Recruiting
Phase 2

S-606001 with ERT

Sponsor:

Shionogi

Code:

NCT07123155

Conditions

Pompe Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

S-606001

Placebo

Study Details

Brief 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.

Conditions

Pompe Disease

Study ID

NCT07123155

Start date

Oct 30, 2025

Status verified date

Jun, 2026

Completion date

Aug 8, 2027

Anticipated

Primary completion date

Aug 8, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Key Inclusion Criteria:

  • 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:

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.
  • 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.

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

Study Design

Enrollment

45 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

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.

Interventions

S-606001

S-606001 administered orally

Placebo

S-606001 matching placebo administered orally

Primary outcome measure

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

Central Contacts and Locations

Central contacts

Shionogi Clinical Trials Administrator Clinical Support Help Line

800-849-9707Shionogiclintrials-admin@shionogi.co.jp

Locations

University of California - Irvine Medical Center

Recruiting

Irvine, California, United States, 92868

University of Florida (UF) - Gainesville

Recruiting

Gainesville, Florida, United States, 32611

Emory University Hospital

Recruiting

Atlanta, Georgia, United States, 30322

Washington University in St. Louis

Recruiting

St Louis, Missouri, United States, 63130

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

University of Pennsylvania

Recruiting

Philadelphia, Pennsylvania, United States, 19104

University of Pittsburgh School of Medicine

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

Recruiting

Fairfax, Virginia, United States, 22030

More Information

Sponsor

Shionogi

Last update posted

Jun 23, 2026

Last verified

Jun, 2026

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Shionogi on 2026-06-23.