A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
试验速览
- 阶段
- 2 期
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
研究概览
简要总结
Multicenter, randomized, double-blind, placebo controlled study to assess the tolerabilty and efficacy of CuATSM in patients with ALS/MND. Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment.
详细描述
Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment. Study drug is administered orally, once a day in fasted state (before breakfast). Assessments for safety (physical examination, vital signs, hematology, serum chemistry adverse events) will be conducted at baseline and following each cycle of treatment. Assessments for efficacy (Revised ALS Functional Rating Scale [ASLFRS-R] score, and Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen [ECAS] score, and seated slow vital capacity [SVC]) will be conducted at baseline and following 2, 4 and 6 cycles of treatment. Analysis of covariance (ANCOVA) will be used to compare efficacy endpoints between CuATSM and placebo groups.
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)
盲法说明
placebo controlled
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •signed informed consent
- •familial or sporadic ALS/MNS by Awaji-shima Consensus Recommendations
- •not taking riluzole or on stable dose of riluzole for 4 weeks prior to screening visit
- •no prior exposure to agents other than riluzole for treatment of ALS
- •adequate bone marrow reserve, renal and liver function
- •women of childbearing potential must have a negative pregnancy test and be non-lactating
- •women and men with partners of childbearing potential must take effective contraception while on treatment
排除标准
- •presence of a gastrointestinal disorder (eg, malabsorption) that might jeopardize intestinal absorption of study drug
- •inability to perform seated SVC
- •known immune compromising illness or treatment
- •drug abuse or alcoholism
- •clinically significant or active cardiovascular disease
- •acute or chronic infection
- •diagnosis of malignancy within 2 years prior to screening
- •dementia that may affect patient understanding and/or compliance with study requirements and procedures
- •current use of strong inducers or inhibitors of CYPs 2C19 and 2D6
- •current us of medications (other than riluzole) that are metabolized predominantly by CYP 1A2
研究组 & 干预措施
Cu(II)ATSM
Cu(II)ATSM Powder for Oral Suspension, 36 mg, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day.
干预措施: Cu(II)ATSM (Drug)
Placebo Powder for Oral Suspension
Placebo Powder for Oral Suspension, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day.
干预措施: Placebos (Drug)
结局指标
主要结局
Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
时间窗: 24 weeks
assessment of cognitive function
Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])
时间窗: 24 weeks
assessment of disease severity
次要结局
- rate of adverse events(24 weeks)
- seated slow vital capacity (SVC)(24 weeks)