NCT06867107Enrolling By Invitation2 期
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
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 入组人数
- 10
- 试验地点
- 3
- 主要终点
- Treatment emergent adverse events
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 40 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Treatment Arm
Experimental
SAT-3247 60 mg administered orally in a 5-days on/2-days off (weekday) dosing regimen
干预措施: SAT-3247 (Drug)
结局指标
主要结局
Treatment emergent adverse events
时间窗: 11 months
Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs)
次要结局
- SAT-3247 effect on fat fraction in biceps brachii(12 months)
- SAT-3247 effects on muscle force(12 months)
- Potential for improvement in muscle function with treatment of SAT-3247(12 months)
研究者
研究点 (3)
Loading locations...
相似试验
已完成
1 期
A Long-Term Study of the Effects of Orally Administered SAR302503 in Patients With MyelofibrosisMyelofibrosisNCT00724334Bristol-Myers Squibb42
终止
2 期
A Study to Evaluate the Long-term Safety and Tolerability of SAGE-324 in Participants With Essential TremorEssential TremorNCT05366751Sage Therapeutics750
已完成
2 期
A Study of Cariprazine in Patients With Chronic Stable SchizophreniaSchizophreniaNCT00839852Forest Laboratories97
进行中（未招募）
2 期
Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular DegenerationStargardt's DiseaseNCT01736592Sanofi27
进行中（未招募）
2 期
A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1BUsher's SyndromeNCT02065011Sanofi9