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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

Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®

NCT04334057

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Kurume University Hospital, Pediatrics Fukuoka, Japan
  • Gifu University Hospital_The Third Dept. of Internal Medicine Gifu, Japan
  • Gifu University Hospital Gifu, Japan
  • Saitama Medical University Hospital, Pediatrics Iruma-gun, Saitama, Japan
View all 20 locations
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Completed

Study of REGN4461, a Leptin Receptor Agonist Antibody, in Patients With Generalized Lipodystrophy

NCT04159415

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Generalized Lipodystrophy, Genetic Diseases, Inborn, +10 more
Locations
  • Regeneron Research Site Bethesda, Maryland, United States
  • Regeneron Research Site Ann Arbor, Michigan, United States
  • Regeneron Research Site Dallas, Texas, United States
  • Regeneron Research Site Piura, Peru
View Trial Details
Completed

Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia

NCT05053932

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +19 more
Locations
  • Hôpital Robert Debré AP-HP Paris, IDF, France
  • Groupe hospitalier Pellegrin Bordeaux, France
  • Centre Hospitalier Intercommunal de Créteil Créteil, France
  • Hôpital Henri Monr - APHP Créteil, France
View all 14 locations
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Completed

Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease

NCT04255875

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • New Haven Clinical Research Unit New Haven, Connecticut, United States
  • Howard University College of Medicine Washington D.C., District of Columbia, United States
  • Golisano Children's Hospital of Southwest Florida Fort Myers, Florida, United States
  • Lee Health - Golisano Children's Hospital of Southwest Florida Fort Myers, Florida, United States
View all 16 locations
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Completed

Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults

NCT05615220

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +11 more
Locations
  • Harmonex Neuroscience Research Dothan, Alabama, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Advanced Research Center Anaheim, California, United States
  • CenExel CIT-IE Bellflower, California, United States
View all 95 locations
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