Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Start Date
10/30/2025
Completion Date
8/8/2027
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.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
S-606001
Placebo
Conditions
Locations
University of California - Irvine Medical Center
Irvine, California 92868
United States
University of Florida (UF) - Gainesville
Gainesville, Florida 32611
United States
Emory University Hospital
Atlanta, Georgia 30322
United States
Washington University in St. Louis
St Louis, Missouri 63130
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
University of Pennsylvania
Philadelphia, Pennsylvania 19104
United States
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania 15213
United States
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia 22030
United States
UZ Leuven
Leuven,
Belgium
Aarhus University Hospital
Aarhus,
Denmark
HLC Hopital Pierre Wertheimer
Bron,
France
AP-HP Hopital Raymond Poincare
Garches,
France
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
Marseille,
France
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
Nice,
France
Universitaetsklinikum Halle (Saale)
Halle,
Germany
SphinCS GmbH
Höchheim,
Germany
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München,
Germany
A.O.U. Policlinico "G. Martino"
Messina,
Italy
AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette
Torino,
Italy
Erasmus MC
GE Rotterdam, GE Rotterdam
Netherlands
Hospital Universitario 12 de Octubre
Madrid,
Spain
Hospital Universitari i Politecnic La Fe
Valencia,
Spain
Queen Elizabeth Hospital Birmingham
Birmingham,
United Kingdom
National Hospital for Neurology & Neurosurgery
London,
United Kingdom
Royal Free London NHS Foundation Trust
London,
United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne,
United Kingdom
Salford Royal Hospital
Salford,
United Kingdom