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RecruitingNCT07123155Updated Jun 23, 2026

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

A Phase 2 interventional study of S-606001 and Placebo in Pompe Disease, sponsored by Shionogi. Recruiting at 28 sites in 9 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-06-23.

Sponsored by Shionogi · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Oct 2025; still recruiting 1 year later.
Phase
Phase 2
Study type
Interventional
Enrollment
45
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

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.

02

Conditions studied

  • Pompe Disease

Keywords

  • Late-onset Pompe disease
  • Enzyme replacement therapy
  • LOPD
  • ERT
  • S-606001
  • Muscle glycogen synthase
  • Liver glycogen synthase
  • Rare disease
  • Autosomal disease
  • Acid alpha-glucosidase
  • GAA
  • Glycogen storage disorder
  • GSD
03

In context

Glycogen Storage Disease Type II

162 studies on the registry are indexed under Glycogen Storage Disease Type II; 34 are open to participants now.

This study's planned enrollment of 45 is above the median of 20 across 89 interventional studies indexed under Glycogen Storage Disease Type II.

Browse Glycogen Storage Disease Type II studies →

Lead sponsor

Shionogi is the lead sponsor of 105 studies on the registry; 10 are open to participants now.

Of its 39 completed or terminated interventional studies of FDA-regulated products, 21 (54%) have results posted.

Browse Shionogi studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the .

  • Participant must have a diagnosis of LOPD based on documentation of 1 of the following:

    1. Deficiency of acid alpha-glucosidase (GAA) enzyme
    2. 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:

    1. Screening values of 6-minute walk distance (6MWD) are ≥75 meters
    2. 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.

Key Exclusion Criteria:

  • 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.
  • within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of 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 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.

Note: Other protocol-specified inclusion and exclusion criteria may apply.

Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
45 participants (estimated)

Study 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.

    Drug: S-606001

  • Experimental
    S-606001 High Dose

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

    Drug: S-606001

  • Placebo comparator
    Placebo

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

    Drug: Placebo

Interventions

  • DrugS-606001

    S-606001 administered orally

  • DrugPlacebo

    S-606001 matching placebo administered orally

06

What researchers measure

Primary outcomes

  1. Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52

    Time frame: Baseline, Week 52

Secondary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: Baseline up to Week 53

  2. Plasma Concentration of S-606001

    Time frame: Up to Week 12

  3. Change From Baseline in Serum Creatine Kinase Levels at Week 52

    Time frame: Baseline, Week 52

  4. Change From Baseline in 6-minute Walk Test (6MWT) at Week 52

    Time frame: Baseline, Week 52

  5. Change From Baseline in Pulmonary Function Parameter: Maximal Inspiratory Pressure (MIP) at Week 52

    Time frame: Baseline, Week 52

  6. Change From Baseline in Pulmonary Function Parameter: Maximal Expiratory Pressure (MEP) at Week 52

    Time frame: Baseline, Week 52

  7. Change From Baseline in Motor Function Parameter: Gait, Stair, Gower's Maneuver, Chair (GSGC) Score at Week 52

    Time frame: Baseline, Week 52

  8. Change From Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS-Fatigue-8a) Score at Week 52

    Time frame: Baseline, Week 52

  9. Change From Baseline in Patient-Reported Outcomes Measurement Information System Physical Function 20-item short form (PROMIS-PF-20) Score at Week 52

    Time frame: Baseline, Week 52

  10. Change From Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 PAIN) Score at Week 52

    Time frame: Baseline, Week 52

  11. Change From Baseline in 36-item Short Form Health Survey (SF-36) Score at Week 52

    Time frame: Baseline, Week 52

  12. Change From Baseline in Patient Global Impression of Severity (PGI-S) Score at Week 52

    Time frame: Baseline, Week 52

07

Study locations

27 of 28 sites recruiting
08

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Oct 2025 (actual). Primary completion is estimated for Aug 2027, 21 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Jul 25, 2025
First posted
Aug 14, 2025
StartActual
Oct 30, 2025
Primary completionEst.
Aug 8, 2027
CompletionEst.
Aug 8, 2027
Last update
Jun 23, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jun 23, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT07123155
Lead sponsor
Shionogi
Responsible party
Sponsor
First posted
Aug 14, 2025
Start date
Oct 30, 2025
Primary completion
Aug 8, 2027 (estimated)
Completion
Aug 8, 2027 (estimated)
Last update
Jun 23, 2026

Study contacts

Shionogi Clinical Trials Administrator Clinical Support Help Line
Contact
Shionogiclintrials-admin@shionogi.co.jp
800-849-9707

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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