Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) (NCT07123155)
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
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of California - Irvine Medical Center — Irvine, California
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (28 Sites)
Showing the first 25 of 28 sites.
- University of California - Irvine Medical Center — Irvine, California
- University of Florida (UF) - Gainesville — Gainesville, Florida
- Emory University Hospital — Atlanta, Georgia
- Washington University in St. Louis — St Louis, Missouri
- Duke University Medical Center — Durham, North Carolina
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- University of Pennsylvania — Philadelphia, Pennsylvania
- University of Pittsburgh School of Medicine — Pittsburgh, Pennsylvania
- Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) — Fairfax, Virginia
- UZ Leuven — Leuven,
- Aarhus University Hospital — Aarhus,
- HLC 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,
- Erasmus MC — GE Rotterdam, GE Rotterdam
- Hospital Universitario 12 de Octubre — Madrid,
- Hospital Universitari i Politecnic La Fe — Valencia,
- Queen Elizabeth Hospital Birmingham — Birmingham,
- National Hospital for Neurology & Neurosurgery — London,
📞 Contact the Study Coordinator
Phone: (800) 849-9707
Email: Shionogiclintrials-admin@shionogi.co.jp
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Pompe Disease
- Developing a Management Approach for Patients With "Late-Onset" Pompe Disease — Durham, North Carolina
- A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) — Phoenix, Arizona
- A Global Prospective Observational Registry of Patients With Pompe Disease — Little Rock, Arkansas
- Cognitive and Neurological Pathologies in Pompe Disease — Durham, North Carolina
- Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease — Durham, North Carolina
Explore by condition: All Pompe Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.