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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.

12,674 public trials 2,143 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)

NCT00745420

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • University of Miami Miami, Florida, United States
  • Children's Healthcare of Atlanta Atlanta, Georgia, United States
View all 19 locations
View Trial Details
Completed

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

NCT01998633

Conditions
Blood Protein Disorders, Chronic Active Epstein-Barr Virus Infection, Chronic Disease, +26 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Children's Healthcare of Atlanta Atlanta, Georgia, United States
  • Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 22 locations
View Trial Details
Completed

Evaluation of 611 in Chinese Adult Patients With Moderate-to-Severe Atopic Dermatitis

NCT05641558

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, Dermatitis, Atopic, +8 more
Locations
  • The First Affiliated Hospital of Wannan Medical College Wuhu, Anhui, China
  • Peking University Shougang Hospital Beijing, Beijing Municipality, China
  • Nanyang First People's Hospital Nanyang, Henan, China
  • Dermatology Hospital of Jiangxi Province Nanchang, Jiangxi, China
View Trial Details
Completed

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

NCT02312011

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • Stanford University Medical Center Stanford, California, United States
  • University of Florida Gainesville, Florida, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • Kennedy Krieger Institute Baltimore, Maryland, United States
View Trial Details
Completed

The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease

NCT03487913

Conditions
Abnormalities, Multiple, Autosomal Dominant Polycystic Kidney Disease, Ciliopathies, +12 more
Locations
  • Palladio Biosciences Clinical Site Los Angeles, California, United States
  • Palladio Biosciences Clinical Site Jacksonville, Florida, United States
  • Palladio Biosciences Clinical Site Miami, Florida, United States
  • Palladio Biosciences Clinical Site Miami, Florida, United States
View all 13 locations
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Completed

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

NCT01362595

Conditions
Anemia, Anemia, Aplastic, Anemia, Diamond-Blackfan, +14 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Stanford University Medical Center Palo Alto, California, United States
  • Riley Hospital for Children Indianapolis, Indiana, United States
  • University of Louisville Louisville, Kentucky, United States
View Trial Details

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