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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.

12,573 public trials 2,159 recruiting studies
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Genetic Diseases, Inborn trial results

Enrolling by Invitation

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

NCT06075537

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Hackensack University Medical Center Hackensack, New Jersey, United States
  • UNC Children's Research Institute Chapel Hill, North Carolina, United States
View all 29 locations
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Enrolling by Invitation

GABA and GSH in FRDA

NCT07635030

Conditions
Brain Diseases, Central Nervous System Diseases, Cerebellar Diseases, +13 more
Locations
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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Enrolling by Invitation

Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)

NCT06101940

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • Chinese People's Liberation Army General Hospital Beijing, Beijing Municipality, China
  • Peking University First Hospital Beijing, Beijing Municipality, China
  • First Affiliated Hospital of Chongqing Medical University Chongqing, Chongqing Municipality, China
  • Fujian Medical University Union Hospital Fuzhou, Fujian, China
View all 22 locations
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Enrolling by Invitation

A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

NCT06431893

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Emory University Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 52 locations
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