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Taking people in FrancePhase 2

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.

Key facts

Who runs it
Shionogi
Where
Bron, Garches, Marseille, and Nice, FranceSee the 4 sitesAlso in 8 other countries, 24 sites
Registry title
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Status
Recruiting
Phase
Phase 2
People taking part
45 people, planned
Started
30 Oct 2025
Expected to end
8 Aug 2027
Type of study
Interventional, 3 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • S-606001
  • Placebo
Record updated
23 June 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with late-onset Pompe disease who already take enzyme replacement therapy (ERT). They would receive S-606001 or a placebo on top of ERT.

Age
18 years or older, and at least 40 kg (88 lb) body weight.
Sex
All sexes can join.
Disease stage
Late-onset Pompe disease, shown by a lack of the GAA enzyme or by the GAA genotype. Breathing test (%FVC) must be between 30% and 80% sitting upright, or the %FVC must drop 10% or more when lying down and be at least 20% lying down. Must be able to do the 6-minute walk test, walking at least 75 meters and at most 90% of the predicted distance for healthy adults.
Earlier treatment
Must be taking ERT for at least 24 months, with no change in the ERT regimen in the last 6 months. Cannot have had gene therapy or small interfering RNA therapy for Pompe disease.
Biomarker or test result
Lack of GAA enzyme or a GAA genotype that confirms Pompe disease.

Main reasons for not taking part

  • Active infection at screening
  • Cancer in the past 5 years (skin cancers removed with no spread for 3 years are allowed)
  • Current or long-term liver disease
  • Known loss-of-function changes in both copies of the GYG gene or the PYGM gene
  • Pregnant or breastfeeding, or planning to conceive during the study

The registry criteria, word for word

Show all 14 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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

Where in France

Also in 8 other countries, 24 sites

  • HLC Hopital Pierre WertheimerBron, FranceContact: Shionogi Clinical Trials Administrator Clinical Support Help LineTaking people
  • AP-HP - hôpital Raymond PoincaréGarches, FranceContact: Shionogi Clinical Trials Administrator Clinical Support Help LineTaking people
  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La TimoneMarseille, FranceContact: Shionogi Clinical Trials Administrator Clinical Support Help LineTaking people
  • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies NeuromusculairesNice, FranceContact: Shionogi Clinical Trials Administrator Clinical Support Help LineNot yet recruiting

About the trial

What the trial wants to find out

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.

The 3 groups in the trial

  • S-606001 Low DoseTreatment being tested

    Participants will receive S-606001 at a low dose level twice daily (BID) after a meal for 52 weeks.

    Treatment: S-606001

  • S-606001 High DoseTreatment being tested

    Participants will receive S-606001 at a high dose level BID after a meal for 52 weeks.

    Treatment: S-606001

  • PlaceboPlacebo, for comparison

    Participants will receive S-606001 matching placebo BID after a meal for 52 weeks.

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
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
Plain-language title
Tests S-606001 added to enzyme replacement therapy in late-onset Pompe diseaseGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
23 June 2026