Phase 2 Study of Oral S-606001 Added to Enzyme Replacement Therapy for Adults with Late-Onset Pompe Disease

What this Trial Is About

Researchers are studying the safety, pharmacodynamics, and initial clinical effects of S-606001 in adults with late-onset Pompe disease LOPD. This study focuses on adding S-606001 to the existing enzyme replacement therapy ERT to evaluate its impact over time. Participants are randomly assigned to receive either a low dose or high dose of S-606001, or a matching placebo, taken orally twice daily after meals for 52 weeks. The study is double-blind, meaning neither participants nor researchers know who receives which dose or placebo during the trial. Throughout the 52 weeks, participants will be evaluated using lung function tests, 6-minute walk tests, muscle strength assessments, and patient-reported questionnaires on fatigue, pain, and physical function. Safety monitoring includes tracking adverse events and blood tests. Changes from baseline in forced vital capacity %FVC at week 52 is the primary outcome measured.

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