2023-509853-29-00招募中3 期
A randomized, phase II/III trial on the biological and clinical effects of acetyl-L-carnitine in ALS
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 236
- 试验地点
- 15
- 主要终点
- The proportion of participants remaining self-sufficient after 48 weeks in each treatment arm (those scoring three or higher in all the three ALSFRS-R items for swallowing, cutting food, handling utensils, and walking).
研究概览
简要总结
Assess the efficacy of acetyl-L-carnitine (ALCAR) (two different dosages will be tested: 1.5g/day and 3g/day) on the progression of functional disability (loss of self-sufficiency), as measured by the ALSFRS-R scale
研究设计
- 分配方式
- Randomized
- 主要目的
- Treatment
- 盲法
- Double (Analyst, Subject, Monitor, Investigator)
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Intact cognitive function, again determined by the Principal Investigator.
- •ALS diagnosis according to the Gold Coast Criteria
- •Disease duration ≤ 24 months from symptom onset, as indicated by limb weakness or bulbar symptoms, at the randomization/baseline visit
- •Self-sufficiency [Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking)];
- •Satisfactory respiratory function (FVC ≥80% of predicted);
- •Documented progression of symptoms as measured by the ALSFRS-R scale. Disease progression rate (DFS) must be>= 0.
- •DFS =(48- ALSFRS-R at screening)/months from onset to screening;
- •Ability to understand and comply with the study requirements;
- •Ability to give written informed consent personally or, as an alternative, via a legally authorized representative;
- •Treatment with riluzole 50 mg twice/day for at least 4 weeks prior to randomization visit;
排除标准
- •Antecedent polio infection or other active infection;
- •Unwillingness or inability to take riluzole.
- •Motor neuron disease (MND) other than ALS;
- •Involvement of other systems possibly determining a functional impairment (as measured by the endpoints) for the entire duration of the study;
- •Other severe clinical conditions (e.g., cardiovascular disorders, neoplasms) with an impact on survival or functional disability in the next 12 months;
- •Previous use of ALCAR for any reason;
- •Poor compliance with previous treatments;
- •Other experimental treatments in the three months prior to the screening visit (if a subject is receiving another experimental drug, a 3-month wash-out period before participating in the present clinical trial will be required);
- •Women who are lactating or able to become pregnant (e.g. who are not post-menopausal, surgically sterile, or using inadequate birth control) and men unable to practice contraception for the duration of the treatment and three months after its completion;
- •Inability to understand and comply with the study requirements;
结局指标
主要结局
The proportion of participants remaining self-sufficient after 48 weeks in each treatment arm (those scoring three or higher in all the three ALSFRS-R items for swallowing, cutting food, handling utensils, and walking).
The proportion of participants remaining self-sufficient after 48 weeks in each treatment arm (those scoring three or higher in all the three ALSFRS-R items for swallowing, cutting food, handling utensils, and walking).
次要结局
- Mean change of ALSFRS-R total score in each treatment arm from baseline to week 48.
- Mean change of FVC% in each treatment arm from baseline to week 48.
- Mean change in the five domains of ALSAQ-40 measuring different aspects of quality of life (physical mobility, ADL/independence, eating and drinking, emotional reactions, communication) in each treatment arm from baseline to week 48.
- Mean change in ECAS total score in each treatment arm from baseline to week 48.
- Cumulative probability of remaining self-sufficient in each treatment arm during 48 weeks.
- Cumulative probability of remaining free from a 6-point or greater decline in ALSFRS-R total score in each treatment arm during 48 weeks.
- Cumulative probability of remaining without gastrostomy in each treatment arm during 48 weeks.
- Cumulative probability of remaining without non-invasive ventilation (NIV) support (≥12 hours a day in a 24-hour period) in each treatment arm during 48 weeks.
- Cumulative survival probability (of being alive and without tracheostomy) in each treatment arm during 48 weeks
- The mean change in the levels of PGC-1 alpha, 3-NT, acetyl-PPIA in the peripheral blood mononuclear cells (PBMCs) and of NFL, MMP-9, MCP-1, CK, MiR-206, Musclin/osteocrin, Uric acid, HNE in plasma in each treatment arm, during the entire treatment period (from baseline to 48 weeks).
- Number of adverse events and serious adverse events in each treatment arm during 48 weeks of treatment.
研究者
Elisabetta Pupillo
Scientific
Istituto Di Ricerche Farmacologiche Mario Negri
研究点 (15)
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