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Clinical trial condition

Glycogen Storage Disease

Explore clinical trials studying Glycogen Storage Disease. Study availability and eligibility vary by location and protocol.

129 public trials 27 recruiting studies
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Glycogen Storage Disease trial results

Completed

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

NCT05139316

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +8 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • University of Connecticut Health Center Farmington, Connecticut, United States
  • Mount Sinai The Bronx, New York, United States
View all 20 locations
View Trial Details
Completed

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +9 more
Locations
  • UCONN Health Farmington, Connecticut, United States
  • Michigan Medicine University of Michigan Ann Arbor, Michigan, United States
  • University of Texas Health Science Center at Houston Houston, Texas, United States
  • Montreal Children Hospital, McGill University Health Centre Montreal, Quebec, Canada
View Trial Details
Completed

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

NCT04138277

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • University of California, Irvine Irvine, California, United States
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Emory Clinic Atlanta, Georgia, United States
View all 60 locations
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Completed

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Neuromuscular Research Centre Phoenix, Arizona, United States
  • University of California Irvine Orange, California, United States
  • University of Florida Gainesville, Florida, United States
  • Emory University Division of Medical Genetics Decatur, Georgia, United States
View all 19 locations
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Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Neuromuscular Research Center Phoenix, Arizona, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • University of California, Irvine Irvine, California, United States
  • UF Helath: University of Florida Clinical Research Center Gainesville, Florida, United States
View all 73 locations
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Completed

Triheptanoin in Mc Ardle

NCT02919631

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +5 more
Locations
  • Pitié-Salpêtrière,centre de référence de pathologie neuromusculaire, 47 Bd de l'hôpital Paris, France
View Trial Details

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