An Open-Label, Safety and Tolerability, Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 74
- 试验地点
- 19
- 主要终点
- Number of Participants With Potentially Clinically Significant Hematology Results
研究概览
简要总结
This is an open-label, multi-center study designed to extend the evaluation of the safety, tolerability, and clinical effects of oral administration of KNS-760704 in patients with ALS.
详细描述
Patients who complete the Part 2 Week 28 visit in study KNS-760704-CL201 and patients with ALS who were actively receiving RTPB [(6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydro-chloride monohydrate] under Research IND #60,948 were eligible to participate in this study.
Eligible patients received 1 tablet of KNS-760704 (150 mg) every 12 hours (Q12H) (300 mg total daily dose) for up to 180 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient has provided signed informed consent for this trial before the commencement of any study-related procedure
- •Patient is actively participating in Knopp Protocol KNS-760704-CL201 and has completed the Part 2 Week 28 visit in that study or patient is actively receiving RTPB under Research IND #60,948
排除标准
- •Patient did not participate in Knopp Protocol KNS-760704-CL201 or patient did not receive RTPB under Research IND #60,
- •Patient discontinued from KNS-760704-CL201 for any reason other than enrollment into this study (applies to patients enrolled in KNS-760704-CL201 only)
- •Patient was not taking RTPB under Research IND #60,948 prior to April 30, 2009 (applies to patients enrolled under Research IND #60,948 only)
研究组 & 干预措施
KNS-760704 300 mg/day
Open-label KNS-760704 (150 mg Q12H)
干预措施: KNS-760704 (Drug)
结局指标
主要结局
Number of Participants With Potentially Clinically Significant Hematology Results
时间窗: 180 weeks
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
时间窗: 180 weeks
Number of participants with potentially clinically significant liver enzyme abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.
Number of Participants With Potentially Clinically ECG Abnormalities
时间窗: 180 weeks
Number of participants with potentially clinically significant ECG abnormalities for the safety population are presented. Percentages are based on the number of patients in the safety population who had at least one non-missing, post-baseline value.
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
时间窗: 180 weeks
Number of participants with potentially clinically significant vital sign abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.
次要结局
- Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12(12 weeks)
- Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24(24 weeks)
- Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48(48 weeks)
- Change in Upright Vital Capacity From Baseline to Week 12(12 weeks)
- Change in Upright Vital Capacity From Baseline to Week 24(24 weeks)
- Change in Upright Vital Capacity From Baseline to Week 48(48 weeks)
- Change in McGill Single-Item Scale (SIS) From Baseline to Week 12(12 weeks)
- Change in McGill Single-Item Scale (SIS) From Baseline to Week 24(24 weeks)
- Change in McGill Single-Item Scale (SIS) From Baseline to Week 48(48 weeks)
- Number of Subjects With Feeding Tube Placed During the Study.(144 weeks)