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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 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors

NCT04431726

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of Colorado Denver, Children's Hospital Aurora, Colorado, United States
  • Tulane University Health Sciences Center New Orleans, Louisiana, United States
View all 32 locations
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Active, not recruiting

An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

NCT06961344

Conditions
Acid-Base Imbalance, Acidosis, Acidosis, Lactic, +23 more
Locations
  • UC San Diego - Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
View all 16 locations
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Active, not recruiting

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

NCT05606614

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +12 more
Locations
  • City of Hope Duarte, California, United States
  • UC San Diego La Jolla, California, United States
  • UC San Francisco Benioff Children's Hospital Oakland, California, United States
  • Rush University Medical Center Chicago, Illinois, United States
View Trial Details
Active, not recruiting

Pediatric Long-Term Follow-up and Rollover Study

NCT03975829

Conditions
Anaplastic Astrocytoma, Anaplastic Ganglioglioma, Anaplastic Oligodendroglioma, +62 more
Locations
  • Phoenix Children s Hospital Phoenix, Arizona, United States
  • Childrens National Hospital Washington D.C., District of Columbia, United States
  • Nicklaus Childrens Hospital Miami, Florida, United States
  • Indiana Uni School of Medicine Indianapolis, Indiana, United States
View all 55 locations
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Active, not recruiting

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +32 more
Locations
  • Emory University Atlanta, Georgia, United States
  • The Cleveland Clinic Cleveland, Ohio, United States
  • Lysosomal and Rare Disorders Research and Treatment Center, Inc Fairfax, Virginia, United States
  • Royal Melbourne Hospital Parkville, Victoria, Australia
View Trial Details

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