Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

45

Participants

Timeline

Start Date

October 30, 2025

Primary Completion Date

August 8, 2027

Study Completion Date

August 8, 2027

Conditions
Pompe Disease
Interventions
DRUG

S-606001

S-606001 administered orally

DRUG

Placebo

S-606001 matching placebo administered orally

Trial Locations (28)

15213

University of Pittsburgh School of Medicine, Pittsburgh

19104

University of Pennsylvania, Philadelphia

22030

Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), Fairfax

27710

Duke University Medical Center, Durham

30322

Emory University Hospital, Atlanta

32611

University of Florida (UF) - Gainesville, Gainesville

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

63130

Washington University in St. Louis, St Louis

92868

University of California - Irvine Medical Center, Irvine

Unknown

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

Erasmus MC, 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

Royal Free London NHS Foundation Trust, London

Royal Victoria Infirmary, Newcastle upon Tyne

Salford Royal Hospital, Salford

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Shionogi

INDUSTRY