Duchenne Muscular DystrophyPhase 2BASECAMPNCT07287189
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
A Phase 2 study testing SAT-3247 in people with Duchenne Muscular Dystrophy and Duchenne, run by Satellos Bioscience, Inc. at 20 sites in 8 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 7 to 9 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Duchenne, DMD
- What is being tested?
- SAT-3247
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 51 participants
- Where
- 20 sitesin 8 countries
- Study dates
- 8 Dec 2025to 28 Feb 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 9 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene.
- Male DMD patients who are ambulatory and aged ≥ 7 to < 10 years at the time of screening.
- Stable dose of systemic glucocorticoids (i.e., prednisolone, deflazacort, or vamorolone) according to the standard of care for ≥ 3 months prior to the Screening Visit and for the duration of the trial. Patients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit.
- Stable doses of prescription medicines including ACE inhibitors, β-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care ≥ 1 month prior to the Screening Visit and for the duration of the trial.
- Participants that have previously received delandistrogene moxeparvovec (brand name Elevidys) either in a prior clinical trial or in the commercial setting > 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.
- Participants that have previously received an exon skipper > 6 months prior to Screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.
Show all 9Show fewer
- Participants receiving a stable dose of givinostat (brand name Duvyzat) for at least 18 months or longer prior to the Screening Visit will be eligible. Participants unable to tolerate givinostat who discontinued treatment before 18 months are eligible to enroll if date of last dose is ≥ 30 days from the Screening date. Givinostat should not be discontinued, if tolerated, to meet study entry criteria.
- Participants that have received prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) ≥ 24 months prior to the Screening Visit.
- If participating in a physical therapy/strength training regimen, must be stable for ≥ 2 months prior to the Screening Visit and for the duration of the trial.
Who cannot join
- Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.
- Participants for whom MRI or open muscle biopsy are contraindicated.
- Evidence of significant hepatic dysfunction, defined as GLDH > 2X upper limit of normal (ULN) at the Screening Visit.
- Impaired cardiac function defined as a left ventricular ejection fraction of < 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy.
- A forced vital capacity < 60% predicted at the Screening Visit.
- Ongoing participation in any other therapeutic clinical trial or follow-up study for a therapeutic intervention
Show all 9Show fewer
- Consumption of grapefruit juice or grapefruit containing products
- Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.
- Additional entry criteria will be reviewed with the clinical site investigator.
Trial locations and contacts
20 sites in 8 countries.
20 sites in 8 countries
United States7 sites
Colorado Children's
Aurora, Colorado, 80045
Lurie Children's
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of California Los Angeles
Los Angeles, California, 90095
Seattle Children's
Seattle, Washington, 98105
Washington University
St Louis, Missouri, 63110
UMass Memorial Medical Center
Worcester, Massachusetts, 01655
Spain3 sites
Hospital Infantil i Hospital de la Dona
Barcelona, 08035
Hospital Universitario Donostia
Donostia / San Sebastian, Basque Country, 20014
Hospital Universitario y Politécnico La Fe
Valencia, Valencia, 46026
Australia2 sites
Royal Children's Hospital Melbourne
Melbourne, Victoria, 3052
Children's Hospital at Westmead
Westmead, New South Wales, 2145
Belgium2 sites
UZ Gent
Ghent, Oost-Vlaanderen, 9000
Hôpital De La Citadelle (CHR)
Liège, Liège, 4000
Poland2 sites
Klinika Neurologii Rozwojowej Uniwersyteckie
Gdansk, Pomeranian Voivodeship, 80-952
Instytut Centrum Zdrowia Matki Polki
Lodz, Łódź Voivodeship, 93-338
Serbia2 sites
University Children's Clinic Tirsova
Belgrade, Serbia, 11000
Mother and Child Health Care Institute
Belgrade, Serbia, 11070
About this study
Official title: A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients
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.
Read the detailed description
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).
- Being tested
- SAT-3247, Placebo
- Keywords
- muscle regeneration, satellite cell, asymmetric division, dystrophin
- Medical subject headings
- Muscular Dystrophy, Duchenne, Neuromuscular Diseases, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAT-3247Drug
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.
PlaceboDrug
matching placebo oral tablets
| Study group | Role | What they receive |
|---|---|---|
| SAT-3247 60 mg | Active comparator | Part 1: SAT-3247 60 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 60 mg oral tablets administered daily for an additional 9 months after completing Part 1 Receives: Drug: SAT-3247 |
| SAT-3247 120 mg | Active comparator | Part 1: SAT-3247 120 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 120 mg oral tablets administered daily for an additional 9 months after completing Part 1; note the 120 mg dose will not be studied in the US and Canada Receives: Drug: SAT-3247 |
| placebo | Placebo comparator | Part 1: placebo oral tablets administered daily for 12 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of SAT-3247
Occurrence of treatment emergent adverse events and relationship to investigational product
Measured: 12 weeks in Part 1 and up to 12 months in part 2
Tolerability of SAT-3247
occurrence of clinically significant changes in physical exam, clinical laboratory measures, vital signs, and ECG
Measured: 12 weeks in Part 1 and up to 12 months in Part 2
SAT-3247 effects on muscle strength
change from baseline in muscle force as determined by dynamometry
Measured: 12 weeks in Part 1 and up to 12 months in Part 2
Secondary outcomes (4)
SAT-3247 effects on muscle quality
change from baseline in intramuscular fat fraction in quantitative magnetic resonance in vastus lateralis (thigh muscle)
Measured: 12 weeks in Part 1 and up to 12 months in Part 2
SAT-3247 effects on muscle function
changes from baseline in north star ambulatory assessment
Measured: 12 weeks in Part 1 and up to 12 months in Part 2
SAT-3247 effects on muscle function
changes from baseline in stride velocity 95th Centile
Measured: 12 weeks in Part 1 and up to 12 months in Part 2
SAT-3247 effects on muscle regeneration
changes from baseline in the regeneration index as measured from an open biopsy of biceps brachii
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2025
- Study start8 Dec 2025
- Primary completion31 Mar 2027 (estimated)
- Study completion28 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- randomized, placebo-controlled, 2-dose comparison
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 51 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Satellos Bioscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Satellos Chief Medical Officer, Study director, Satellos Bioscience, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07287189
- Sponsor's protocol ID
- SAT-3247-CL-201
- Other IDs
- 2025-522522-13-01, 2025
- Acronym
- BASECAMP
- Status last verified
- Aug 2026
- First submitted
- 11 Dec 2025
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No