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
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: SAT-3247.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy (DMD). Basic parameters: 18 years — 40 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Australia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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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
Overview
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD that previously participated in SAT-3247-CL-101. The study will assess the long-term safety, tolerability and potential efficacy of long-term dosing of 60 mg of orally administered SAT-3247 in a 5-days on/2-days off (i.e. weekday dosing) regimen in an open-label design through 11 months- for a total of 12 months of treatment including the duration of the SAT-3247-CL-101 study. The study will enroll up to 10 participants that previously participated in the SAT-3247-CL-101 study.
Interventions
- Drug SAT-3247
AAK1 inhibitor
Primary outcome measures
- Treatment emergent adverse events [Time frame: 11 months]
Secondary outcome measures (3)
- SAT-3247 effect on fat fraction in biceps brachii [Time frame: 12 months]
- SAT-3247 effects on muscle force [Time frame: 12 months]
- Potential for improvement in muscle function with treatment of SAT-3247 [Time frame: 12 months]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Australia · 2 centers
- The Royal Children's Hospital — Melbourne
- St. Vincent Hospital — Melbourne
Identifiers
NCT: NCT06867107 · SAT-3247-LT-001