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

Phase 2 Recruiting 45 enrolled Shionogi

RandomizedParallel-groupQuadruple-blindTreatment

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.

Timeline

Start: 2025-10-30
Primary completion: 2027-08-08
Completion: 2027-08-08

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	S-606001	Unknown	—	Oral

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

Summary

Timeline

Drugs

Indications