Glycogen Storage Disease Type II: all 152 studies

Page 2 of 2. Open to participants first (31), then studies with results posted, then the rest; newest registration first within each.

• Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERTStatus unknownNCT02761421
• Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle WeaknessCompletedNCT02746718Not applicable
• Pompe Disease QMUS and EIMCompletedNCT02742298Not applicable
• An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-Status unknownNCT02708784
• Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.CompletedNCT02654886Not applicable
• Fat and Sugar Metabolism During Exercise in Patients With Metabolic MyopathyStatus unknownNCT02635269Not applicable
• Prevalence of Pompe's Disease in Respiratory ClinicsStatus unknownNCT02527239
• Immune Modulation Therapy for Pompe DiseaseStatus unknownNCT02525172Phase 4
• CPAP for Infantile Pompe DiseaseStatus unknownNCT02405624Phase 4
• Evaluation of Salbutamol as an Adjuvant Therapy for Pompe DiseaseCompletedNCT02405598Phase 4
• Biomarker for Glycogen Storage Diseases (BioGlycogen)WithdrawnNCT02385162
• Diet and Exercise in Pompe DiseaseCompletedNCT02363153Not applicable
• A Pilot Study of Pyridostigmine in Pompe DiseaseTerminatedNCT02357225Early Phase 1
• Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe DiseaseCompletedNCT02240407Phase 1
• A Prospective, Noninterventional, Observational Study of Late-Onset Pompe DiseaseTerminatedNCT02221362
• A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated ReactionTerminatedNCT02185651Phase 1
• Investigating Lysosomal Storage Diseases in Minority GroupsStatus unknownNCT02120235
• A Natural History Study of Adult Onset Pompe Disease Using Muscle MRIStatus unknownNCT01914536
• Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.CompletedNCT01898364Phase 1
• Albuterol in Individuals With Late Onset Pompe Disease (LOPD)CompletedNCT01859624Phase 1
• Newborn Screening Assay of Pompe's DiseaseCompletedNCT01758354Not applicable
• Alglucosidase Alfa Pompe Safety Sub-RegistryCompletedNCT01710813
• The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe DiseaseCompletedNCT01701154
• High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe DiseaseWithdrawnNCT01656590Phase 2
• Pregnancy and Birth Outcome in Women With Pompe DiseaseCompletedNCT01556516
• Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emiaStatus unknownNCT01482494
• Biomarker for Pompe Disease (BioPompe)WithdrawnNCT01457443
• Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation TherapiesCompletedNCT01451879
• Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass SpectrometryStatus unknownNCT01409486
• Pompe Prevalence Study in Patients With Muscle Weakness Without DiagnosisCompletedNCT00830583Not applicable
• Extension 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.CompletedNCT00765414Phase 2
• Extension 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) StudiesCompletedNCT00763932Phase 2
• Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®CompletedNCT00731081
• Muscle Response to Enzyme Replacement Therapy in Pompe DiseaseStatus unknownNCT00713453
• A Long Term Follow up Study in Late-onset Pompe DiseaseStatus unknownNCT00713245
• Pompe Lactation Sub-RegistryCompletedNCT00566878
• Alglucosidase Alfa Temporary Access ProgramApproved for marketingNCT00520143
• A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe DiseaseCompletedNCT00515398
• 3D Echocardiography Managing Infantile Pompe's DiseaseTerminatedNCT00277979
• Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportCompletedNCT00268944Phase 3
• A Study of rhGAA in Patients With Late-Onset Pompe DiseaseCompletedNCT00250939Phase 2
• Assessment of an Alpha-glucosidase Inhibitor to Block Cardiac Events in Patients With MI and IGTTerminatedNCT00212017Not applicable
• Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602CompletedNCT00125879Phase 2/3
• Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe DiseaseCompletedNCT00113035
• A Prospective, Observational Study in Patients With Late-Onset Pompe DiseaseCompletedNCT00077662
• Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe DiseaseCompletedNCT00074932Not applicable
• Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe DiseaseApproved for marketingNCT00074919
• A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe DiseaseCompletedNCT00059280Phase 2/3
• rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)CompletedNCT00053573Phase 1/2
• A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type IICompletedNCT00051935Phase 2
• Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe DiseaseCompletedNCT00025896Phase 2
• Genetic and Family Studies of Inherited Muscle DiseasesCompletedNCT00001331

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