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 completedNCT00113035What this trial is testingScreening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe DiseaseWho this might be right forPompe Disease Genzyme, a Sanofi Company 60
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
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
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
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
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
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
Testing effectiveness (Phase 2)Study completedNCT00053573What this trial is testingRhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)Who this might be right forGlycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company 20
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
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
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
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 applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
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
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)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
Post-approval studies (Phase 4)Study completedNCT00483379What this trial is testingHigh Dose or High Dose Frequency Study of Alglucosidase AlfaWho this might be right forPompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency Genzyme, a Sanofi Company 13
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
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