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

Sponsor
Shionogi
Study ID
NCT07750990
Phase
PHASE2
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • S-606001 — DRUG
    S-606001 will be administered orally.
  • ERT — DRUG
    ERT will be administered as an intravenous infusion.

Study Details

The primary purpose of this study is to evaluate the long-term safety and tolerability profile of S-606001 in participants with LOPD. Participants who completed other S-606001 studies (for example, NCT07123155), including participants in a S-606001 study whose allocated arm was terminated by the sponsor and who are willing to participate in this study, will be enrolled globally.

Key Dates

First listed
Aug 6, 2026
Start date
Aug 31, 2026
Status verified
Aug 2026
Primary completion
Aug 31, 2028
Completion
Aug 31, 2028

Study Design

Enrollment
60 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: S-606001
    Participants will receive S-606001. Participants who were previously using enzyme replacement therapy (ERT) at the time of enrollment will receive the same regimen of ERT as standard of care.

Primary Outcome Measure

Number of Participants With Treatment-emergent Adverse Events [ Time Frame: Baseline through Week 96 ]

Central Contacts

  • Shionogi Clinical Trials Administrator Clinical Support Help Line
    1-800-849-9707
    [email protected]

Related Studies