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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

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

NCT04886518

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disorders of Excessive Somnolence, Dyssomnias, +16 more
Locations
  • UCI Center for Clinical Research Irvine, California, United States
  • University of Colorado School of Medicine Aurora, Colorado, United States
  • University of South Florida Tampa, Florida, United States
  • Rare Disease Research Atlanta, Georgia, United States
View all 13 locations
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Completed

Phosphoproteomic Profile of Children With Down Syndrome

NCT06279208

Conditions
Abnormalities, Multiple, Chromosome Disorders, Congenital Abnormalities, +7 more
Locations
  • CHRU de Brest Brest, France
  • CHU Grenoble Grenoble, France
  • Medilab La Châtaigneraie, France
  • Hospices Civils de Lyon Lyon, France
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Completed

Study of TDM-180935 in Atopic Dermatitis Patients

NCT06363461

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Site 5 Rolling Meadows, Illinois, United States
  • Site 8 Covington, Louisiana, United States
  • Site 4 New Brighton, Minnesota, United States
  • Site 7 Anderson, South Carolina, United States
View Trial Details
Completed

Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)

NCT02766465

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Benioff Children's Hospital at Oakland Oakland, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • University of Florida Gainsville Gainesville, Florida, United States
  • Foundation for Sickle Cell Research/Florida Sickle Inc. Hollywood, Florida, United States
View all 35 locations
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Completed

A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A

NCT04565236

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Beijing Children's Hospital, Capital Medical University Beijing, Beijing Municipality, China
  • The Children's Hospital Zhejiang University School of Med Hangzhou, Hangzhou Province, China
  • Shijiazhuang General Hospital Shijiazhuang, Hebei, China
  • NJ Drum Tower Hospital, the Affil Hos of NJ Univ Med School Nanjing, Jiangsu, China
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