A Randomized Placebo-controlled Trial of Nicotinamide/Pterostilbene Supplement in ALS: The NO-ALS Study
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 380
- 试验地点
- 9
- 主要终点
- Disease progression as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
研究概览
简要总结
Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is 3 years. Apart from Riluzole, there is no effective treatment. Care of advanced ALS will have a cost of 4-8 million NOK per year
Research i.a. from the investigators department has shown that increased activity in histone deacetylation enzymes (sirtuins) together with increased access to NAD can delay disease progression. Nicotinamide riboside (NR) can increase cells' access to NAD and Pterostilben will stimulate sirtuins.
The investigators want to study whether combination therapy with NR and Pterostilben can inhibit neurodegeneration in ALS and thereby delay disease development, increase survival and improve quality of life in ALS.
In the study, the investigators will use 2 different dosages on the active treatment and strength calculations show that 180 patients are needed to show a rather weak effect. Patients will be recruited in collaboration with hospitals in Helse Vest, AHUS, Drammen, OUS and St. Olavs hospital.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 35 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Arm 1 (newly diagnosed ALS patients)
- •Have a clinical diagnosis of probable ALS according to the revised El Escorial criteria.
- •MR of the brain and cervical spine cannot explain symptoms.
- •Diagnosed with likely ALS within 6 months from enrolment and treated with Riluzole 50mg x 2
- •Symptom onset no longer than 2 year prior to inclusion.
- •ALS-FRC-R of 36 or more (not any item below 2).
- •Age equal to or greater than 35 years at time of enrollment
- •Arm 2 (earlier diagnosed ALS patients)
- •Have a clinical diagnosis of probable ALS according to the revised El Escorial criteria.
- •MR of the brain and cervical spine cannot explain symptoms.
- •Treated with Riluzole 50mg x 2.
排除标准
- •Dementia, FTD or other neurodegenerative disorder at baseline visit
- •Any psychiatric disorder that would interfere with compliance in the study.
- •Use of high dose vitamin B3 supplementation within 30 days of enrollment
- •Metabolic, neoplastic, or other physically or mentally debilitating disorder at baseline visit.
- •Genetically confirmed mitochondrial disease
- •Patients who become tracheostomized as part of the treatment of ALS
- •Patients with short expected survival at the discretion of the investigator. Such cases cannot be expected to follow protocol procedures.
研究组 & 干预措施
Newly diagnosed ALS patients
- High dose EH301 (1500mg Nicotinamide riboside / 300mg Pterostilbene)
- Single dose EH301 (1000mg Nicotinamide riboside / 200mg Pterostilbene)
- Placebo
干预措施: EH301 (Nicotinamide Riboside/Pterostilbene) (Dietary Supplement)
Earlier diagnosed ALS patients
- High dose EH301 (1500mg Nicotinamide riboside / 300mg Pterostilbene)
- Placebo
干预措施: EH301 (Nicotinamide Riboside/Pterostilbene) (Dietary Supplement)
结局指标
主要结局
Disease progression as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
时间窗: Change from baseline to 1 year
ALSFRS-R is a validated rating instrument for monitoring the progression of disability in patients with ALS. The minimum score is 0 and the maximum score is 48, the higher score the more function is retained.
次要结局
- Change in cognitive functions as assessed by the Edinburgh Cognitive Scale (ECAS)(Change from baseline to 1 year)
- Change in vital capacity(Change from baseline to 1 year)
- Change of Neurofilament light chain (NFL) levels in serum(Change from baseline to 1 year)
- Change in quality of life as assessed by the quality of life questionnaire SF-36(Change from baseline to 1 year)