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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

Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)

NCT01983241

Conditions
Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +14 more
Locations
  • Grifols Investigative Site Phoenix, Arizona, United States
  • Grifols Investigative Site Gainesville, Florida, United States
  • Grifols Investigative Site Leesburg, Florida, United States
  • Grifols Investigative Site Miami, Florida, United States
View all 52 locations
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Active, not recruiting

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

NCT05166161

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Children's Hospital Colorado Aurora, Colorado, United States
  • UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism Gainesville, Florida, United States
  • Indiana University School of Medicine Department of Medical and Molecular Genetics Indianapolis, Indiana, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View all 46 locations
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Active, not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

NCT05031780

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of California San Diego La Jolla, California, United States
  • UCLA Health Los Angeles, California, United States
  • Children's Hospital Oakland Oakland, California, United States
  • University of Connecticut Health Center Farmington, Connecticut, United States
View all 91 locations
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Active, not recruiting

Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options

NCT02650401

Conditions
Adenocarcinoma, Adenocarcinoma, Papillary, Brain Diseases, +59 more
Locations
  • University of California San Diego La Jolla, California, United States
  • UCSF Benioff Children's Hospital San Francisco, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Egleston Children's Hospital at Emory University Atlanta Atlanta, Georgia, United States
View all 26 locations
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Active, not recruiting

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

NCT06079281

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +4 more
Locations
  • Research Site Indianapolis, Indiana, United States
  • Research Site Garden City, New York, United States
  • Research Site Durham, North Carolina, United States
  • Research Site Columbus, Ohio, United States
View all 65 locations
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Active, not recruiting

Safety and Efficacy in Patients With Moderate to Severe Subacute and Chronic Atopic Dermatitis

NCT04137562

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Chungnam National University Hospital Daejeon, Chungcheongnam-do, South Korea
  • Korea University AnSan Hospital Ansan, Gyeonggi-do, South Korea
  • Chung-Ang University Hospital Seoul, Seoulteukbyeolsi, South Korea
  • Kyunghee University Medical Center Seoul, Seoulteukbyeolsi, South Korea
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Active, not recruiting

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

NCT03974113

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Children's Hospital Los Angeles Site Number : 8400002 Los Angeles, California, United States
  • Hackensack University Medical Center- Site Number : 8400008 Hackensack, New Jersey, United States
  • University Hospitals of Cleveland- Site Number : 8400007 Cleveland, Ohio, United States
  • Penn State Milton S. Hershey Medical Center- Site Number : 8400006 Hershey, Pennsylvania, United States
View all 16 locations
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