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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.

12,633 public trials 2,161 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Upadacitinib for Refractory Behcet's Syndrome

NCT07080346

Conditions
Behcet Syndrome, Cardiovascular Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +15 more
Locations
  • Department of Rheumatology and Immunology, Peking University People's Hospital Beijing, Beijing Municipality, China
View Trial Details
Completed

A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

NCT04935879

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +11 more
Locations
  • University of South Alabama Children's and Women's Hospital Mobile, Alabama, United States
  • University of South Alabama Mitchell Cancer Institute Mobile, Alabama, United States
  • University of South Alabama Strada Patient Care Center Mobile, Alabama, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
View all 58 locations
View Trial Details
Completed

Repeatability and Reproducibility of Multiparametric MRI

NCT03743272

Conditions
Autoimmune Diseases, Autoimmune Hepatitis, Bile Duct Diseases, +33 more
Locations
  • Leiden University Medical Centre Leiden, Netherlands
  • Addenbrookes Hospital Cambridge, Cambridgeshire, United Kingdom
  • University of Oxford Centre for Clinical Magnetic Resonance Research (OCMR) Oxford, Oxfordshire, United Kingdom
  • Southampton General Hospital Southampton, United Kingdom
View Trial Details
Completed

Evaluation of PKU Sphere in Maternal PKU

NCT04224142

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +14 more
Locations
  • Royal Victoria Hospital Belfast, United Kingdom
  • University Hospital of Wales Cardiff, United Kingdom
  • University College London Hospitals NHS Foundation Trust London, United Kingdom
View Trial Details
Completed

A Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Repeat Doses of Inhaled ETD001 in People With Cystic Fibrosis

NCT06478706

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Hospices Civils de Lyon Lyon, France
  • CHU de Montpellier Montpellier, France
  • Hôpital Cochin Paris, France
  • Hôpitaux de Toulouse Toulouse, France
View all 21 locations
View Trial Details
Completed

Performances and Safety of MuCopilot, a Digital Tool for the Unsupervised Objective Assessment of Cystic Fibrosis

NCT06147778

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Centre Hospitalier Lyon Sud (Adultes) Lyon, France
  • APHM - Hôpital Nord de Marseille Marseille, France
  • Centre de compétence Maladies pulmonaires rares Hôpital Pasteur CHU de Nice Nice, France
  • American Memorial Hospital Reims, France
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