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

Part of paid clinical trials in Irvine, California.

Sponsor
Shionogi
Study ID
NCT07123155
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

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

Interventions

  • S-606001 — DRUG
    S-606001 administered orally
  • Placebo — DRUG
    S-606001 matching placebo administered orally

Study Details

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.

Key Dates

Start date
Oct 30, 2025
Status verified
May 2026
Primary completion
Aug 8, 2027
Completion
Aug 8, 2027

Study Design

Enrollment
45 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: S-606001 Low Dose
    Participants will receive S-606001 at a low dose level twice daily (BID) after a meal for 52 weeks.
  • Experimental: S-606001 High Dose
    Participants will receive S-606001 at a high dose level BID after a meal for 52 weeks.
  • Placebo Comparator: Placebo
    Participants will receive S-606001 matching placebo BID after a meal for 52 weeks.

Primary Outcome Measure

Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52 [ Time Frame: Baseline, Week 52 ]

Central Contacts

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

Locations (9)

FacilityCityStateZIPSite coordinators
University of California - Irvine Medical CenterIrvineCalifornia92868-
University of Florida (UF) - GainesvilleGainesvilleFlorida32611-
Emory University HospitalAtlantaGeorgia30322-
Washington University in St. LouisSt LouisMissouri63130-
Duke University Medical CenterDurhamNorth Carolina27710-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-
University of PennsylvaniaPhiladelphiaPennsylvania19104-
University of Pittsburgh School of MedicinePittsburghPennsylvania15213-
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)FairfaxVirginia22030-

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