New drug SAT-3247 tested for long-term muscle protection in duchenne MD
NCT ID NCT06867107
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study looks at the long-term safety and effects of a drug called SAT-3247 in 10 people with Duchenne muscular dystrophy who were in a previous trial. Participants take the drug by mouth five days a week for about 11 months. Researchers will check for side effects and measure changes in muscle fat and strength.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- SAT-3247 (a drug taken by mouth that may help muscle repair)
- What this could lead to
- If it works, this could point toward a treatment that slows muscle damage in Duchenne muscular dystrophy.
- What could go wrong
- This is a very small, early-phase study with only 10 participants, so results may not apply to everyone. The drug's long-term safety and effectiveness are still unknown.
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 10 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2025
- Expected to finish
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Aug 2026
An estimate. End dates often move.
- Lead sponsor
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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
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18 to 40 years
- Sex
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Male participants only
- Healthy volunteers
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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 Hide 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.
Inclusion Criteria: * Previously participated in the SAT-3247-CL-101 parent clinical trials. * Continued status of stable glucocorticosteroid dose or no glucocorticosteroid dose from parent clinical trial. * Continued stable doses of prescription medicines (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care from parent clinical trial. * 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. Exclusion Criteria: * 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. * 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. * 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. a. 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. a. 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. * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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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
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St. Vincent Hospital
Melbourne, Victoria, Australia
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The Royal Children's Hospital
Melbourne, Victoria, 3052, Australia
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Other studies related to the condition(s) this trial covers.
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