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NCT07123155 · ClinicalTrials.gov registry record · Phase 2
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
A Phase 2 study of Pompe Disease, sponsored by Shionogi.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 45
- Enrollment target
- 20
- Study locations
NCT07123155: Recruiting Phase 2 study of Pompe Disease, sponsored by Shionogi.
NCT07123155 is a Phase 2 study of Pompe Disease that is actively recruiting participants, run by Shionogi. The registered enrollment target is 45 participants, below the 229-participant average among 14 other Pompe Disease trials with a reported enrollment target (80% lower). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07123155, a Phase 2 study of Pompe Disease, is actively recruiting participants, sponsored by Shionogi.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 45 participants
- Enrollment target
- 20
- Study locations
Study 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 Studied
Interventions
- DRUG Placebo
- DRUG S-606001
Study Locations (20)
Other
- UZ Leuven - Leuven
- Aarhus University Hospital - Aarhus
- HCL Hopital Pierre Wertheimer - Bron
- AP-HP Hopital Raymond Poincare - Garches
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone - Marseille
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires - Nice
- Universitaetsklinikum Halle (Saale) - Halle
- SphinCS GmbH - Höchheim
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen - München
- A.O.U. Policlinico "G. Martino" - Messina
- AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette - Torino
Pennsylvania
- University of Pennsylvania - Philadelphia
- University of Pittsburgh School of Medicine - Pittsburgh
California
- University of California - Irvine Medical Center - Irvine
Florida
- University of Florida (UF) - Gainesville - Gainesville
Georgia
- Emory University Hospital - Atlanta
Missouri
- Washington University in St. Louis - St Louis
North Carolina
- Duke University Medical Center - Durham
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2025-10-30 |
| Est. Completion | 2027-08-08 |
| Phase | Phase 2 |
What NCT07123155 shows while recruiting
NCT07123155 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 45 participants, a relatively small participant target, below the 229-participant average among 14 other Pompe Disease trials with a reported enrollment target (80% lower).
The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT07123155 names 20 study sites across 9 states, led by Other, Pennsylvania, California.
Frequently Asked Questions
What is clinical trial NCT07123155 about?
NCT07123155 is a clinical study titled "Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)". 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.
What is the current status of trial NCT07123155?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 45 participants. The study started on 2025-10-30. Estimated completion is 2027-08-08.
What conditions does trial NCT07123155 study?
This clinical trial studies the following conditions: Pompe Disease.
What interventions are being tested in trial NCT07123155?
The interventions under investigation include: Placebo (DRUG), S-606001 (DRUG).
Who is sponsoring clinical trial NCT07123155?
This trial is sponsored by Shionogi, which has 54 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07123155 being conducted?
This trial has 20 study locations across California, Florida, Georgia, Missouri, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Pompe Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT07123155's enrollment target sits among peer trials
45 6th of 14 higher than 8 of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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