ClinicalTrials.gov record NCT07750990

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

A Phase 2, Open-label, Multicenter, Extension Study to Investigate the Long-term Safety and Efficacy of S-606001 in Patients With Late-onset Pompe Disease

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age18 years old and older
SexAll
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2, open-label extension study evaluates long-term safety and tolerability of oral S-606001 in people with late-onset Pompe Disease who previously took S-606001. Participants who were using enzyme replacement therapy (ERT) at enrollment will continue their same ERT regimen alongside S-606001. Safety data are collected from baseline through Week 96.

What it is studying. The study is testing the long-term safety and tolerability of oral S-606001 in people with late-onset Pompe Disease. It also collects measures from baseline to Week 96 of lung function (% forced vital capacity), walking ability (6-minute walk test), muscle function (GSGC score), daily living activities, and multiple patient-reported outcomes for fatigue, physical function, breathlessness, pain, overall quality of life (SF-36), and patient global impression of severity.

What the researchers hope to learn. To evaluate the long-term safety and tolerability profile of S-606001 and to assess changes from baseline to Week 96 in lung function (%FVC), walking distance (6MWT), GSGC score, daily living activity levels, several patient-reported outcome measures (fatigue, physical function, dyspnea, pain), SF-36 quality of life, and patient global impression of severity. The primary outcome is the number of participants with treatment-emergent adverse events from baseline through Week 96.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The primary purpose of this study is to evaluate the long-term safety and tolerability profile of S-606001 in participants with LOPD. Participants who completed other S-606001 studies (for example, NCT07123155), including participants in a S-606001 study whose allocated arm was terminated by the sponsor and who are willing to participate in this study, will be enrolled globally.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • S-606001
  • ERT

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Participant must be ≥40 kilograms of body weight at the time of signing the informed consent
  • Participants who previously completed a S-606001 Pompe treatment study, including participants whose assigned treatment arm was terminated by the sponsor
  • Key

Exclusion criteria

  • Has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements
  • Current enrollment in any other clinical study involving an investigational intervention or any other type of medical research
  • Participant, if female, is pregnant or breastfeeding at visit 1
  • 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 description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will take the study drug S-606001 orally; those already on enzyme replacement therapy (ERT) will continue their usual intravenous ERT infusions. They will have a baseline visit and follow-up assessments through Week 96 while researchers monitor and record any treatment-emergent adverse events. Study assessments include lung function testing (percent forced vital capacity), a 6-minute walk test, physical function tests (Gait, Stair, Gower's Maneuver, and Chair [GSGC] score), investigator-rated daily-activity levels, and patient questionnaires (PROMIS measures, SF-36, and PGI‑S). Exact visit timing and any additional procedures are not detailed in the registry record.

How the study is structured. Open-label, single-group Phase 2 extension study. All enrolled participants receive oral S-606001. Participants already on enzyme replacement therapy will continue their usual intravenous ERT regimen while taking S-606001. Key outcomes and assessments are recorded at baseline and through Week 96; there is no masking.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • No sites are listed on the registry yet. Where will the study take place?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Pompe disease

Clinical trials32 trials listed for Pompe diseaseEvery trial the registry lists for Pompe disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Pompe disease researchLatest monthly update: September 2026. Includes: Antibody effects. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Aug 31, 2026 (estimated)
Primary completion
Aug 31, 2028 (estimated)
Study completion
Aug 31, 2028 (estimated)
Target enrollment
60 participants (estimated)
Last updated on registry
Aug 6, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Shionogi

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

primary purpose
The main reason an interventional study is being done, for example treatment, prevention, diagnosis, supportive care or basic science.
sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
treatment arm
One of the groups in a study. Each arm receives a particular intervention, comparator or no intervention, so the groups can be compared.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Key Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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