Not applicableLooking for participantsNCT05734521What this trial is testingAvalglucosidase Alfa Pregnancy StudyWho this might be right forPompe DiseasePregnancy Sanofi 100
Post-approval studies (Phase 4)Ended earlyNCT01526785What this trial is testingEvaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe DiseaseWho this might be right forPompe Disease Genzyme, a Sanofi Company 113
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
Not applicableStudy completedNCT04907162What this trial is testingMusculoskeletal Nociceptive Pain in Participants With Neuromuscular DisordersWho this might be right forPompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more LMU Klinikum 82
Early research (Phase 1)Study completedNCT03533673What this trial is testingAAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe DiseaseWho this might be right forPompe Disease AskBio Inc 7
Post-approval studies (Phase 4)UnknownNCT02405624What this trial is testingCPAP for Infantile Pompe DiseaseWho this might be right forPompe Disease National Taiwan University Hospital 3
Post-approval studies (Phase 4)Looking for participantsNCT06875245What this trial is testingComparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment IntervalsWho this might be right forWet Age-related Macular Degeneration Faculty Hospital Kralovske Vinohrady 100
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 applicableApproved For MarketingNCT00074919What this trial is testingExpanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe DiseaseWho this might be right forGlycogen Storage Disease Type IIGlycogenosis 2 Genzyme, a Sanofi Company
Testing effectiveness (Phase 2)Study completedNCT00051935What this trial is testingThe Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type IIWho this might be right forGlycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company 2
Large-scale testing (Phase 3)Study completedNCT03729362What this trial is testingA Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe DiseaseWho this might be right forPompe Disease (Late-onset) Amicus Therapeutics 125
Not applicableAvailableNCT04327973What this trial is testingExpanded Access for ATB200/AT2221 for the Treatment of IOPDWho this might be right forPompe Disease Infantile-Onset Amicus Therapeutics
Not applicableStudy completedNCT02363153What this trial is testingDiet and Exercise in Pompe DiseaseWho this might be right forPompe DiseaseGlycogen Storage Disease Type IILysosomal Storage Diseases University of Florida 2
Not applicableUnknownNCT02635269What this trial is testingFat and Sugar Metabolism During Exercise in Patients With Metabolic MyopathyWho this might be right forMetabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more Rigshospitalet, Denmark 60
Not applicableStudy completedNCT00001331What this trial is testingGenetic and Family Studies of Inherited Muscle DiseasesWho this might be right forDermatomyositisGlycogen Storage Disease Type IIGlycogen Storage Disease Type VII+2 more National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Not applicableStudy completedNCT01710813What this trial is testingAlglucosidase Alfa Pompe Safety Sub-RegistryWho this might be right forPompe Disease Genzyme, a Sanofi Company 110
Post-approval studies (Phase 4)Study completedNCT03687333What this trial is testingEvaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa TreatmentWho this might be right forGlycogen Storage Disease Type II Genzyme, a Sanofi Company 10
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
Not applicableUnknownNCT03812042What this trial is testingScreening of Lysosomal Storage Disorders Diseases in Minority GroupsWho this might be right forLysosomal Storage Diseases Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100,000
Large-scale testing (Phase 3)Active Not RecruitingNCT04910776What this trial is testingClinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaWho this might be right forGlycogen Storage Disease Type II Sanofi 17