An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 616
- 试验地点
- 80
- 主要终点
- Number of Subjects Who Reported an Adverse Event
研究概览
简要总结
The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
详细描述
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, degenerative disease of motor neurons in the brain and spinal cord that leads to muscle atrophy and spasticity in limb and bulbar muscles resulting in weakness and loss of ambulation, oropharyngeal dysfunction, weight loss, and ultimately respiratory failure. The purpose of this study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has the ability to understand the purpose and risks of the study and provide signed and dated informed consent (or have the consent confirmed by a witness if unable to write) and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
- •Subject was enrolled in either CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
- •Subject has completed their last visit in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
- •Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.
排除标准
- •Subject withdrew prematurely from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
- •Subject permanently discontinued study treatment in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) for any reason other than enrollment into this study.
- •Subject from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) has a significant change in medical history (including laboratory tests or a clinically significant condition) that in the opinion of the Investigator would impair the subject's medical fitness for participation and preclude treatment.
- •Female subject who is pregnant or breastfeeding.
- •Subject is currently enrolled in any investigational drug study other than Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
- •Subject is taking pramipexole, other dopamine agonists, any other agent with dopaminergic activity, or any other disallowed concomitant medication.
- •Subject is unwilling or unable to comply with the requirements of the protocol including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to comply with the protocol. At a minimum, subjects who are not able to travel to the study site must be willing to agree to remote blood draws for clinical laboratory evaluations and telephone visits to report Adverse Events, concomitant medications, and Amyotrophic Lateral Sclerosis Functional Rating Scale (revised) (ALSFRS-R) scores.
研究组 & 干预措施
Dexpramipexole
Dexpramipexole open-label
干预措施: Dexpramipexole (Drug)
结局指标
主要结局
Number of Subjects Who Reported an Adverse Event
时间窗: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
The number of subjects who reported an adverse event during the study
Number of Subjects Who Experienced a Serious Adverse Event
时间窗: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
The number of subjects enrolled who reported a serious adverse event during the study
Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event
时间窗: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study
Number of Participants With Potentially Clinically Significant Hematology Results
时间窗: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants with Potentially Clinically Significant Hematology Results. 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 Blood Chemistry Results
时间窗: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants with Potentially Clinically Significant Blood Chemistry Results. 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 ECG Results
时间窗: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants with Potentially Clinically Significant ECG Results. 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 Vital Sign Results
时间窗: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. 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.
次要结局
- Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study(Up to maximum 226 days: approximately 32.2 weeks)
- Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study(Up to maximum 226 days: approximately 32.2 weeks)
- Death up to 6 Months(6 Months)
- Percentage of Participants With Death or Death Equivalent up to 6 Months(6 months)