Tests S-606001 added to enzyme replacement therapy in late-onset Pompe disease
Tests S-606001 alongside enzyme replacement therapy in adults with late-onset Pompe disease.
Plain-language title generated from the registry record
Registry title: Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Key facts
- Where
- Halle, Höchheim, and München, GermanySee the 3 sites
- Phase
- Phase 2
Where in Germany
- Universitaetsklinikum Halle - SaaleHalle, GermanyTaking people
- SphinCS GmbHHöchheim, GermanyTaking people
- Klinikum der Ludwig-Maximilians-Universitaet MuenchenMünchen, GermanyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Shionogi
- Trial code
- NCT07123155
- Record updated
- 23 June 2026
- Original description
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.Read full original descriptionShow less
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
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.
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:; Deficiency of acid alpha-glucosidase (GAA) enzyme; GAA genotype; 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:; Screening values of 6-minute walk distance (6MWD) are ≥75 meters; Screening values of 6MWD are ≤90% of the predicted value for healthy adults; 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 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; 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) ; Participant, if female, is pregnant or breastfeeding at screening; Participant, whether male or female, is planning to conceive a child during the study; Note: Other protocol-specified inclusion and exclusion criteria may apply.