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Condition: Pompe Disease Synced from ClinicalTrials.gov · July 28, 2026

Recruiting studies: Pompe Disease

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Phase 2 · 28 location(s) · Shionogi · NCT07123155Registry of Patients Diagnosed With Lysosomal Storage Diseases
Phase N/A · 1 location(s) · University of California, San Francisco · NCT05619900Cognitive and Neurological Pathologies in Pompe Disease
Phase N/A · 1 location(s) · Duke University · NCT04639336FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Phase N/A · 1 location(s) · xCures · NCT06539169Pompe Disease Registry Protocol
Phase N/A · 272 location(s) · Genzyme, a Sanofi Company · NCT00231400China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Phase 4 · 1 location(s) · Genzyme, a Sanofi Company · NCT06666413Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Phase N/A · 1 location(s) · Duke University · NCT01665326Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
NA · 1 location(s) · Assistance Publique Hopitaux De Marseille · NCT06833489A Long-term Study for the Outcome of Pompe Disease
Phase N/A · 1 location(s) · National Taiwan University Hospital · NCT02399748Long-Term Development of Muscular Dystrophy Outcome Assessments
Phase N/A · 1 location(s) · Virginia Commonwealth University · NCT05989620A Global Prospective Observational Registry of Patients With Pompe Disease
Phase N/A · 41 location(s) · Amicus Therapeutics · NCT06121011Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Phase 1/Phase 2 · 1 location(s) · GeneCradle Inc · NCT06391736PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Phase 1 · 1 location(s) · University of California, San Francisco · NCT04532047A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects A
Phase 3 · 14 location(s) · Amicus Therapeutics · NCT04808505A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Lat
Phase 1/Phase 2 · 9 location(s) · AskBio Inc · NCT07282847Pompe Pregnancy Sub-Registry
Phase N/A · 35 location(s) · Genzyme, a Sanofi Company · NCT00567073Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease
Phase N/A · 1 location(s) · IRCCS National Neurological Institute "C. Mondino" Foundatio · NCT07664930Comparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment Intervals
Phase 4 · 1 location(s) · Faculty Hospital Kralovske Vinohrady · NCT06875245Avalglucosidase Alfa Pregnancy Study
Phase N/A · 1 location(s) · Sanofi · NCT05734521A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
NA · 1 location(s) · Oculox Technologies SA · NCT06557369Natural History of Pompe Disease
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT03564561A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset P
Phase 1 · 3 location(s) · Denali Therapeutics Inc. · NCT07354724Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
NA · 1 location(s) · Centre Hospitalier Universitaire de Liege · NCT07136844Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
NA · 1 location(s) · University of Missouri-Columbia · NCT07478172
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.