New pill could boost breathing in pompe disease – early trial underway

NCT ID NCT07123155

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental oral drug called S-606001 in 45 adults with late-onset Pompe disease. Participants take the drug or a placebo on top of their standard enzyme replacement therapy. The main goal is to see if the drug improves lung function and walking ability over 52 weeks, while also checking safety.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: S-606001 (an oral drug taken as a pill)
What this could lead to: If it works, this could lead to a new add-on treatment that improves breathing and muscle function for people with late-onset Pompe disease.
What could go wrong: This is an early Phase 2 trial with only 45 participants, so results may not apply to everyone. The drug is unproven and may cause side effects or fail to show benefit.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

disorder of glycogen metabolism glycogen storage disease due to acid maltase deficiency, late-onset glycogen storage disease II Glycogen Storage Disease Type II Rare Diseases

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

POMPE DISEASE

Contacts and locations

Locations

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

London, United Kingdom
Queen Elizabeth Hospital Birmingham
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

Salford, 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

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