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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.

12,603 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

NCT03754790

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Phoenix Childrens Hospital Site Number : 8400009 Phoenix, Arizona, United States
  • Children's Hospital Los Angeles Site Number : 8400019 Los Angeles, California, United States
  • Center for Inherited Blood Disorders (CIBD) Site Number : 8400016 Orange, California, United States
  • Nemours Children's Clinic Site Number : 8400008 Jacksonville, Florida, United States
View all 79 locations
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Active, not recruiting

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Mayo Clinic Florida Jacksonville, Florida, United States
  • University of Miami Miami, Florida, United States
View all 19 locations
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Active, not recruiting

A Study to Learn if a Medicine That Reduces Stomach Acid Affects the Blood Level of Study Medicine PF-08049820 in Healthy Chinese Adults.

NCT07284173

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, Dermatitis, Atopic, +9 more
Locations
  • Beijing Friendship Hospital Affiliate of Capital University Beijing, Beijing Municipality, China
  • Beijing Friendship Hospital Affiliate of Capital University Beijing, Beijing Municipality, China
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Active, not recruiting

A Phase I Study, Evaluating the Safety, Pharmacokinetics and Efficacy of PRJ1-3024 in Subjects With Advanced Solid Tumors

NCT05159700

Conditions
Advanced Solid Malignancies, Advanced Solid Tumor, Congenital Abnormalities, +10 more
Locations
  • Sarah Cannon Research Institute at HealthONE Denver, Colorado, United States
  • Sarah Cannon Research Institute at Florida Cancer Specialists Orlando, Florida, United States
  • Christ Hospital Cincinnati, Ohio, United States
  • NEXT Oncology Austin, Texas, United States
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Active, not recruiting

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

NCT05392114

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Ionis Investigative Site Paradise Valley, Arizona, United States
  • Ionis Investigative Site La Jolla, California, United States
  • Ionis Investigative Site Santa Monica, California, United States
  • Ionis Investigative Site Walnut Creek, California, United States
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Active, not recruiting

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)

NCT05464381

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epidermolysis Bullosa, +6 more
Locations
  • Sanatorio Mater Dei Buenos Aires, Argentina
  • EB-Haus Austria; Salzburger Landeskliniken (SALK) Salzburg, Austria
  • Fundacion Debra Chile Santiago, Chile
  • Universitaetsklinik fuer Dermatologie und Allergologie Nice, France
View all 16 locations
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