Clinical Trial

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Recruiting Phase 2
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Summary
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
Protocol Amendment History 16 amendments
This ClinicalTrials.gov record has been amended 16 times since 2025-08-08; most recent amendment 2026-06-19.
Status change: Not Yet Recruiting → Recruiting 2025-11-21
Trial Details
NCT Number NCT07123155
Lead Sponsor Shionogi
Conditions Pompe Disease
Enrollment 45 participants
Start Date 2025-10-30
Primary Completion 2027-08-08 (estimated)
Study Completion 2027-08-08 (estimated)
Updated on ClinicalTrials.gov 2026-06-23