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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

Prophylaxis Regimen for Hemophilia A Patients

NCT05036278

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Dr. Akshat Jain - Loma Linda University Medical Center Loma Linda, California, United States
  • Loma Linda University Children's Hospital - Hematology / Oncology Loma Linda, California, United States
  • Luskin Orthopaedic Institute for Children Los Angeles, California, United States
  • University of Colorado | Renal Research Office Aurora, Colorado, United States
View Trial Details
Completed

Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease

NCT06482437

Conditions
Charcot-Marie-Tooth Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +9 more
Locations
  • University of Kansas Medical Center, Department of Neurology Kansas City, Kansas, United States
  • Mass General Neurology Boston, Massachusetts, United States
  • NextGen Precision Health Columbia, Missouri, United States
  • Columbia University Medical Center New York, United States
View all 19 locations
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Completed

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

NCT03587116

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Clinical and Translational Research Unit (CTRU) Palo Alto, California, United States
  • Lucile Packard Childrens Hospital Palo Alto, California, United States
  • University of California, San Francisco - Outpatient Hematology Clinic San Francisco, California, United States
  • Stanford Health Care Stanford, California, United States
View all 75 locations
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Completed

Long-term Safety and Efficacy Study of Lebrikizumab (LY3650150) in Participants With Moderate-to-Severe Atopic Dermatitis (ADjoin)

NCT04392154

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • Pinnacle Research Group, LLC Anniston, Alabama, United States
  • Clinical Research Center of Alabama- Birmingham Birmingham, Alabama, United States
  • Investigate MD Scottsdale, Arizona, United States
  • Johnson Dermatology Fort Smith, Arkansas, United States
View all 321 locations
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Completed

Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)

NCT03945292

Conditions
Alpha 1-Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +9 more
Locations
  • Research Site 5 Birmingham, Alabama, United States
  • Research Site 9 Phoenix, Arizona, United States
  • Research Site 14 La Jolla, California, United States
  • Research Site 17 Los Angeles, California, United States
View all 21 locations
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Completed

Molecular-Guided Therapy for Childhood Cancer

NCT02162732

Conditions
Adenocarcinoma, Brain Diseases, Brain Neoplasms, +65 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Rady Children's Hospital San Diego, California, United States
  • Connecticut Children's Hospital Hartford, Connecticut, United States
  • Arnold Palmer Hospital for Children Orlando, Florida, United States
View all 19 locations
View Trial Details

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