A Phase 2b/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, 12 Month Clinical Trial to Evaluate the Efficacy and Safety of MN-166 (Ibudilast) Followed by Open-Label Extension Phase in Subjects With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- MediciNova
- 入组人数
- 234
- 试验地点
- 30
- 主要终点
- Change from baseline in ALSFRS-R score at Month 12 (or last measurement before death in case of censoring) and survival time.
研究概览
简要总结
A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.
详细描述
This is a Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 followed by an open-label extension phase compared to matching placebo in subjects diagnosed with ALS.
The study will consist of a screening phase (up to 30 days) followed by a double-blind phase (12 months). Following the screening phase, subjects who continue to meet entry criteria will be randomly assigned to one of two treatment groups: MN-166 or matching placebo in a 1:1 ratio. Upon completion of the double-blind phase, subjects will be given the option to continue to the Open-label Extension Phase for a period of six months.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Major Inclusion Criteria:
- •Male or female subjects age 18 - 80 years, inclusive;
- •Diagnosis of familial or sporadic ALS as defined by the El Escorial-Revised (2000) research diagnostic criteria for ALS [clinically definite, clinically probable, probable-laboratory-supported];
- •ALS onset of ≤18 months from first clinical signs of weakness prior to screening;
- •If currently using riluzole, subject must be on a stable dose for at least 30 days prior to initiation of study drug;
- •If currently using edaravone, subject should have completed at least 14 days of their initial treatment cycle prior to initiation of study drug;
- •Last documented pulmonary function test result (i.e., slow vital capacity or forced vital capacity) must be greater than or equal to 70% predicted;
- •Able to swallow study medication capsules;
- •No known allergies to the study drug or its excipients;
- •Received pneumococcal vaccine within 6 years prior to starting clinical trial.
排除标准
- •Confirmed hepatic insufficiency or abnormal liver function (AST and/or ALT >3 times upper limit of normal);
- •Currently diagnosed with a clinically significant psychiatric disorder or dementia that would preclude evaluation of symptoms;
- •Currently use or treated with parenteral (intramuscular or intravenous) high dose (>25 mg/week) Vitamin B12 within 30 days prior to study drug administration;
- •Poor peripheral venous access that will limit the ability to draw blood as judged by the Investigator;
- •Currently participating, or has participated in a study with an investigational or marketed compound or device within 30 days or 5 half-lives, whichever is shorter, prior to signing the informed consent;
- •Use of tracheostomy or >22/24-hour ventilatory support.
研究组 & 干预措施
MN-166
Subjects will take MN-166 10 mg capsules, up to 50 mg twice a day for 12 months.
干预措施: MN-166 (Drug)
placebo
Subjects will take up to 5 matching placebo capsules twice a day for 12 months.
干预措施: placebo (Drug)
结局指标
主要结局
Change from baseline in ALSFRS-R score at Month 12 (or last measurement before death in case of censoring) and survival time.
时间窗: 12 months
The amyotrophic lateral sclerosis functional rating scale-revised, or ALSFRS-R, measures the functional status of subjects with ALS. It is based on 12 items, each of which is rated on a 5-point scale (0 to 4). The rate of total functional disability thus ranges from 0 (maximum disability) to 48 (normal function) points.
次要结局
- Mean change from baseline of functional activity measured by ALSFRS-R at Month 12(12 months)
- Responders, measured in percent of subjects overall, whose ALSFRS-R total score was stable or improved(12 months)
- Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v4.0(12 months)
- Changes from Baseline in Laboratory Values(12 months)
- Mean change from baseline of muscle strength measured by hand-held dynamometry(Baseline, Treatment Phase Week 6, Months 3, 6, 9 and12 time points.)
- Time to survival(12 months)
- Mean change from baseline on quality of life assessed by ALSAQ-5 at Month 12(12 months)