A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 280
- 试验地点
- 67
- 主要终点
- Number of Participants With Adverse Events
研究概览
简要总结
The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).
详细描述
Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form
- •Completed participation on study drug and the Follow-Up Visit in the CY 4031 study
- •Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree for the duration of their participation in the study to either:
- •Use a condom during sexual intercourse with female partners who are of childbearing potential AND to have female partners use a highly effective means of contraception OR
- •Abstain from sexual intercourse during participation in the study
- •Female patients who are not post-menopausal (≥ 1 year) or sterilized, must:
- •Not be breastfeeding
- •Have a negative pregnancy test
- •Have no intention to become pregnant during participation in the study AND
- •Practice sexual abstinence, defined as refraining from intercourse during the duration of the study OR if male partners are not vasectomized with a confirmed zero sperm count, require use of a condom AND use of a highly effective contraceptive measure
排除标准
- •Has a diaphragm pacing system (DPS) at study entry or anticipate DPS placement during the course of the study
- •Has taken an investigational study drug (other than tirasemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
- •Use of tizanidine and theophylline-containing medications during study participation
- •Participation or planning to participate in any form of stem cell therapy for the treatment of ALS or another investigational drug
研究组 & 干预措施
Delayed Start Treatment
The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.
干预措施: tirasemtiv (Drug)
Early Start Treatment
The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033.
干预措施: tirasemtiv (Drug)
结局指标
主要结局
Number of Participants With Adverse Events
时间窗: From the first dose of tirasemtiv through 28 days after the last dose
The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv.
次要结局
- Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033(baseline and 24 weeks)
- Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033(baseline and 48 weeks)
- Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24(baseline and 24 weeks)
- Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48(baseline and 48 weeks)