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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.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

NCT06204809

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • UCI Center for Clinical Research Irvine, California, United States
  • Stanford University Palo Alto, California, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • University of Kansas Medical Center Fairway, Kansas, United States
View Trial Details
Completed

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT03655678

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • Lucile Packard Children's Hospital Palo Alto, California, United States
  • Ann & Robert Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Columbia University Medical Center (21+ years) New York, United States
  • Columbia University Medical Center New York, United States
View all 14 locations
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Completed

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

NCT04987489

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +15 more
Locations
  • TOI Clinical Research Cerritos, California, United States
  • [Legal] Children's Hospital Los Angeles Los Angeles, California, United States
  • University of Californ LA-UCLA Los Angeles, California, United States
  • UCSF Oakland Benioff ChildHosp Oakland, California, United States
View all 32 locations
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Completed

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

NCT04722887

Conditions
Alpha1-Antitrypsin Deficiency, Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +14 more
Locations
  • UCLA Medical Center Los Angeles, California, United States
  • University of Florida Gainesville, Florida, United States
  • University of Miami Miami, Florida, United States
  • Cleveland Clinic Cleveland, Ohio, United States
View Trial Details
Completed

Study of Infigratinib in Children With Achondroplasia

NCT04265651

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Nemours Alfred I. Dupont Hospital for Children Wilmington, Delaware, United States
  • Johns Hopkins School of Medicine Baltimore, Maryland, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
View all 19 locations
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