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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Arm | Experimental | SAT-3247 60 mg administered orally in a 5-days on/2-days off (weekday) dosing regimen |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SAT-3247 | Drug | AAK1 inhibitor |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment emergent adverse events | Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs) | 11 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 12 months |
| SAT-3247 effects on muscle force |
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Inclusion Criteria:
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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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Royal Children's Hospital | Melbourne | Victoria | 3052 | Australia | ||
| St. Vincent Hospital |
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open-label study of 60 mg SAT-3247 in 5-days on/2-days off (i.e., weekday dosing) regimen
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Changes from baseline in muscle force measurements as determined by dynamometry following treatment.
| 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. | 12 months |
| Melbourne |
| Victoria |
| Australia |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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