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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

Recruiting

Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)

NCT07587021

Conditions
Calculi, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +21 more
Locations
  • University of Alabama at Birmingham (UAB Hospital) Birmingham, Alabama, United States
  • Childrens Hospital of Colorado Aurora, Colorado, United States
  • Washington University/Barnes Jewish Hospital St Louis, Missouri, United States
  • University of Pennsylvania Health System (UPHS) - Penn Medicine Philadelphia, Pennsylvania, United States
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Recruiting

Pivotal Study of N-acetyl-L-leucine for CACNA1A

NCT07221292

Conditions
Ataxia, Brain Diseases, CACNA1A, +17 more
Locations
  • Johns Hopkins University Baltimore, Maryland, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • The University of Texas Health (UT Health) Houston, Texas, United States
  • Medical University of Innsbruck Innsbruck, Austria
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Recruiting

Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients

NCT06730126

Conditions
Autoimmune Diseases, Autoimmune Lymphoproliferative Syndrome, Autoimmune Lymphoproliferative Syndrome, Type IA, +9 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Texas Children's Hospital Houston, Texas, United States
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Recruiting

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

NCT04883905

Conditions
Acute Hepatic Porphyria, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Coproporphyria, Hereditary, +10 more
Locations
  • Clinical Trial Site Birmingham, Alabama, United States
  • Clinical Trial Site Gilbert, Arizona, United States
  • Clinical Trial Site San Francisco, California, United States
  • Clinical Trial Site Baltimore, Maryland, United States
View all 30 locations
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Recruiting

The Mechanistic Biology of Primary Immunodeficiency Disorders

NCT03394053

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Immune System Diseases, +3 more
Locations
  • Children's National Health System (CNHS) Washington D.C., District of Columbia, United States
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
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Recruiting

SCD Stem Cell Mobilization and Apheresis Using Motixafortide

NCT06442761

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • National Heart, Blood and Lung Institute-Cellular and Molecular Therapy Branch Bethesda, Maryland, United States
  • St. Jude Children's Research Hospital Memphis, Tennessee, United States
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Recruiting

Pan-Amyloid PET/CT in Various Amyloid-Related Disease

NCT07130565

Conditions
Alzheimer Disease, Amyloid, Amyloid Neuropathies, +21 more
Locations
  • The First Affiliated Hospital of University of Science and Technology of China Hefei, Anhui, China
  • Tianjin Medical University General Hospital Tianjin, Tianjin Municipality, China
  • The Affiliated Hospital of Qingdao University Qingdao, Shandong, China
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