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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

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

NCT07416526

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Nara Medical University Hospital Kashihara-shi, Nara, Japan
  • Tokyo Medical University Hospital Shinjuku-Ku, Tokyo, Japan
  • University of Colorado Hemophilia and Thrombosis Center Aurora, Colorado, United States
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
View all 27 locations
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Recruiting

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

NCT07491172

Conditions
Cardiovascular, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, +20 more
Locations
  • Integrity Clinical Research Hialeah, Florida, United States
  • Jacksonville Center for Clinical Research Jacksonville, Florida, United States
  • Flourish Orlando Orlando, Florida, United States
  • United Medical Research Port Orange, Florida, United States
View all 17 locations
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Recruiting

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old

NCT07536269

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +16 more
Locations
  • Neurocrine Clinical Site San Francisco, California, United States
  • Neurocrine Clinical Site Hartford, Connecticut, United States
  • Neurocrine Clinical Site Atlanta, Georgia, United States
  • Neurocrine Clinical Site Chicago, Illinois, United States
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Recruiting

Specimen Collection From Pregnant Women at Increased Risk for Fetal Aneuploidy

NCT01429389

Conditions
Abnormalities, Multiple, Aneuploidy, Chromosome Aberrations, +14 more
Locations
  • University of Alabama Birmingham Birmingham, Alabama, United States
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • Sharp-Rees Stealy Medical Group San Diego, California, United States
  • UCSD San Diego, California, United States
View all 14 locations
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Recruiting

A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)

NCT05582993

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +8 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Bleeding and Clotting Disorders Institute Peoria, Illinois, United States
  • Riley Hospital for Children Indiana University Health Indianapolis, Indiana, United States
  • University of Iowa Hospitals & Clinics PARENT Iowa City, Iowa, United States
View all 23 locations
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Recruiting

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

NCT06546670

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama Birmingham Birmingham, Alabama, United States
  • Quotient Sciences Sea View Miami, Florida, United States
  • Boston Childrens Hospital Boston, Massachusetts, United States
  • East Carolina University Greenville, North Carolina, United States
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