A Double-blind, Randomized, Multicenter, Placebo-Controlled, Parallel, Phase III Clinical Trial to Evaluate the Efficacy and Safety of Lenzumestrocel(Neuronata-R® Inj.) in Patients with Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 115
- 试验地点
- 5
- 主要终点
- Joint rank scores (CAFS, Combined Assessment of Functional and Survival)
研究概览
简要总结
ALSUMMIT is a double-blind, randomized, placebo-controlled, multi-center, parallel, phase III clinical trial to evaluate and confirm the efficacy and long-term safety of repeated Lenzumestrocel (Neuronata-R® inj.) treatment.
详细描述
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by selective and progressive loss of motor neurons. Disease progression leads to death within 2-4 years, but there exists no definite treatment so far.
Based on phase I/II clinical trial(NCT01363401), twice intrathecal autologous bone marrow-derived mesenchymal stem cells (Lenzumestrocel) injections showed significant therapeutic benefit lasting at least six months with safety in patients with ALS.
Additionally, the switch from pro- to anti-inflammatory conditions, which was indicated from the inverse correlation between TGF-β1 and MCP-1 levels after Lenzumestrocel injections in the good responder, has been considered a plausible beneficial action mechanism.
This study is designed to investigate the following. First, to reconfirm and evaluate the long-term efficacy of twice injections (single cycle) of Lenzumestrocel, group 1 will receive a single cycle injection with a 26-day interval.
Second, to evaluate the long-term safety and efficacy of Lenzumestrocel repeated injections, group 2 will receive a single cycle injection a 26-day apart followed by three times injections every three-month interval.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 25 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Single cycle administration group
Study drug injections twice in a 26-day interval followed by three times comparator injections every three months.
干预措施: Riluzole (Drug)
Single cycle administration group
Study drug injections twice in a 26-day interval followed by three times comparator injections every three months.
干预措施: Lenzumestrocel (Biological)
Single cycle administration group
Study drug injections twice in a 26-day interval followed by three times comparator injections every three months.
干预措施: Placebo Comparator (Drug)
Multiple administation group
Study drug injections twice in a 26-day interval followed by repeated three times study drug injections every three months.
干预措施: Lenzumestrocel (Biological)
Multiple administation group
Study drug injections twice in a 26-day interval followed by repeated three times study drug injections every three months.
干预措施: Riluzole (Drug)
Control group
Comparator injections twice in a 26-day interval followed by three times comparator injections every three months.
干预措施: Riluzole (Drug)
Control group
Comparator injections twice in a 26-day interval followed by three times comparator injections every three months.
干预措施: Placebo Comparator (Drug)
结局指标
主要结局
Joint rank scores (CAFS, Combined Assessment of Functional and Survival)
时间窗: at 12 months, and 6 months
Joint rank score is derived from Combined Assessment of Function and Survival. Functional assessment is based on ALSFRS-R scores and survival assessment is based on the period from randomization to physical death. Joint rank score will be calculated with ALSFRS-R score data and survival. For each pairwise comparison, a study participant is assigned a score and then the summed scores are ranked for all participants. The higher ranking, the higher score, and the lower ranking, the lower score. And the average rank score is then calculated for each treatment group. A higher mean rank score indicates that participants in that treatment group, on average, fared better. 1. The difference in joint rank scores between multiple administration group and control group at 12 months. 2. The difference in joint rank scores between single cycle administration group and control group at 6 months
次要结局
- Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score(at 12 months, 6 months)
- Time to event(at 12 months, 6 months)
- Joint rank scores (CAFS, Combined Assessment of Functional and Survival)(at 6 months)