Glycogen Storage Disease Type II Clinical Trials

47 clinical trials for Glycogen Storage Disease Type II across international registries, including 3 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Pompe Disease Registry Protocol — RECRUITING
• Pompe Pregnancy Sub-Registry — RECRUITING
• China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD — RECRUITING · PHASE4
• Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) — COMPLETED · PHASE2
• A French multicenter Phase 4 open label extension study of long-term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France — TERMINATED · PHASE4
• A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) — ACTIVE_NOT_RECRUITING
• Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa — ACTIVE_NOT_RECRUITING · PHASE3
• Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) — ACTIVE_NOT_RECRUITING · PHASE4
• An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD) — OTHER · PHASE3
• A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) — ACTIVE_NOT_RECRUITING · PHASE1
• Treatment Frequency Reduction in Pompe Disease — NOT_YET_RECRUITING · PHASE4
• Nutritional Therapy in Late-onset Pompe Disease — OTHER · PHASE2
• Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease — OTHER · NA
• Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients. — COMPLETED · PHASE1
• Biomarker for Glycogen Storage Diseases (BioGlycogen) — WITHDRAWN
• Growth and Development Study of Alglucosidase Alfa — COMPLETED · PHASE4
• Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment — COMPLETED · PHASE4
• Immune Tolerance Induction Study — TERMINATED · PHASE4
• Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease — COMPLETED · PHASE4
• Higher Dose of Alglucosidase Alpha for Pompe Disease — NOT_YET_RECRUITING
• Identification of Tongue Involvement in Late-Onset Pompe Disease — COMPLETED
• Diet and Exercise in Pompe Disease — COMPLETED · NA
• Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy — OTHER · NA
• Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) — COMPLETED · NA
• Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT — OTHER
• A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease — TERMINATED · PHASE4
• A Prospective, Observational Study in Patients With Late-Onset Pompe Disease — COMPLETED
• A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease — COMPLETED · PHASE3
• High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease — WITHDRAWN · PHASE2
• Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa — COMPLETED · PHASE4
• Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease — COMPLETED · PHASE2
• An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease — COMPLETED · PHASE4
• Late-Onset Treatment Study Extension Protocol — COMPLETED · PHASE4
• High Dose or High Dose Frequency Study of Alglucosidase Alfa — COMPLETED · PHASE4
• Alglucosidase Alfa Temporary Access Program — OTHER
• Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme® — COMPLETED
• A Study of rhGAA in Patients With Late-Onset Pompe Disease — COMPLETED · PHASE2
• Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease — COMPLETED · NA
• Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 — COMPLETED · PHASE2
• rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) — COMPLETED · PHASE1
• A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease — COMPLETED · PHASE2
• A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II — COMPLETED · PHASE2
• Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease — OTHER
• Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support — COMPLETED · PHASE3
• 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) Studies — COMPLETED · PHASE2
• A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease — COMPLETED
• Genetic and Family Studies of Inherited Muscle Diseases — COMPLETED

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