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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.

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)

NCT07062588

Conditions
Bone Diseases, Bone Diseases, Developmental, Collagen Diseases, +8 more
Locations
  • Phoenix Children's Phoenix, Arizona, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Nemours/Alfred I. duPont Hospital for Children Wilmington, Delaware, United States
  • Indiana University School of Medicine, Department of Medicine and Pediatrics Division of Endocrinology Indianapolis, Indiana, United States
View all 26 locations
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Recruiting

A Long-term Safety and Efficacy Study Evaluating APG777 in Atopic Dermatitis

NCT07003425

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Investigational Site Fountain Valley, California, United States
  • Investigational Site Los Angeles, California, United States
  • Investigational Site Coral Gables, Florida, United States
  • Investigational Site Jacksonville, Florida, United States
View all 63 locations
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Recruiting

Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

NCT04509050

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • The Children's Hospital Alabama, University of Alabama at Birmingham Birmingham, Alabama, United States
  • Stanford University Medical Center Palo Alto, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Nemours Children's Clinic Jacksonville, Florida, United States
View all 35 locations
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Recruiting

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06379789

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Orthopaedic Hemophilia Treatment Center Los Angeles, California, United States
  • David Geffen School of Medicine at UCLA Los Angeles, California, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California Davis Sacramento, California, United States
View all 44 locations
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Recruiting

Study of Inherited Neurological Disorders

NCT00004568

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Motor Neuron Disease, +11 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
  • University of Mali Bamako, Mali
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Recruiting

Hungarian National Systemic Amyloidosis Registry

NCT07689331

Conditions
AL Amyloidosis, ATTR Amyloidosis, Amyloid Cardiomyopathy, +24 more
Locations
  • Balatonfüredi Állami Szívkórház Balatonfüred, Hungary
  • Budapesti Szent Margit Kórház Budapest, Hungary
  • Budapesti Péterfy Sándor utcai Kórház - Rendelőintézet Budapest, Hungary
  • Department of Internal Medicine and Hematology, Semmelweis University Budapest, Hungary
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Recruiting

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

NCT06971731

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of California Los Angeles (UCLA) School of Medicine Los Angeles, California, United States
  • University of Florida (UF) Health Shands Hospital Gainesville, Florida, United States
  • University of South Florida Tampa, Florida, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 26 locations
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