RecruitingPhase 2

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

United States45 participantsStarted 2025-10-30

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Deficiency of acid alpha-glucosidase (GAA) enzyme
✓. GAA genotype
✓. Screening values of 6-minute walk distance (6MWD) are ≥75 meters
✓. Screening values of 6MWD are ≤90% of the predicted value for healthy adults

What they're measuring

Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52

Timeframe: Baseline, Week 52

Trial details

NCT IDNCT07123155

SponsorShionogi

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-08-08

Contact for this trial

Shionogi Clinical Trials Administrator Clinical Support Help Line

800-849-9707 Shionogiclintrials-admin@shionogi.co.jp

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