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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.

12,578 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Enrolling by Invitation

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

NCT02402660

Conditions
Autosomal Recessive Stargardt Disease 1 (ABCA4-related), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, +8 more
Locations
  • Alkeus Site Phoenix, Arizona, United States
  • Alkeus Site Los Angeles, California, United States
  • Alkeus Site Aurora, Colorado, United States
  • Alkeus Site Gainesville, Florida, United States
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Enrolling by Invitation

A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability

NCT06940193

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Birmingham Women's and Children's NHS Foundation Trust Birmingham, United Kingdom
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Enrolling by Invitation

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama Birmingham, Alabama, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Healthcare of Atlanta Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
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