Japanese Early-stage Clinical Trial of Ultra-high Dose Methylcobalamin for Amyotrophic Lateral Sclerosis: a Pivotal Phase 3 Randomized Controlled Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 130
- 试验地点
- 16
- 主要终点
- Treatment Phase: ALSFRS-R
研究概览
简要总结
To examine the clinical efficacy and safety of ultra-high dose (50mg, im, twice a week) methylcobalamin in retarding the progression of symptoms in amyotrophic lateral sclerosis (ALS) patients, we enroll ALS patients diagnosed by Updated Awaji Criteria within 12 months after the clinical onset. First they are followed for 12 weeks with Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) scores, and only those who exhibit drops of 1-2 points are allowed to enter into the test period. A total of 128 patients are randomized and the half having placebo. They are blindly evaluated for drops of ALSFRS-R in 16 weeks, as the primary outcome. After this, all subjects receive methylcobalamin.
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
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Treatment and OLE Phase:
- •ALS patients within 12 months after clinical onset at the entry
- •Diagnosed based on updated Awaji criteria: definite, probably or laboratory supported probable
- •Decline of ALSFRSR being 1 or 2 during observation period of 12 weeks
- •Japanese Clinical Severity Scale 1 or 2
- •Those who can visit the participating medical centers
- •Tsunagi Phase:
- •The subjects for the Tsunagi phase are patients with ALS who are continuing the administration of this drug at the time of approval, and have obtained consent for the transition to the Tsunagi phase.
排除标准
- •Treatment and OLE Phase:
- •Those who have tracheostomy
- •Those who had NIPPV
- •%FVC<60%
- •Those who have Chronic Obstructive Pulmonary Disease (COPD)
- •Those who have symptoms and signs of B12 deficiency
- •Those who had edaravone less than 4 weeks prior to entry
- •Those who changed the schedule and dosing of riluzole
- •Those who have dementia
- •Those who have the possibility of pregnancy
- •Those who have serious respiratory or cardiac diseases
- •Those who have malignancies
- •Those who participated other clinical trials within 12 weeks
- •Those who have allergies to B12 and related compounds
- •Tsunagi Phase:
- •Not applicable.
研究组 & 干预措施
OLE Phase: methylcobalamin
intramuscular injection of methylcobalamin
干预措施: methylcobalamin (Drug)
Treatment Phase: Placebo
intramuscular injection of saline solution
干预措施: saline solution (Drug)
Treatment Phase: methylcobalamin
intramuscular injection of methylcobalamin
干预措施: methylcobalamin (Drug)
Tsunagi Phase: methylcobalamin
intramuscular injection of methylcobalamin
干预措施: methylcobalamin (Drug)
结局指标
主要结局
Treatment Phase: ALSFRS-R
时间窗: during 16 weeks of test period
Drop of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (maximum or normal 48 and minimum 0; higher values represent better condition)
次要结局
- Treatment Phase: %Functional Vital Capacity (FVC)(during 16 weeks of test period)
- Treatment Phase: time to event(during 16 weeks of test period)
- Treatment Phase: Manual Muscle Testing (MMT)(during 16 weeks of test period)
- Treatment Phase: 40-item Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40)(during 16 weeks of test period)
- Treatment Phase: homocystein(during 16 weeks of test period)
- Treatment Phase: Norris scale(during 16 weeks of test period)
- Treatment Phase: Grip Power(during 16 weeks of test period)