S-606001

DRUG4 trials

Sponsors

Shionogi B.V., Shionogi

Conditions

Late-onset Pompe disease.Pompe Disease

Phase 1

A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)

Not yet recruitingNCT07787650

ShionogiPompe Disease

Start: 2026-09-30End: 2028-10-31Target: 15Updated: 2026-08-26

Phase 2

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

RecruitingNCT07123155

ShionogiPompe Disease

Start: 2025-10-30End: 2027-08-08Target: 45Updated: 2026-06-23

A phase 2, multicenter, randomized, placebo-controlled, double-blind study to investigate the safety, pharmacodynamics, and preliminary efficacy of S-606001 as an add-on to enzyme replacement therapy in patients with late-onset Pompe disease.

Not yet recruitingCTIS2025-522146-40-00

Shionogi B.V.Late-onset Pompe disease.

Target: 19Updated: 2025-12-19

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Not yet recruitingNCT07750990

ShionogiPompe Disease

Start: 2026-08-31End: 2028-08-31Target: 60Updated: 2026-08-06