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Clinical trial condition

Metabolism, Inborn Errors

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

2,493 public trials 456 recruiting studies
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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 (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 35 locations
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Recruiting

Clinical and Basic Investigations Into Congenital Disorders of Glycosylation

NCT04199000

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital Disorders of Glycosylation, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +4 more
Locations
  • Rady Children's Hospital San Diego, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Mayo Clinic Florida Jacksonville, Florida, United States
  • Tulane University School of Medicine New Orleans, Louisiana, United States
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Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California, Irvine (UCI) Irvine, California, United States
  • Stanford Neuroscience Health Center Palo Alto, California, United States
  • NYU Langone New York, United States
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Recruiting

Study of Skeletal Disorders

NCT05031507

Conditions
Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +15 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
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Recruiting

Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia

NCT06451770

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +11 more
Locations
  • Clinical Study Center Clearwater, Florida, United States
  • Clinical Study Center Miami Lakes, Florida, United States
  • Clinical Study Center Port Orange, Florida, United States
  • Clinical Study Center New York, United States
View all 16 locations
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