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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.

12,584 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A

NCT06297655

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Fuyang Hospital Affiliated to Anhui Medical University Fuyang, Anhui, China
  • Anhui Provincial Hospital Hefei, Anhui, China
  • The Second Affiliated Hospital of Chongqing Medical University Chongqing, Chongqing Municipality, China
  • The First Hospital of Lanzhou University Lanzhou, Gansu, China
View Trial Details
Active, not recruiting

Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis

NCT04779593

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Haemochromatosis, +7 more
Locations
  • Hopital Avicenne Bobigny, France
  • CHU Dupuytren Limoges, France
  • GHBS site du Scorff Lorient, France
  • GHRMSA - Hôpital Emile Muller Mulhouse, France
View Trial Details
Active, not recruiting

A Trial Investigating Lu AG22515 in Adult Participants With Moderate-to-Severe Thyroid Eye Disease

NCT06557850

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations
  • University Clinical Center of The Republic Of Srpska Banja Luka, Republika Srpska, Bosnia and Herzegovina
  • Diagnostic-Consultative Center Alexandrovska Sofia, Bulgaria
  • University Specialized Hospital for Active Treatment in Endocrinology (USHATE) "Acad. Ivan Penchev" Sofia, Bulgaria
  • Jagiellonian University Medical College Krakow, Lesser Poland Voivodeship, Poland
View Trial Details
Active, not recruiting

A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

NCT05981092

Conditions
Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM), Cardiomegaly, Cardiomyopathies, +6 more
Locations
  • Research Site Palo Alto, California, United States
  • Research Site Aurora, Colorado, United States
  • Research Site Boston, Massachusetts, United States
  • Research Site Rochester, Minnesota, United States
View all 15 locations
View Trial Details
Active, not recruiting

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow

NCT05330325

Conditions
Cardiovascular Abnormalities, Cardiovascular Diseases, Chromosome Disorders, +24 more
Locations
  • Univ of AL at Birmingham_BRM Birmingham, Alabama, United States
  • Children's Hospital Los Angeles - Endocrinology Los Angeles, California, United States
  • Sutter Valley Med Fdt Ped Endo Sacramento, California, United States
  • Rady Childrens Hosp San Diego San Diego, California, United States
View all 199 locations
View Trial Details
Active, not recruiting

A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)

NCT05252585

Conditions
Angiomyolipoma, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +18 more
Locations
  • Novartis Investigative Site Taichung, Taiwan ROC, Taiwan
  • Novartis Investigative Site Taoyuan, Taiwan ROC, Taiwan
  • Novartis Investigative Site Taipei, Taiwan
  • Novartis Investigative Site Taoyuan, Taiwan
View Trial Details
Active, not recruiting

LGMD R1 Natural History Study

NCT05618080

Conditions
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +11 more
Locations
  • University of California, Irvine Orange, California, United States
  • University of Colorado Anschutz Medical Campus Aurora, Colorado, United States
  • University of Florida Gainesville, Florida, United States
  • The Community Health Clinic, Inc. Shipshewana, Indiana, United States
View Trial Details
Active, not recruiting

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California Irvine, Department of Neurology Orange, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Utah, Division of Medical Genetics Salt Lake City, Utah, United States
  • Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility Newcastle upon Tyne, United Kingdom
View Trial Details

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