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 applicableStudy completedNCT00731081What this trial is testingStudy About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®Who this might be right forPompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency Genzyme, a Sanofi Company 8
Post-approval studies (Phase 4)Study completedNCT00701129What this trial is testingAn Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe DiseaseWho this might be right forPompe DiseaseGlycogen Storage Disease Type II Genzyme, a Sanofi Company 4
Not applicableApproved For MarketingNCT00520143What this trial is testingAlglucosidase Alfa Temporary Access ProgramWho this might be right forGlycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-Onset)Acid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company
Not applicableLooking for participantsNCT01665326What this trial is testingDetermination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe DiseaseWho this might be right forPompe Disease Duke University 400
Testing effectiveness (Phase 2)Study completedNCT00763932What this trial is testingExtension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) StudiesWho this might be right forPompe Disease Infantile-OnsetGlycogen Storage Disease Type II Genzyme, a Sanofi Company 7
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
Not applicableStudy completedNCT00077662What this trial is testingA Prospective, Observational Study in Patients With Late-Onset Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II Genzyme, a Sanofi Company 61
Post-approval studies (Phase 4)Not Yet RecruitingNCT06575829What this trial is testingTreatment Frequency Reduction in Pompe DiseaseWho this might be right forPompe Disease (Late-onset)GAA DeficiencyGlycogen Storage Disease Type II+1 more Erasmus Medical Center 10
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)
Testing effectiveness (Phase 2)Looking for participantsNCT07282847What this trial is testingEvaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)Who this might be right forPompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD AskBio Inc 12
Early research (Phase 1)Not Yet RecruitingNCT07354724What this trial is testingEvaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe DiseaseWho this might be right forLate-onset Pompe Disease Denali Therapeutics Inc. 32
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
Post-approval studies (Phase 4)Ended earlyNCT01597596What this trial is testingA Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe DiseaseWho this might be right forPompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more Genzyme, a Sanofi Company 4
Post-approval studies (Phase 4)Study completedNCT01288027What this trial is testingExploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase AlfaWho this might be right forPompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency Genzyme, a Sanofi Company 16
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349What this trial is testingA Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)Who this might be right forPompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more Spark Therapeutics, Inc. 4
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
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
Testing effectiveness (Phase 2)Study completedNCT00025896What this trial is testingSafety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe DiseaseWho this might be right forPompe DiseaseGlycogen Storage Disease Type IIAcid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company 8