New hope for pompe patients: trial tests Add-On to standard therapy

NCT ID NCT07123155

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

Summary

This study is testing whether adding an investigational drug called S-606001 to standard enzyme replacement therapy (ERT) is safe and can help adults with late-onset Pompe disease. It will measure changes in breathing, walking ability, muscle strength, and fatigue over one year. The goal is to see if the combination provides better disease control than ERT alone.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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
NOT_YET_RECRUITING

Torino, Italy
AP-HP Hopital Raymond Poincare
NOT_YET_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
NOT_YET_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
HCL Hopital Pierre Wertheimer
NOT_YET_RECRUITING

Bron, France
Hospital Universitari i Politecnic La Fe
NOT_YET_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
NOT_YET_RECRUITING

London, United Kingdom
Royal Victoria Infirmary
RECRUITING

Newcastle upon Tyne, United Kingdom
Salford Royal Hospital
RECRUITING

Stott Lane, Statford, United Kingdom
SphinCS GmbH
NOT_YET_RECRUITING

Höchheim, Germany
UZ Leuven
RECRUITING

Leuven, Belgium
Universitaetsklinikum Halle (Saale)
NOT_YET_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