Not applicableLooking for participantsNCT00231400What this trial is testingPompe Disease Registry ProtocolWho this might be right forGlycogen Storage Disease Type IIPompe Disease Genzyme, a Sanofi Company 2,000
Not applicableStudy completedNCT00731081What this trial is testingStudy About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®Who this might be right forPompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency Genzyme, a Sanofi Company 8
Testing effectiveness (Phase 2)Ended earlyNCT02898753What this trial is testingVAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe DiseaseWho this might be right forPompe Disease Valerion Therapeutics, LLC 12
Testing effectiveness (Phase 2)Active Not RecruitingNCT04174105What this trial is testingGene Transfer Study in Patients With Late Onset Pompe DiseaseWho this might be right forPompe Disease (Late-onset) Astellas Gene Therapies 11
Not applicableLooking for participantsNCT07478172What this trial is testingEffects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseWho this might be right forNeuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more University of Missouri-Columbia 50
Not applicableStudy completedNCT04120168What this trial is testingStudy Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe DiseaseWho this might be right forDuchenne Muscular DystrophyPompe Disease (Late-onset) Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition 590
Early research (Phase 1)Study completedNCT02240407What this trial is testingRe-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe DiseaseWho this might be right forPompe Disease University of Florida 2
Testing effectiveness (Phase 2)Looking for participantsNCT07123155What this trial is testingStudy of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)Who this might be right forPompe Disease Shionogi 45
Testing effectiveness (Phase 2)Study completedNCT01942590What this trial is testingSafety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement TherapyWho this might be right forPompe Disease Dwight Koeberl, M.D., Ph.D. 17
Not applicableUnknownNCT04981210What this trial is testingHigh Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient ClinicsWho this might be right forPompe Disease National Taiwan University Hospital 1,500
Not applicableStudy completedNCT02801539What this trial is testingRespiratory Muscle Training in L-Onset Pompe Disease (LOPD)Who this might be right forGlycogen Storage Disease Type II Duke University 22
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Not applicableUnknownNCT03045042What this trial is testingSearch for Serum/Plasma Biomarkers in Pompe's DiseaseWho this might be right forLate Onset Pompe DiseasePompe Disease Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 35
Post-approval studies (Phase 4)Study completedNCT00455195What this trial is testingLate-Onset Treatment Study Extension ProtocolWho this might be right forPompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD-II)Glycogenesis Type II+1 more Genzyme, a Sanofi Company 81
Not applicableActive Not RecruitingNCT04929002What this trial is testingCarbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage DiseasesWho this might be right forGlycogen Storage DiseaseMcArdle DiseasePompe Disease (Late-onset) Rigshospitalet, Denmark 50
Large-scale testing (Phase 3)Study completedNCT00158600What this trial is testingA Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe DiseaseWho this might be right forPompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company 90
Post-approval studies (Phase 4)Study completedNCT01288027What this trial is testingExploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase AlfaWho this might be right forPompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency Genzyme, a Sanofi Company 16
Not applicableStudy completedNCT03285126What this trial is testingPompe Gene Therapy- Screening for EligibilityWho this might be right forPompe Disease (Late-onset) Duke University 19
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349What this trial is testingA Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)Who this might be right forPompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more Spark Therapeutics, Inc. 4
Testing effectiveness (Phase 2)WithdrawnNCT04094948What this trial is testingPhase II Clinical Trial of Clenbuterol in Adult Patients With Pompe DiseaseWho this might be right forPompe Disease (Late-onset) Duke University