Pompe Disease Clinical Trials

119 clinical trials for Pompe Disease across international registries, including 18 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) — RECRUITING · PHASE1
• Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease — RECRUITING
• Pompe Disease Registry Protocol — RECRUITING
• Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) — RECRUITING · PHASE2
• Pompe Pregnancy Sub-Registry — RECRUITING
• Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease — RECRUITING
• Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease — RECRUITING · NA
• A Global Prospective Observational Registry of Patients With Pompe Disease — RECRUITING
• Cognitive and Neurological Pathologies in Pompe Disease — RECRUITING
• Phase 1/2 Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults with Late-Onset Pompe Disease — RECRUITING · PHASE2
• Avalglucosidase Alfa Pregnancy Study — RECRUITING
• Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders — RECRUITING
• The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. — RECRUITING · NA
• Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug — RECRUITING · PHASE1
• Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases — RECRUITING · NA
• FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases — RECRUITING
• Multispectral optoacoustic tomography (MSOT) for translational molecular imaging in neuromuscular disease — RECRUITING
• A Long-term Study for the Outcome of Pompe Disease — RECRUITING
• Immune Tolerance Induction Study — OTHER · PHASE4
• A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease — TERMINATED · PHASE3
• A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa. — COMPLETED · PHASE4
• A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease — ACTIVE_NOT_RECRUITING · PHASE2
• Drug-drug interaction study between AT2220 and ERT — COMPLETED · PHASE2
• An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD) — NOT_YET_RECRUITING · PHASE2
• A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease — ACTIVE_NOT_RECRUITING · NA
• Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years — ACTIVE_NOT_RECRUITING · PHASE4
• Gene Transfer Study in Patients With Late Onset Pompe Disease — ACTIVE_NOT_RECRUITING · PHASE1
• AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease — COMPLETED · PHASE1
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) — COMPLETED · PHASE3
• ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD — ACTIVE_NOT_RECRUITING · PHASE3
• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 — COMPLETED · PHASE1
• Open label, single-center pilot study to investigate enzyme replacement therapy (ERT) frequency reduction from 2 to 4 weeks in a subgroup of elderly patients with late-onset Pompe disease — TERMINATED · PHASE4
• Developing a Management Approach for Patients With "Late-Onset" Pompe Disease — ACTIVE_NOT_RECRUITING
• Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease — OTHER
• Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease — TERMINATED · PHASE2
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease — COMPLETED · PHASE3
• Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study. — ACTIVE_NOT_RECRUITING
• GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE — TERMINATED · PHASE4
• Baby Detect : Genomic Newborn Screening — COMPLETED
• Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases — ACTIVE_NOT_RECRUITING
• Drug-drug Interaction Study — COMPLETED · PHASE2
• MSOT in Pompe Disease — COMPLETED · NA
• A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) — ACTIVE_NOT_RECRUITING · PHASE1
• Treatment Frequency Reduction in Pompe Disease — NOT_YET_RECRUITING · PHASE4
• Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease — COMPLETED
• Pompe Lactation Sub-Registry — COMPLETED
• A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting — COMPLETED
• Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) — NOT_YET_RECRUITING · PHASE1
• Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease — COMPLETED · NA
• Nutritional Therapy in Late-onset Pompe Disease — OTHER · PHASE2

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