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
Not applicableEnded earlyNCT03347253What this trial is testingSTRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERTWho this might be right forLate-onset Pompe Disease Amicus Therapeutics 12
Large-scale testing (Phase 3)Active Not RecruitingNCT03911505What this trial is testingZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDWho this might be right forPompe Disease (Late-onset) Amicus Therapeutics 21
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
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
Early research (Phase 1)Study completedNCT01859624What this trial is testingAlbuterol in Individuals With Late Onset Pompe Disease (LOPD)Who this might be right forPompe Disease Duke University 8
Post-approval studies (Phase 4)Active Not RecruitingNCT07652814What this trial is testingSafety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 YearsWho this might be right forGlycogen Storage Disease Type II Late OnsetPompe Disease (Late-onset)Pompe Disease Late-Onset+2 more Iris Plug 6
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07750990What this trial is testingAn Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)Who this might be right forPompe Disease Shionogi 60
Not applicableStudy completedNCT05431127What this trial is testingHigh Dose Inspiratory Muscle Training in LOPDWho this might be right forLate-Onset Pompe DiseaseLysosomal Disease Duke University 34
Not applicableStudy completedNCT02824068What this trial is testingLong-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)Who this might be right forPompe Disease LMU Klinikum 112
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
Testing effectiveness (Phase 2)Study completedNCT00765414What this trial is testingExtension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.Who this might be right forPompe Disease Late-OnsetGlycogen Storage Disease Type II GSD II Genzyme, a Sanofi Company 1
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
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
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
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
Very early researchStudy completedNCT06109948What this trial is testingStudy to Assess the Safety, Tolerability, PK and PD of ABX1100Who this might be right forHealthyLate Onset Pompe Disease Aro Biotherapeutics 46
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 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
Large-scale testing (Phase 3)Study completedNCT00268944What this trial is testingSafety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportWho 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 5