Testing effectiveness (Phase 2)Study completedNCT01380743What this trial is testingDrug-drug Interaction StudyWho this might be right forPompe Disease Amicus Therapeutics 25
Not applicableUnknownNCT05272969What this trial is testingPompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseaseWho this might be right forPompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more LMU Klinikum 95
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 applicableUnknownNCT04755751What this trial is testingExercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.Who this might be right forPompe Disease Infantile-Onset Rambam Health Care Campus 4
Not applicableLooking for participantsNCT05989620What this trial is testingLong-Term Development of Muscular Dystrophy Outcome AssessmentsWho this might be right forLGMD1BLGMD1CLGMD1D+26 more Virginia Commonwealth University 1,000
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
Not applicableEnrolling By InvitationNCT07072676What this trial is testingThe Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Who this might be right forInclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more LMU Klinikum 30
Testing effectiveness (Phase 2)Study completedNCT01230801What this trial is testingSafety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe DiseaseWho this might be right forPompe Disease BioMarin Pharmaceutical 22
Not applicableLooking for participantsNCT04639336What this trial is testingCognitive and Neurological Pathologies in Pompe DiseaseWho this might be right forPompe Disease Duke University 45
Large-scale testing (Phase 3)Ended earlyNCT01924845What this trial is testingBMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)Who this might be right forLate-onset Pompe Disease BioMarin Pharmaceutical 24
Not applicableLooking for participantsNCT07834320What this trial is testingPompe Disease RWE Long-term Monitoring ProgramWho this might be right forPompe Disease National Taiwan University Hospital 40
Not applicableStudy completedNCT01758354What this trial is testingNewborn Screening Assay of Pompe's DiseaseWho this might be right forPompe Disease National Taiwan University Hospital 236,536
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)Looking for participantsNCT06391736What this trial is testingEvaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy DrugWho this might be right forPompe Disease (Late-onset) GeneCradle Inc 33
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 applicableWithdrawnNCT01457443What this trial is testingBiomarker for Pompe Disease (BioPompe)Who this might be right forCardiac DiseasesMuscular WeaknessHepato-splenomegaly+4 more CENTOGENE GmbH Rostock
Not applicableWithdrawnNCT02385162What this trial is testingBiomarker for Glycogen Storage Diseases (BioGlycogen)Who this might be right forFructose Metabolism, Inborn ErrorsGlycogen Storage DiseaseGlycogen Storage Disease Type I+7 more CENTOGENE GmbH Rostock
Early research (Phase 1)Study completedNCT01898364What this trial is testingSafety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.Who this might be right forPompe DiseaseGlycogen Storage Disease Type II (GSD II)Acid Maltase Deficiency Genzyme, a Sanofi Company 24
Not applicableEnded earlyNCT00277979What this trial is testing3D Echocardiography Managing Infantile Pompe's DiseaseWho this might be right forCongenital Disorders Children's Healthcare of Atlanta 2
Large-scale testing (Phase 3)Study completedNCT04138277What this trial is testingAssess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)Who this might be right forPompe Disease (Late-onset) Amicus Therapeutics 119