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Study of S-606001 for Late-onset Pompe Disease

This study is testing S-606001, an oral medication, as an add-on treatment to enzyme replacement therapy (ERT) for adults with Late-onset Pompe Disease (LOPD). Researchers want to learn about the safety of S-606001, how it affects the body (pharmacodynamics), and if it helps improve lung function. You may be eligible if you are at least 18 years old, weigh at least 40 kg, and have a confirmed diagnosis of LOPD. The main way they will measure success is by looking at changes in your forced vital capacity (%FVC), which measures how much air you can exhale, after 52 weeks. The current recruitment status is unclear.

Study design
This interventional study plans to enroll 45 participants. Some participants will receive S-606001, while others will receive a matching placebo.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured at Week 52, suggesting follow-up for at least this duration.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07123155

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

Recruiting
PHASE2Ages 18+InterventionalTreatment
Shionogi
~45 participants
Updated 2026-06-23 on ClinicalTrials.gov
What's tested:S-606001Placebo

At a glance

Recruiting sites
27 of 28 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52
Measured over Baseline, Week 52

Conditions

Pompe Disease

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NCT07123155

Where you'd take part

This study runs at 28 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • A.O.U. Policlinico "G. Martino"

    Messina, Italyno site contact published

    Recruiting

  • Aarhus University Hospital

    Aarhus, Denmarkno site contact published

    Recruiting

  • AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette

    Torino, Italyno site contact published

    Recruiting

  • AP-HP Hopital Raymond Poincare

    Garches, Franceno site contact published

    Recruiting

  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

    Marseille, Franceno site contact published

    Recruiting

  • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires

    Nice, Franceno site contact published

    Not yet recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohiono site contact published

    Recruiting

  • Duke University Medical Center

    Durham, North Carolinano site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Shionogi Clinical Trials Administrator Clinical Support Help Line
[email protected]
800-849-9707
Email the study team

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Eligibility criteria

Inclusion

Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.
Participant must have a diagnosis of LOPD based on documentation of 1 of the following:
Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position.
Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:
Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months.

Exclusion

Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements.
Has active infections at screening.
Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
Current or chronic history of liver disease.
Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .
Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study.
Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.
Participant, if female, is pregnant or breastfeeding at screening.
Participant, whether male or female, is planning to conceive a child during the study.

What this trial measures

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