Trials / Recruiting
RecruitingNCT07123155
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
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
- Status
- Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 45 (estimated)
- Sponsor
- Shionogi · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | S-606001 | S-606001 administered orally |
| DRUG | Placebo | S-606001 matching placebo administered orally |
Timeline
- Start date
- 2025-10-30
- Primary completion
- 2027-08-08
- Completion
- 2027-08-08
- First posted
- 2025-08-14
- Last updated
- 2026-04-13
Locations
28 sites across 9 countries: United States, Belgium, Denmark, France, Germany, Italy, Netherlands, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07123155. Inclusion in this directory is not an endorsement.