NCT05178810已完成3 期
A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 300
- 试验地点
- 38
- 主要终点
- Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score After 48 Weeks.
研究概览
简要总结
Multicenter, multinational, double-blind, randomized (2:1), placebo-controlled Phase III study to investigate the efficacy and safety of 100 mg FAB122 once daily as oral formulation in ALS patients.
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
- •Age 18 - 80 years (both inclusive), male or female;
- •Diagnosis of definite, probable, probable laboratory supported or possible ALS as based on the El Escorial and the revised Airlie House diagnostic criteria for ALS;
- •Onset of first symptoms* no longer than 24 months prior to randomization;
- •*Date of onset is the date the patient reported one or more of the following symptoms:
- •Muscle weakness in limbs
- •Speech/swallowing difficulties
- •Respiratory symptoms: dyspnea was noticed
- •SVC equal to or more than 70% of the predicted normal value for gender, height and age at screening visit;
- •Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit;
- •Capable of providing informed consent and complying with trial procedures.
排除标准
- •Diagnosis of Primary Lateral Sclerosis;
- •Diagnosis of Frontotemporal Dementia;
- •Diagnosis of other neurodegenerative diseases (e.g. Parkinson disease, Alzheimer disease);
- •Diagnosis of polyneuropathy;
- •Other causes of neuromuscular weakness;
- •Have a significant pulmonary disorder not attributed to ALS and/or require treatment interfering with the evaluation of ALS on respiratory function;
- •Use of intravenous (IV) edaravone within 6 months of the screening visit;
- •Depend on mechanical ventilation (invasive or non-invasive) or require tracheostomy at Screening;
- •Renal impairment as indicated by a creatinine clearance of less than 50 mL/min as calculated by the Cockcroft Gault equation;
- •Subject has a history of clinically significant hepatic disease, hepatitis or biliary tract disease, or subject has a positive screening test for HIV, hepatitis B or C;
研究组 & 干预措施
FAB122
Experimental
干预措施: FAB122 (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score After 48 Weeks.
时间窗: 48 weeks
Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.
次要结局
- Overall Survival: Proportion of Subjects Alive (Survival Rate) After 24, 48 and 72* Weeks;(24, 48 and 72* weeks;)
- Proportion of Subjects Alive and no Tracheostomy, or no Initiation of Non-invasive Ventilation for More Than 20 Hours a Day for More Than 10 Consecutive Days After 24, 48 and 72* Weeks(24, 48 and 72* weeks)
- Change From Baseline in Slow Vital Capacity (SVC, Liters) at 24, 48 and 72* Weeks;(24, 48 and 72* weeks)
- Change From Baseline in the Overall Mega Score for the Hand-held Dynamometer (HHD) at 24, 48 and 72* Weeks.(24, 48 and 72* weeks)
- Change From Baseline in the Total Score on the ALS Assessment Questionnaire-40-Item (ALSAQ-40) Form at 24, 48 and 72* Weeks;(24, 48 and 72* weeks)
- Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks.(24, 48 and 72* weeks)
- Change From Baseline in Visual Analogue Scale (VAS) Score at 24, 48 and 72* Weeks.(24, 48 and 72* weeks)
- Proportion of Subjects With a Change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and Behavioural ALS Screen) Total Score;(72 weeks)
- Combined Assessment of Function and Survival (CAFS) at 48 and 72 Weeks.(48 weeks and 72 weeks)
- Survival Probability(72 weeks)
- Change From Baseline in ALSFRS-R Score After 24 and 72* Weeks(24 weeks, 72 weeks)
- The Slope of the Decrease in ALSFRS-R Score Over Time at 24, 48 and 72* Weeks;(24, 48, 72 weeks)
- Change From Baseline in ALSFRS-R Score on Bulbar Function (Question 1-3 of the ALSFRS-R) After 24, 48 and 72* Weeks;(24, 48 and 72 weeks)
- Change From Baseline ALS Functional Rating Scale - Revised Score - Fine Motor Function(24, 48 and 72 weeks)
- Change From Baseline ALS Functional Rating Scale - Revised Score - Gross Motor Function(24, 48, 72 weeks)
- Change From Baseline ALS Functional Rating Scale - Revised Score - Respiratory Function(24, 48, 72 weeks)
- Time to a 3, 6, 9 and 12 Points Change or Death From Baseline in ALSFRS-R Score Over 72* Weeks;(72 weeks (+/-1 week visit window))
- Change in Clinical Staging (King's Staging System and MiToS) Over 72 Weeks(72 weeks)
研究者
研究点 (38)
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