Not applicableUnknownNCT02527239What this trial is testingPrevalence of Pompe's Disease in Respiratory ClinicsWho this might be right forPompe's Disease Royal Brompton & Harefield NHS Foundation Trust 400
Large-scale testing (Phase 3)Looking for participantsNCT04808505What this trial is testingEvaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18Who this might be right forGlycogen Storage Disease Type II Infantile Onset Amicus Therapeutics 36
Not applicableUnknownNCT00713453What this trial is testingMuscle Response to Enzyme Replacement Therapy in Pompe DiseaseWho this might be right forPompe Disease National Taiwan University Hospital 16
Post-approval studies (Phase 4)Looking for participantsNCT06666413What this trial is testingChina Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPDWho this might be right forGlycogen Storage Disease Type IIPompe's Disease Genzyme, a Sanofi Company 13
Large-scale testing (Phase 3)Study completedNCT02782741What this trial is testingStudy to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II;Pompe's Disease Genzyme, a Sanofi Company 101
Very early researchEnded earlyNCT02357225What this trial is testingA Pilot Study of Pyridostigmine in Pompe DiseaseWho this might be right forPompe Disease University of Florida 2
Post-approval studies (Phase 4)Study completedNCT01410890What this trial is testingPharmacokinetics of Alglucosidase Alfa in Patients With Pompe DiseaseWho this might be right forPompe DiseaseGlycogen Storage Disease Type II (GSD II) Genzyme, a Sanofi Company 21
Not applicableLooking for participantsNCT03564561What this trial is testingNatural History of Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II, Adult Assistance Publique - Hôpitaux de Paris 20
Not applicableLooking for participantsNCT05734521What this trial is testingAvalglucosidase Alfa Pregnancy StudyWho this might be right forPompe DiseasePregnancy Sanofi 100
Not applicableLooking for participantsNCT04639336What this trial is testingCognitive and Neurological Pathologies in Pompe DiseaseWho this might be right forPompe Disease Duke University 45
Not applicableStudy completedNCT00566878What this trial is testingPompe Lactation Sub-RegistryWho this might be right forGlycogen Storage DiseasePompe Disease Genzyme, a Sanofi Company 2
Testing effectiveness (Phase 2)Study completedNCT00467935What this trial is testingScotoma Reduction in AMD Patients Treated With RanibizumabWho this might be right forMacular Degeneration Retina Research Foundation 20
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
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
Not applicableUnknownNCT01914536What this trial is testingA Natural History Study of Adult Onset Pompe Disease Using Muscle MRIWho this might be right forPompe Disease Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 30
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
Testing effectiveness (Phase 2)Study completedNCT01885936What this trial is testingSafety and Efficacy of Albuterol in Individuals With Late-onset Pompe DiseaseWho this might be right forPompe Disease Duke University 16
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
Not applicableUnknownNCT02120235What this trial is testingInvestigating Lysosomal Storage Diseases in Minority GroupsWho this might be right forLysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more O & O Alpan LLC 20,000
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