Duchenne Muscular Dystrophy (DMD)
Conditions
Keywords
muscle regeneration, SAT-3247
Brief summary
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
DRUGSAT-3247
AAK1 inhibitor
Sponsors
Satellos Bioscience, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
open-label study of 60 mg SAT-3247 in 5-days on/2-days off (i.e., weekday dosing) regimen
Eligibility
Sex/Gender
MALE
Age
18 Years to 40 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment emergent adverse events | 11 months | Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| SAT-3247 effect on fat fraction in biceps brachii | 12 months | Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in biceps brachii following treatment with SAT-3247. |
| SAT-3247 effects on muscle force | 12 months | Changes from baseline in muscle force measurements as determined by dynamometry following treatment. |
| Potential for improvement in muscle function with treatment of SAT-3247 | 12 months | Changes from baseline in Performance of Upper Limb (PUL2.0) assessment following SAT-3247 treatment. |
Countries
Australia
Outcome results
None listed