Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Recruiting now · Phase 2 · Has a placebo group
Conditions studied: Pompe Disease
In brief
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.
Key facts
- Study ID
- NCT07123155
- Run by
- Shionogi
- People needed
- 45
- Starts
- 2025-10-30
- Expected to finish
- 2027-08-08
- Last updated by the study team
- 2026-06-23
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California - Irvine Medical Center — Irvine, California, United States (enrolling)
- University of Florida (UF) - Gainesville — Gainesville, Florida, United States (enrolling)
- Emory University Hospital — Atlanta, Georgia, United States (enrolling)
- Washington University in St. Louis — St Louis, Missouri, United States (enrolling)
- Duke University Medical Center — Durham, North Carolina, United States (enrolling)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (enrolling)
- University of Pittsburgh School of Medicine — Pittsburgh, Pennsylvania, United States (enrolling)
- Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) — Fairfax, Virginia, United States (enrolling)
- UZ Leuven — Leuven, Belgium (enrolling)
- Aarhus University Hospital — Aarhus, Denmark (enrolling)
- HLC Hopital Pierre Wertheimer — Bron, France (enrolling)
- AP-HP Hopital Raymond Poincare — Garches, France (enrolling)
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone — Marseille, France (enrolling)
- Salford Royal Hospital — Salford, United Kingdom (enrolling)
- Universitaetsklinikum Halle (Saale) — Halle, Germany (enrolling)
- SphinCS GmbH — Höchheim, Germany (enrolling)
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen — München, Germany (enrolling)
- A.O.U. Policlinico "G. Martino" — Messina, Italy (enrolling)
- AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette — Torino, Italy (enrolling)
- Erasmus MC — GE Rotterdam, GE Rotterdam, Netherlands (enrolling)
- Hospital Universitario 12 de Octubre — Madrid, Spain (enrolling)
- Hospital Universitari i Politecnic La Fe — Valencia, Spain (enrolling)
- Queen Elizabeth Hospital Birmingham — Birmingham, United Kingdom (enrolling)
- National Hospital for Neurology & Neurosurgery — London, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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