An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- Biogen
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Number of Participants with Adverse Events (AEs)
研究概览
简要总结
The primary objective is to evaluate the long-term safety and tolerability of BIIB078 in participants with chromosome 9 open reading frame 72-amyotrophic lateral sclerosis (C9ORF72-ALS).
The secondary objective is to evaluate the pharmacokinectic (PK) of BIIB078 in participants with C9ORF72-ALS.
详细描述
This study is an extension study of NCT03626012.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
盲法说明
During the blinded loading period, the following individuals will be masked:
- Investigator
- Study staff
- Participants
After the loading period has been completed, subsequent doses will be open-label.
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have completed study NCT03626012 through the first follow-up clinic visit that follows the final dosing visit without missing more than 1 dose of study treatment.
- •Participants taking concomitant riluzole at study entry must be on a stable dose for ≥30 days prior to the first dose of study treatment (Day 1). Participants taking concomitant riluzole must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that riluzole should be discontinued for medical reasons, in which case it may not be restarted during the study.
- •Participants taking concomitant edaravone at study entry must be on a stable dose for ≥60 days prior to the first dose of study treatment (Day 1). Participants taking concomitant edaravone must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.
排除标准
- •History of drug abuse or alcoholism ≤6 months before study enrollment that would limit participation in the study, as determined by the Investigator.
- •Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.
- •History of or positive test result at Screening for human immunodeficiency virus. The requirement for testing at Screening may be omitted if it is not permitted by local regulations.
- •Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs) or biological agent within 1 month of Screening or 5 half-lives of study agent, whichever is longer.
- •Note: Other protocol-specific inclusion/exclusion criteria may apply.
研究组 & 干预措施
Cohort A: BIIB078 First Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
干预措施: BIIB078 (Drug)
Cohort B: BIIB078 Second Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
干预措施: BIIB078 (Drug)
Cohort C: BIIB078 Third Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
干预措施: BIIB078 (Drug)
Possible Cohort D: BIIB078 Fourth Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
干预措施: BIIB078 (Drug)
结局指标
主要结局
Number of Participants with Adverse Events (AEs)
时间窗: Baseline up to Day 785
AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.
Number of Participants with Serious Adverse Events (SAEs)
时间窗: Screening up to Day 785
An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment.
次要结局
- Serum concentration of BIIB078(Baseline and at multiple time points up to Day 729)
- Cerebrospinal Fluid (CSF) concentration of BIIB078(Baseline and at multiple time points up to Day 729)