A Phase I, Open Label, Single Dose Study to Evaluate the Safety and Efficacy of an Injectable Follistatin Plasmid Gene Therapy in Healthy Subjects
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 43
- 试验地点
- 2
- 主要终点
- Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
研究概览
简要总结
This study examines the safety and effects of an injectable plasmid gene therapy. Plasmids are circular pieces of DNA which have been widely studied as a non-permanent & non-heritable method for transferring genes and inducing gene expression. In this study the plasmid is a gene vector which contains the human FST344 gene intended to express and secrete bioidentical human follistatin into serum circulation. Follistatin is a myostatin and activin inhibitor that has demonstrated improved functional outcomes in mouse models of neuromuscular disease.
Participants will undergo dual energy x-ray absorptiometry scans before and after the treatment's administration to compare change from baseline and rates of change in fat vs muscle tissue and bone density. Participants will also undergo metabolic and epigenetic blood panels to observe any changes. Participants will be monitored at the clinic site for a short time period after receiving the therapy and participants will be able to report any adverse events through an online form. Lastly, participants will have blood drawn just prior to, and three months after, the gene therapy's administration in order to assess circulating levels of follistatin. This study is administered at the Global Alliance for Regenerative Medicine clinical research site on the island of Roatan and is sponsored by Minicircle. The main contact for this study is Mac Davis.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 22 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Openness to morphological change
- •Will practice contraception for the duration of trial (if female)
排除标准
- •Women who are pregnant, nursing or of childbearing potential who are unwilling or unable to maintain contraceptive therapy for the duration of the study
- •Life expectancy < 6 months due to concomitant illness
- •Pre-existing joint, ligament, or heart issues
- •Systolic blood pressure (supine) ≤90 mmHg;
- •Resting heart rate > 100 bpm
- •Unwilling or unable to give written informed consent.
结局指标
主要结局
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
时间窗: 3 months
Change from Baseline in Follistatin Serum Concentration at 3 months
时间窗: 3 months
次要结局
- Change in intrinsic and extrinsic epigenetic clock age(3 months)
- Change from Baseline in fat-free mass at 3 months via dual x-ray absorptiometry(3 months)
- Change from Baseline in fat mass at 3 months via dual x-ray absorptiometry(3 months)