New drug combo aims to improve breathing and mobility in pompe patients

NCT ID NCT07123155

Recruiting now Disease control Sponsor: Shionogi Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 26 times

Summary

This study tests whether adding an experimental drug called S-606001 to standard enzyme replacement therapy can help adults with late-onset Pompe disease. Pompe disease causes muscle weakness, including breathing muscles. The trial will measure changes in lung function and walking ability over 52 weeks. About 45 participants will receive either the study drug or a placebo, and safety will be closely monitored.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

A.O.U. Policlinico "G. Martino"
NOT_YET_RECRUITING

Messina, Italy
AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette
RECRUITING

Torino, Italy
AP-HP Hopital Raymond Poincare
RECRUITING

Garches, France
Aarhus University Hospital
RECRUITING

Aarhus, Denmark
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
NOT_YET_RECRUITING

Nice, France
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
RECRUITING

Marseille, France
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Duke University Medical Center
RECRUITING

Durham, North Carolina, 27710, United States
Emory University Hospital
NOT_YET_RECRUITING

Atlanta, Georgia, 30322, United States
Erasmus MC
RECRUITING

GE Rotterdam, GE Rotterdam, Netherlands
HLC Hopital Pierre Wertheimer
RECRUITING

Bron, France
Hospital Universitari i Politecnic La Fe
RECRUITING

Valencia, Spain
Hospital Universitario 12 de Octubre
RECRUITING

Madrid, Spain
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
RECRUITING

München, Germany
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
RECRUITING

Fairfax, Virginia, 22030, United States
National Hospital for Neurology & Neurosurgery
NOT_YET_RECRUITING

London, United Kingdom
Queen Elizabeth Hospital Birmingham
NOT_YET_RECRUITING

Birmingham, United Kingdom
Royal Free London NHS Foundation Trust
RECRUITING

London, United Kingdom
Royal Victoria Infirmary
RECRUITING

Newcastle upon Tyne, United Kingdom
Salford Royal Hospital
RECRUITING

Statford, Statford, United Kingdom
SphinCS GmbH
RECRUITING

Höchheim, Germany
UZ Leuven
RECRUITING

Leuven, Belgium
Universitaetsklinikum Halle (Saale)
RECRUITING

Halle, Germany
University of California - Irvine Medical Center
RECRUITING

Irvine, California, 92868, United States
University of Florida (UF) - Gainesville
RECRUITING

Gainesville, Florida, 32611, United States
University of Pennsylvania
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh School of Medicine
RECRUITING

Pittsburgh, Pennsylvania, 15213, United States
Washington University in St. Louis
RECRUITING

St Louis, Missouri, 63130, United States

Conditions

Explore the condition pages connected to this study.

POMPE DISEASE