Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Public ClinicalTrials.gov record NCT07123155. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease
Study identification
- NCT ID
- NCT07123155
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Shionogi
- Industry
- Enrollment
- 45 participants
Conditions and interventions
Conditions
Interventions
- Placebo Drug
- S-606001 Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 29, 2025
- Primary completion
- Aug 7, 2027
- Completion
- Aug 7, 2027
- Last update posted
- Apr 29, 2026
2025 – 2027
United States locations
- U.S. sites
- 9
- U.S. states
- 8
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California - Irvine Medical Center | Irvine | California | 92868 | Recruiting |
| University of Florida (UF) - Gainesville | Gainesville | Florida | 32611 | Recruiting |
| Emory University Hospital | Atlanta | Georgia | 30322 | Not yet recruiting |
| Washington University in St. Louis | St Louis | Missouri | 63130 | Recruiting |
| Duke University Medical Center | Durham | North Carolina | 27710 | Recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | Recruiting |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | Recruiting |
| University of Pittsburgh School of Medicine | Pittsburgh | Pennsylvania | 15213 | Recruiting |
| Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) | Fairfax | Virginia | 22030 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 19 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07123155, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 29, 2026 · Synced May 5, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07123155 live on ClinicalTrials.gov.