Study of S-606001 as an Add-on to Enzyme Replacement Therapy in Adults With Late-onset Pompe Disease

What this Trial Is About

Researchers are evaluating the safety, pharmacodynamics, and exploratory clinical efficacy of the drug S-606001 when added to enzyme replacement therapy (ERT) in adults with late-onset Pompe disease (LOPD). This Phase 2 trial involves adult participants already receiving ERT to better understand how S-606001 affects their condition over a year-long period. Participants will be randomly assigned to one of three groups receiving either a low dose or high dose of S-606001, or a matching placebo. All doses are taken orally twice daily after meals for 52 weeks. The study is double-blind, meaning neither participants nor researchers know which treatment is given, to ensure unbiased results. During the study, participants will be monitored through various assessments including lung function tests like forced vital capacity (%FVC), a 6-minute walk test (6MWT), muscle strength evaluations, and patient-reported outcome questionnaires. Safety will be tracked by noting any adverse events. The main goal is to measure changes in lung function after 52 weeks of treatment, with follow-up assessments for up to 53 weeks. Participants are expected to engage in scheduled visits and tests throughout this period.

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