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Clinical trial condition

Lipid Metabolism, Inborn Errors

Explore clinical trials studying Lipid Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.

841 public trials 150 recruiting studies
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Lipid Metabolism, Inborn Errors trial results

Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Childrens Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 39 locations
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Recruiting

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • The Kirklin Clinic Of University of Alabama Birmingham Hospital Birmingham, Alabama, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • MHealth Fairview University of Minnesota Medical Center East Bank Minneapolis, Minnesota, United States
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Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

NCT07082725

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 34 locations
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Recruiting

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

NCT05682378

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +10 more
Locations
  • Excel Medical Clinical Trials LLC Boca Raton, Florida, United States
  • Icahn School of Med at Mt Sinai New York, United States
  • Cincinnati Childrens Hospital MC Cincinnati, Ohio, United States
  • Childrens Hosp Pittsburgh UPMC Pittsburgh, Pennsylvania, United States
View all 52 locations
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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 17 locations
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Recruiting

Clinical Trial in Patients With Barth Syndrome- 4TAZPower

NCT07531251

Conditions
Abnormalities, Multiple, Barth Syndrome, Cardiovascular Abnormalities, +12 more
Locations
  • Trial Not Offered in the U.S Needham, Massachusetts, United States
  • Metabolics and Genetics in Canada (MAGIC) Calgary, Alberta, Canada
  • Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building Bristol, United Kingdom
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