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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

Active, Not Recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

NCT04848779

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center- Site Number : 8400001 Cincinnati, Ohio, United States
  • Le Bonheur Children's Hospital- Site Number : 8400005 Memphis, Tennessee, United States
View all 15 locations
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Active, Not Recruiting

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California Irvine, Department of Neurology Orange, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Utah, Division of Medical Genetics Salt Lake City, Utah, United States
  • Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility Newcastle upon Tyne, United Kingdom
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Active, Not Recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Stanford Hospital- Site Number : 8400006 Stanford, California, United States
  • Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 Minneapolis, Minnesota, United States
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
View all 16 locations
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Active, Not Recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Investigational Site Number : 2500004 Bordeaux, France
  • Investigational Site Number : 2500005 Brest, France
  • Investigational Site Number : 2500008 Clermont-Ferrand, France
  • Investigational Site Number : 2500009 Lille, France
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Active, Not Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Wolfson Children's Hospital Jacksonville, Florida, United States
  • Woodruff Memorial Research Building Atlanta, Georgia, United States
  • St. Louis Children's Hospital St Louis, Missouri, United States
View all 17 locations
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Active, Not Recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349

Conditions
Acid Maltase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +17 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California Irvine Health Orange, California, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Kansas Medical Center Research Institute Kansas City, Kansas, United States
View all 29 locations
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