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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.

Disclaimer Read more

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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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

Study contacts

  • Contact

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

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