An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD) (NCT07750990) | Clinical Trial Compass
Not Yet RecruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
60 participantsStarted 2026-08-31
Plain-language summary
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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment-emergent Adverse Events
Timeframe: Baseline through Week 96
Trial details
NCT IDNCT07750990
SponsorShionogi
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2028-08-31
Contact for this trial
Shionogi Clinical Trials Administrator Clinical Support Help Line