At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- S-606001 (drug), Placebo (drug)
- How long the study runs
- Study runs about 22 months (dates as stated)
- About the drug or intervention
- S-606001 — drug: S-606001 administered orally · Placebo — drug: S-606001 matching placebo administered orally
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a new medicine called S-606001 in adults with late-onset Pompe disease (LOPD), a condition where the body cannot break down a substance called glycogen properly. The medicine will be given in addition to the enzyme replacement therapy (ERT) that participants are already taking. The study is run by the company Shionogi.
Who can take part
- Age 18 or over and weighing at least 40 kilograms
- A confirmed diagnosis of late-onset Pompe disease, based on either a blood test showing low levels of an enzyme called acid alpha-glucosidase (GAA) or genetic testing
- Breathing tests within a set range, measured sitting upright, or a set drop in breathing test results when lying down
- Able to walk between 75 metres and up to 90% of the distance expected for a healthy adult in a 6-minute walk test
- Already taking enzyme replacement therapy (ERT) for at least 24 months, with no change to the treatment in the last 6 months
Who may not be able to
- Any other medical condition or circumstance that could pose a safety risk or affect the ability to follow the study rules
- An active infection at the screening visit
- Cancer in the past 5 years, apart from certain fully treated skin cancers
- Current or long-term liver disease
- Certain inherited gene changes affecting glycogen (in the GYG or PYGM genes)
- Taking another experimental Pompe treatment recently or planning to during the study
- Having had gene therapy or small interfering RNA therapy for Pompe disease
- Being pregnant or breastfeeding, or planning to have a child during the study
What taking part involves
- • Taking the study medicine S-606001 as an add-on to your existing enzyme replacement therapy (ERT)
- • Details of how the medicine is given, the dose, and how long treatment lasts: Not stated — ask the trial team
Time commitment: Not stated — ask the trial team (the data does not say how many visits are needed or how long the study lasts).
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years and over
- Who
- All
- Number of participants
- 45
- Started
- 2025-10-30
- Last checked
- 2026-06
Plain English Summary
What is this study?
- • Testing a new treatment for pompe disease
- • Phase2 - 45 participants
- • The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT
Who can take part?
- • Ages 18 Years and over
- • Diagnosed with pompe disease
Where?
- • Birmingham - Queen Elizabeth Hospital Birmingham
- • London - National Hospital for Neurology & Neurosurgery
- • London - Royal Free London NHS Foundation Trust
- • Newcastle upon Tyne - Royal Victoria Infirmary
- • +1 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years and over
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ a diagnosis of LOPD based on documentation of 1 of the following:
What the study is looking for
- ✓Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the agreement to take part.
- ✓Participant must have a diagnosis of LOPD based on documentation of 1 of the following:
- ✓Deficiency of acid alpha-glucosidase (GAA) enzyme
- ✓GAA genotype
- ✓Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following...
Who cannot take part
- ✗Has active infections at screening.
- ✗cancer within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have...
- ✗Current or chronic history of liver disease.
- ✗Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle...
- ✗Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.
See the full criteria
Where Is This Study? (5 UK sites)
Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
National Hospital for Neurology & Neurosurgery
London, United Kingdom
Royal Free London NHS Foundation Trust
London, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Salford Royal Hospital
Salford, United Kingdom
How to Get in Touch
Shionogi Clinical Trials Administrator Clinical Support Help Line
Sponsor contactCONTACT
