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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

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

NCT03826628

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Facial Angiofibroma, +13 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • University of California San Diego La Jolla, California, United States
  • All Children's Research Institute St. Petersburg, Florida, United States
  • Spectrum Health Grand Rapids, Michigan, United States
View all 17 locations
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Completed

A Study to Evaluate the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

NCT04864743

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Nanfang Hospital of Southern Medical University Guangzhou, China
  • People's Hospital of Zhengzhou Zhengzhou, Henan, China
  • Jinan central hospital Jinan, Shandong, China
  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College. Tianjin, Tianjin Municipality, China
View Trial Details
Completed

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation

NCT02725567

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Birmingham, Alabama, United States
  • Palo Alto, California, United States
  • Atlanta, Georgia, United States
  • Chicago, Illinois, United States
View all 23 locations
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Completed

Simplified Diet Approach in Phenylketonuria

NCT02555579

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Monroe-Meyer Insititute Omaha, Nebraska, United States
  • Children's Hospital & Medical Center Omaha, Nebraska, United States
  • Nebraska Medicine, Midtown Health Center Omaha, Nebraska, United States
  • Unversity of Nebraska Medical Center Omaha, Nebraska, United States
View Trial Details
Completed

Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy

NCT06027385

Conditions
Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, +13 more
Locations
  • RoMed Hospital Rosenheim, Bavaria, Germany
  • University Hospital Essen, Center for Pediatrics and Adolescent Medicine Essen, Germany
  • Dr. von Haunersches Kinderspital München, Germany
  • University Hospital Münster, Clinic and Polyclinic for Pediatrics and Adolescent Medicine Münster, Germany
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Completed

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease

NCT04285827

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Illinois Hospital and Health Science Systems Chicago, Illinois, United States
  • The Johns Hopkins Hospital Baltimore, Maryland, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Jacobi Medical Center The Bronx, New York, United States
View all 15 locations
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Completed

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care

NCT01431326

Conditions
Adenoviridae Infections, Adenovirus, Adrenal Gland Diseases, +162 more
Locations
  • Alaska Native Medical Center Anchorage, Alaska, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • University of California at San Diego Medical Center La Jolla, California, United States
  • Axis Clinical Trials Los Angeles, California, United States
View all 52 locations
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