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Published and searchable clinical trials

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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
View Trial Details
Recruiting

Pompe Pregnancy Sub-Registry

NCT00567073

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Barrow Neurol Group- Site Number : 840087 Phoenix, Arizona, United States
  • University of Florida Pediatrics Genetics- Site Number : 157138 Jacksonville, Florida, United States
  • Emory University School Of Medicine- Site Number : 840060 Atlanta, Georgia, United States
  • Indianapolis University School of Medicine- Site Number : 840027 Indianapolis, Indiana, United States
View all 35 locations
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
View Trial Details
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
View Trial Details
Completed

MSOT in Pompe Disease

NCT05083806

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • University Hospital Erlangen Erlangen, Bavaria, Germany
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Completed

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

NCT03893240

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
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
View all 18 locations
View Trial Details
Completed

Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease

NCT04120168

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +21 more
Locations
  • Adana City Hospital Adana, Turkey (Türkiye)
  • Baskent University Adana Hospital Adana, Turkey (Türkiye)
  • Çukurova University Faculty of Medicine, Pediatric Gastroenterology Adana, Turkey (Türkiye)
  • Afyonkarahisar Health Sciences University Afyonkarahisar, Turkey (Türkiye)
View all 51 locations
View Trial Details
Completed

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600

Conditions
Acid Maltase Deficiency Disease, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • Tower Hematology Oncology Medical Group Beverly Hills, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Washington University Medical Center St Louis, Missouri, United States
  • Mount Sinai School of Medicine New York, United States
View Trial Details
Active, Not Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Emory Clinic Atlanta, Georgia, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • University of Michigan Ann Arbor, Michigan, United States
  • Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States
View all 53 locations
View Trial Details