Evaluation of the Safety, Tolerability, Efficacy, and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for Treatment of Amyotrophic Lateral Sclerosis: Open-Label Extension
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 90
- 试验地点
- 24
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500, also known as CENTAUR).
详细描述
The Centaur Open Label Extension Study (CENTAUR-OLE) is an extension study for patients with ALS who participated in the CENTAUR study (Study AMX3500). During the OLE, all participants received active treatment (AMX0035), and the investigators, evaluators, and participants remained blinded to the randomized treatment assigned at the beginning of the double-blind main study. The study was designed to assess the longer term safety and therapeutic potential of AMX0035.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completion of all visits in the randomized, double blind AMX3500 study. Participants who received tracheostomy or permanent assisted ventilation (PAV) during the course of the main study could enroll in the OLE if they completed all visits in the main study.
- •Must enroll in the OLE within 28 days of the Week 24 visit of the main study.
- •Signed informed consent to enter the OLE phase.
排除标准
- •Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV.
- •Exposure to or anticipated requirement for any disallowed medication listed in the protocol.
- •Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug.
- •Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study
- •Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.
研究组 & 干预措施
AMX0035
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (3g) and Taurursodiol (1g)
干预措施: AMX0035 (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
时间窗: From the Baseline Visit in the OLE study through Week 132 or the Early Discontinuation (Final Safety) Visit for each participant (for up to approximately 132 weeks)
Number of participants with TEAEs from baseline in the OLE study through the last participant's last visit in the OLE
次要结局
- Amyotrophic Lateral Sclerosis Rating Scale Revised Total Score (ALSFRS-R) Change in Slope(From baseline in the Main Study through Week 24 of the OLE (48 weeks overall))
- Survival - Time to Death(From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks))
- Composite of Time to Hospitalization, Death or Death Equivalent(From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks))
- Slow Vital Capacity Change in Slope(From baseline in the Main Study through Week 24 of the OLE (48 weeks overall))
- Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Upper Extremities(From baseline in the Main Study through Week 24 of the OLE (48 weeks overall))
- Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Lower Extremities(From baseline in the Main Study through Week 24 of the OLE (48 weeks overall))
- Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change in Slope(From Baseline in the Main Study through Week 24 in the OLE (48 weeks overall))