Early research (Phase 1)Not Yet RecruitingNCT07787650What this trial is testingS-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)Who this might be right forPompe Disease Shionogi 15
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
Large-scale testing (Phase 3)Study completedNCT00125879What this trial is testingExtension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602Who this might be right forGlycogen Storage Disease Type II Genzyme, a Sanofi Company 16
Large-scale testing (Phase 3)Study completedNCT00059280What this trial is testingThe Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II Genzyme, a Sanofi Company 16
Post-approval studies (Phase 4)UnknownNCT02525172What this trial is testingImmune Modulation Therapy for Pompe DiseaseWho this might be right forPompe Disease National Taiwan University Hospital 8
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
Not applicableUnknownNCT04476550What this trial is testingClinical Specimen Collection From Pompe Disease PatientsWho this might be right forPompe Disease Serhat Gumrukcu, MD PhD 12
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 applicableActive Not RecruitingNCT03694561What this trial is testingDeveloping a Management Approach for Patients With "Late-Onset" Pompe DiseaseWho this might be right forPompe DiseasePompe Disease (Late-onset)GAA Deficiency Duke University 20
Not applicableStudy completedNCT03147664What this trial is testingCardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe DiseaseWho this might be right forEnzyme Replacement Therapy in Pompe Disease Rambam Health Care Campus 5
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
Not applicableUnknownNCT05567627What this trial is testingClinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe DiseaseWho this might be right forInfantile-onset Pompe Disease Seventh Medical Center of PLA General Hospital 6
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
Testing effectiveness (Phase 2)Study completedNCT00250939What this trial is testingRhGAA 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 5
Very early researchNot Yet RecruitingNCT06178432What this trial is testingEvaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)Who this might be right forPompe Disease (Late-onset) Huashan Hospital 6
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
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
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
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
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