Pompe DiseasePhase 2NCT07750990
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
A Phase 2 study testing S-606001 and ERT in people with Pompe Disease, run by Shionogi.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease
- What is being tested?
- S-606001 and ERT
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 2 Nov 2026to 31 Aug 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Shionogi Clinical Trials Administrator Clinical Support Help Line contact
About this study
Official title: 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
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.
- Conditions
- Pompe Disease
- Being tested
- S-606001, ERT
- Keywords
- S-606001, Late-onset Pompe Disease, LOPD, ERT, Enzyme Replacement Therapy, NCT07123155, Muscle glycogen synthase, Liver glycogen synthase, Rare disease, Autosomal disease, Acid alpha-glucosidase, GAA, Glycogen storage disorder, GSD
- Medical subject headings
- Glycogen Storage Disease Type II, Rare Diseases, Glycogen Storage Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
S-606001Drug
S-606001 will be administered orally.
ERTDrug
ERT will be administered as an intravenous infusion.
| Study group | Role | What they receive |
|---|---|---|
| S-606001 | Experimental | Participants will receive S-606001. Participants who were previously using enzyme replacement therapy (ERT) at the time of enrollment will receive the same regimen of ERT as standard of care. Receives: Drug: S-606001; Drug: ERT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-emergent Adverse Events
Measured: Baseline through Week 96
Secondary outcomes (10)
Change from Baseline in Percent Forced Vital Capacity (%FVC)
Measured: Baseline, Week 96
Change from Baseline in 6-minute Walk Test (6MWT)
Measured: Baseline, Week 96
Change from Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score
Measured: Baseline, Week 96
Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator
Measured: Baseline, Week 96
Change from Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS Fatigue-8a)
Measured: Baseline, Week 96
Change from Baseline in Patient-Reported Outcomes Measurement Information System Item Bank v2.0 - Physical Function - Short Form 20a (PROMIS-Item Bank-PF-20a)
Measured: Baseline, Week 96
Change from Baseline in Patient-Reported Outcomes Measurement Information System Dyspnea Characteristics (PROMIS-Dyspnea)
Measured: Baseline, Week 96
Change from Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 Pain)
Measured: Baseline, Week 96
Change from Baseline in 36-item Short Form Health Survey (SF-36)
Measured: Baseline, Week 96
Change from Baseline in Patient Global Impression of Severity (PGI-S)
Measured: Baseline, Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2026
- Study start2 Nov 2026 (estimated)
- Primary completion31 Aug 2028 (estimated)
- Study completion31 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe DiseaseStudies of the same condition, open and under way.
- More trials run by ShionogiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shionogi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Shionogi Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07750990
- Sponsor's protocol ID
- 2507N1223
- Other IDs
- 2026-525915-15
- Status last verified
- Oct 2026
- First submitted
- 30 Jul 2026
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No