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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.

12,522 public trials 2,142 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Indiana University School of Medicine Indianapolis, Indiana, United States
  • Women and Children Hospital North Adelaide, Australia
  • The Royal Children's Hospital Parkville, Australia
  • Centre Hospitalier Universitaire (CHU) de Lille - Hôpital Jeanne de Flandre Lille, France
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Recruiting

An Open-label, Rollover Trial to Evaluate the Efficacy and Safety of AMG 732 in Participants With Thyroid Eye Disease

NCT07438405

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations
  • Applied Research Center of Arkansas Little Rock, Arkansas, United States
  • Ilumina Medical Research Kissimmee, Florida, United States
  • Vision Medical Research, Inc. Orland Park, Illinois, United States
  • West Virginia University Morgantown, West Virginia, United States
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Recruiting

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

NCT07491172

Conditions
Cardiovascular, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, +20 more
Locations
  • Integrity Clinical Research Hialeah, Florida, United States
  • Jacksonville Center for Clinical Research Jacksonville, Florida, United States
  • Flourish Orlando Orlando, Florida, United States
  • United Medical Research Port Orange, Florida, United States
View all 19 locations
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Recruiting

FOG-001 in Locally Advanced or Metastatic Solid Tumors

NCT05919264

Conditions
Adamantinomatous Craniopharyngioma, Adenocarcinoma, Adenoma, +65 more
Locations
  • Mayo Clinic Phoenix, Arizona, United States
  • Honor Health Scottsdale, Arizona, United States
  • Arizona Cancer Center at University of Arizona Tucson, Arizona, United States
  • University of California, Los Angeles (UCLA) Los Angeles, California, United States
View all 33 locations
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Recruiting

A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)

NCT07723248

Conditions
Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +24 more
Locations
  • Urology Specialists of the Carolinas - University Place Charlotte, North Carolina, United States
  • Summit Urology Murray, Utah, United States
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