Duchenne Muscular Dystrophy (DMD)Phase 2NCT06867107
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
A Phase 2 study testing SAT-3247 in people with Duchenne Muscular Dystrophy (DMD), run by Satellos Bioscience, Inc. at 6 sites in 2 countries.
Trial summary
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101. The study will assess the long-term safety, tolerability and potential efficacy of long-term dosing of orally administered SAT-3247 60 mg in a… Read more about this study
- Who is it for?
- Ages 16 to 40 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- SAT-3247
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 20 Aug 2025to 30 Sep 2027
- 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 16 to 40 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Previously participated in the SAT-3247-CL-101 parent clinical trials (Group A only).
- No previous treatment with SAT-3247 (Group B only)
- Continued status of stable glucocorticosteroid dose or no glucocorticosteroid dose for the duration of the trial.
- Continued stable doses of prescription medicines (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care for the duration of the trial.
- 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 (Group B only)
- Receiving a stable dose of givinostat (brand name Duvyzat) for at least 18 months or longer prior to the Screening (Group B only)
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- 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 (Group B only)
- Have received any medication indicated for DMD (other than corticosteroids, including vamorolone, gene therapy, givinostat, or an exon-skipping therapy) whose last dose was ≥ 6 months prior to Screening (Group B only)
- have received prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) ≥ 24 months prior to Screening
- Ejection fraction ≥ 40% at Screening (Group B only)
- Ability to understand the nature of the trial and any hazards of participating.
- Ability to communicate satisfactorily with the investigator and physiotherapist and to participate in and comply with the requirements of the entire trial including scheduled visits, procedures, laboratory tests, questionnaires, wearable devices, and study restrictions.
- Willingness to give written consent or assent (if not of cognitive capacity of consent in the jurisdiction where the study is being conducted) and parent/legal guardian willing to give written consent to participate (if participant is not of cognitive capacity to consent) after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or their delegate.
- All participants, if sexually active, agree to follow the contraception requirements and sperm donation limitations of the trial as described in the protocol.
Who cannot join
- Presence of acute medical condition, chronic illness or history of chronic illness (other than DMD) sufficient to invalidate the participant's participation in the trial or make it unnecessarily hazardous in the judgment of the investigator.
- Evidence of significant hepatic dysfunction, defined as GLDH > 2X upper limit of normal (ULN) at Screening (Group B only)
- Entry item score on the Performance of Upper Limb (PUL2.0) assessment > 5 (Group B only)
- Requirement for daytime ventilator assistance (Group B only)
- Participants expected to require spine surgeries or hospitalizations for non-acute health needs within 12 months.
- Participants with acute gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea, heartburn) or acute infection (such as influenza) or a significant infection or known inflammatory process at Screening.
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- Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.
- Development of symptomatic cardiomyopathy since completion of the parent trial.
- Inability to swallow tablets.
- Tablets can be split or crushed and stirred into flavored beverages or food (e.g., apple sauce, yogurt) followed by immediate administration.
- Receipt of an investigational product (including prescription medicines and investigational devices) as part of another clinical trial since completion of the parent trial or in the follow-up period of another clinical trial at the time of Screening for this study.
- Use of deflazacort or vamorolone in jurisdictions where these are considered investigational as they have not received health authority marketing authorization will not be exclusionary.
- Current or expected use during the study of any medications that are known strong or moderate inhibitors of CYP3A4 (Group B only)
- Consumption of grapefruit, grapefruit juice, and grapefruit-containing products within 30 days of the first dose of investigational product and for the duration of the study (Group B only)
- Possibility that the participant will not cooperate with the requirements of the protocol or is unable or unwilling to comply with the study requirements according to investigator's decision.
- Employee, contractors, or consultants of the Sponsor, the CRO, and/or study site or their relatives.
Trial locations and contacts
6 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 2 countries
United States4 sites
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of California, Los Angeles
Los Angeles, California, 90095
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Washington University School of Medicine
St Louis, Missouri, 63110
Australia2 sites
The Royal Children's Hospital
Melbourne, Victoria, 3052
St. Vincent Hospital
Melbourne, Victoria
About this study
Official title: An Open-Label Long-Term Follow-up Study of SAT-3247 in Patients With Duchenne Muscular Dystrophy (DMD) Including Those Who Participated in SAT-3247-CL-101
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101.
The study will assess the long-term safety, tolerability and potential efficacy of long-term dosing of orally administered SAT-3247 60 mg in a 5-days on/2-days off (i.e. weekday dosing) regimen in an open-label design through 11 months (Group A) or 12 months (Group B), respectively. The study will enroll up to 30 participants, including up to 5 who previously participated in the SAT-3247-CL-101 study.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- SAT-3247
- Keywords
- muscle regeneration, SAT-3247
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAT-3247Drug
AAK1 inhibitor
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | SAT-3247 60 mg administered orally in a 5-days on/2-days off (weekday) dosing regimen Receives: Drug: SAT-3247 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment emergent adverse events
Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs)
Measured: 12 months
SAT-3247 effect on fat fraction in biceps brachii
Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in biceps brachii following treatment with SAT-3247.
Measured: 12 months
Secondary outcomes (4)
SAT-3247 effects on muscle force
Changes from baseline in muscle force measurements as determined by dynamometry following treatment.
Measured: 12 months
Potential for improvement in muscle function with treatment of SAT-3247
Changes from baseline in Performance of Upper Limb (PUL2.0) assessment following SAT-3247 treatment.
Measured: 12 months
PK measurement of SAT-3247
Evaluate PK of SAT-3247 concentrations in non-ambulatory participants
Measured: 12 months
Long-term effects of SAT-3247 on muscle quantitative magnetic resonance (qMR)
Evaluate change in proton muscle transverse relaxation time (T2) in biceps brachii
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2025
- Study start20 Aug 2025
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies 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
- Not applicable
- Intervention model
- Single group
- Model description
- open-label study of 60 mg SAT-3247 in 5-days on/2-days off (i.e., weekday dosing) regimen
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Satellos Bioscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06867107
- Sponsor's protocol ID
- SAT-3247-LT-001
- Status last verified
- Oct 2026
- First submitted
- 4 Mar 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No