Czechia
  • Novartis Investigative Site Prague, Czechia
  • Novartis Investigative Site Marseille, France
  • Novartis Investigative Site Nice, France
View all 18 locations
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Completed

An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia

NCT01571596

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • University of California San Francisco San Francisco, California, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Clinical Research Center, Indiana University School of Medicine Indianapolis, Indiana, United States
  • Duke Clinical Research Unit Durham, North Carolina, United States
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