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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.

12,656 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

NCT05266014

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations
  • Sydney Children's Hospitals Network Westmead, New South Wales, Australia
  • Lions Eye Institute Perth, Western Australia, Australia
  • National Taiwan University Hospital Taipei, Taiwan
View Trial Details
Completed

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

NCT04279314

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Translational Genomics Research Institute (TGen) Phoenix, Arizona, United States
  • University of California, San Diego La Jolla, California, United States
  • UC Davis MIND Institute Sacramento, California, United States
View all 21 locations
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Completed

Safety and Efficacy of ASN-002 Combined With a Hedgehog Pathway Inhibitor

NCT04416516

Conditions
Abnormalities, Multiple, Basal Cell Carcinoma, Basal Cell Nevus Syndrome, +19 more
Locations
  • Central Brisbane Dermatology Brisbane, Queensland, Australia
  • Princess Alexandra Hospital Brisbane, Queensland, Australia
  • Veracity Clinical Research Brisbane, Queensland, Australia
  • Sunshine Coast University Hospital Sunshine Coast, Queensland, Australia
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Completed

A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients

NCT03815695

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • Woodland International Research Group (SCD subjects only) Little Rock, Arkansas, United States
  • Collaborative Neuroscience Research, LLC (SCD subjects only) Long Beach, California, United States
  • Pacific Research Partners (SCD subjects only) Oakland, California, United States
  • UCSF Benioff Children's Hospital Oakland (SCD subjects only) Oakland, California, United States
View all 18 locations
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