Glycogen Storage Disease II: Clinical Trials

2 trials for Glycogen Storage Disease II, newest first

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1 active not recruiting, 1 completed. .

Follow Glycogen Storage Disease II: new trials by email Literature Review of Glycogen Storage Disease II trials Research Report on Glycogen Storage Disease II

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Early Check: Expanded Screening in Newborns
Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome
Active not recruitingRTI International300002018-10-152026-07-01
Pompe Telemedicine Developmental Study
Pompe Disease, Glycogen Storage Disease II
CompletedDuke University222015-12-012021-10-28

Narrower conditions

Glycogen Storage Disease Type II (2)  ·  3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1)  ·  3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1)  ·  3-methylcrotonyl CoA carboxylase 1 deficiency (1)  ·  3-methylcrotonyl CoA carboxylase 2 deficiency (1)  ·  Acidemia, isovaleric (1)  ·  Acrodermatitis enteropathica (1)  ·  Adenine phosphoribosyltransferase deficiency (1)  ·  Adrenal hyperplasia, congenital, type 5 (1)  ·  Adrenocorticotropic hormone deficiency (1)  ·  Amaurosis congenita of Leber, type 2 (1)  ·  Anemia, Sickle Cell (1)  ·  Angelman Syndrome (1)  ·  Aortic Stenosis, Supravalvular (1)  ·  Arginine-Glycine Amidinotransferase Deficiency (1)

Broader conditions

Brain Diseases (2)  ·  Brain Diseases, Metabolic (2)  ·  Brain Diseases, Metabolic, Inborn (2)  ·  Carbohydrate Metabolism, Inborn Errors (2)  ·  Central Nervous System Diseases (2)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2)  ·  Genetic Diseases, Inborn (2)  ·  Glycogen Storage Disease (2)  ·  Lysosomal Storage Diseases (2)  ·  Lysosomal Storage Diseases, Nervous System (2)  ·  Metabolic Diseases (2)  ·  Metabolism, Inborn Errors (2)  ·  Nervous System Diseases (2)  ·  Nutritional and Metabolic Diseases (2)  ·  Abnormalities, Multiple (1)

Sponsors

Duke University (1)  ·  RTI International (1)

Countries

United States (2)

Top investigators

Curt Scharfe, MD, PhD (1)  ·  Priya Kishnani (1)

Results publications

PMID 31315600, PMID 35142618