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

NCT ID NCT07123155

Disease control Sponsor: Shionogi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 45 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent. * Participant must have a diagnosis of LOPD based on documentation of 1 of the following: 1. Deficiency of acid alpha-glucosidase (GAA) enzyme 2. GAA genotype * Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position. * Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria: 1. Screening values of 6-minute walk distance (6MWD) are ≥75 meters 2. Screening values of 6MWD are ≤90% of the predicted value for healthy adults * Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months. Key Exclusion Criteria: * Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements. * Has active infections at screening. * Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years. * Current or chronic history of liver disease. * Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) . * Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study. * Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease. * Participant, if female, is pregnant or breastfeeding at screening. * Participant, whether male or female, is planning to conceive a child during the study. Note: Other protocol-specified inclusion and exclusion criteria may apply.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    28 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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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