NCT07750990Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
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
Source: ClinicalTrials.gov · Last updated 13 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 60 (estimated)
- First submitted
- 30 Jul 2026
- Start date
- 2 Nov 2026
- Primary completion date
- 31 Aug 2028
- Lead sponsor
- Shionogi
- Diseases
- Pompe disease
- NCT number
- NCT07750990
- EudraCT number
- 2026-525915-15
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 60
- From 2 Nov 2026 to the primary completion date, 31 Aug 2028
- No sites listed in no country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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
Other
- 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.
Who the study is looking for
- 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
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
This page is information, not medical advice. Discuss any trial with your doctor.