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
- SAT-3247 (drug), Placebo (drug)
- How long the study runs
- Study runs about 26 months (dates as stated)
- About the drug or intervention
- SAT-3247 — drug: SAT-3247 is a selective AAK1 inhibitor for oral tablet administration which promotes functional rescue of asymmetric satellite cell division, resulting in the robust production of muscle progenitor cells, subsequent improvement in muscle regeneration, and enhanced muscle function. · Placebo — drug: matching placebo oral tablets
- Patient visit burden
- Not specified by the sponsor
In plain English
This Phase 2 study is testing a medicine called SAT-3247 in boys with Duchenne Muscular Dystrophy (DMD) who can still walk. It is run by Satellos Bioscience, Inc. The aim, number of participants and where it takes place are not stated — ask the trial team.
Who can take part
- Boys aged 7 to under 10 who can still walk
- A confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) based on clinical findings and genetic testing showing a change in the DMD gene
- Taking a steady dose of steroid medicine (prednisolone, deflazacort or vamorolone) for at least 3 months before screening, or having stopped steroids at least 3 months before
- Stable doses of other medicines, including heart and blood pressure medicines, for at least 1 month before screening
- Some children who have had certain other DMD treatments before may be able to join, including Elevidys (if given more than 18 months ago), exon-skipping medicines (if given more than 6 months ago), or Duvyzat (if taken steadily for at least 18 months, or stopped at least 30 days before screening)
- If doing physiotherapy or strength training, this must have been unchanged for at least 2 months before screening
Who may not be able to
- Children expected to lose the ability to walk within 12 months
- Children who cannot have an MRI scan or a muscle biopsy
- Signs of serious liver problems found in screening blood tests
- Heart problems, including a heart pumping measurement below 50% or symptoms of heart muscle disease
- Breathing test results below 60% of the expected level
- Taking part in another treatment trial or follow-up study
- Drinking grapefruit juice or eating grapefruit products
- Severe behavioural or learning difficulties that would make taking part too difficult, in the researcher's view
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team for details of how long the study lasts, how many visits are needed and what tests are involved.
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
- 7 Years to 9 Years
- Who
- Male
- Number of participants
- 51
- Started
- 2025-12-08
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for duchenne muscular dystrophy
- • Phase2 - 51 participants
- • Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years
Who can take part?
- • Ages 7 Years to 9 Years
- • Diagnosed with duchenne muscular dystrophy
- • Male only
Where?
- • London - Great Ormond Street
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial has two parts. In Part 1, the trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy. In Part 2, the trial will study two doses of SAT-3247 for an additional 9 months.
More detail
This is a global phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial has two parts. In Part 1, the trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy. In Part 2, the trial will study two doses of SAT-3247 for an additional 9 months. One dose of SAT-3247 and placebo will be studied in the US and Canada; two doses of SAT-3247 and placebo will be studied in UK, EU, Serbia, and Australia. Enrollment of up to 51 ambulatory DMD participants aged ≥ 7 and \< 10 years of age is planned globally. Randomization will be stratified by baseline corticosteroid regimen and prior DMD concomitant medications. In Part 1, each participant will receive once daily doses of SAT-3247 or matched placebo for 12 weeks. In Part 2, each participant will receive a once daily dose of SAT-3247 for an additional 9 months. Participants will be screened within 28 days before initiating dosing of investigational product at Baseline. Following the Screening period, participants will complete a Baseline visit (Visit 2), a follow-up phone call at Week 1, and visits at Week 4 (Visit 3), Week 8 (Visit 4), Week 12 (Visit 5), Week 24 (Visit 6), Week 36 (Visit 7) and Week 48 (Visit 8).
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: 7 Years - 9 Years
- Who can join: Male only
Treatment history
Treatments you must have had:
- ✓ an investigational gene therapy product (other than del
What the study is looking for
- ✓Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed...
- ✓Male DMD patients who are ambulatory and aged ≥ 7 to \< 10 years at the time of screening.
- ✓Participants that have received prior treatment with an investigational gene therapy product (other than...
- ✓If participating in a physical therapy/strength training regimen, must be stable for ≥ 2 months prior to the...
Who cannot take part
- ✗Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.
- ✗Participants for whom MRI or open muscle biopsy are not safe to use.
- ✗Evidence of significant liver dysfunction, defined as GLDH \> 2X upper limit of normal (ULN) at the Screening Visit.
- ✗Impaired heart function defined as a left ventricular ejection fraction of \< 50% on screening heart assessments...
- ✗A forced vital capacity \< 60% predicted at the Screening Visit.
See the full criteria
Where Is This Study? (1 UK site)
Great Ormond Street
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700