HEALEY ALS Platform Trial - Regimen I NUZ-001
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 240
- 试验地点
- 1
- 主要终点
- Disease Progression
研究概览
简要总结
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
Regimen I will evaluate the safety and efficacy of a single study drug, NUZ-001, in participants with ALS.
详细描述
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683. Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.
If a participant is randomized to Regimen I NUZ-001, the participant will complete a screening visit to assess additional Regimen I eligibility criteria. Once Regimen I eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active NUZ-001 or matching placebo.
Regimen I will enroll by invitation, as participants may not choose to enroll in Regimen I. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen I.
For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
排除标准
- •The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
- •Clinically significant liver disease (e.g., chronic hepatitis B, untreated hepatitis C)
- •Prior history of drug-induced liver injury (DILI) and/or laboratory results at Master Protocol Screening that indicate inadequate liver function (ALT, AST or alkaline phosphatase >3 times the upper limit of normal [x ULN] and/or total bilirubin level >2 x ULN)
- •a. Note: Elevated total bilirubin attributable to documented Gilbert's syndrome is not considered exclusionary when there is no other evidence of clinically significant hepatic dysfunction.
- •Family history of SOD1 or VCP-associated ALS or known SOD1 or VCP mutation via genetic testing or self-report
- •Use of any prohibited medications as outlined in the Regimen-specific Appendix (RSA) within 30 days prior to Baseline or anticipated use during the study treatment period
- •Participants who are taking Nuedexta® and have a prolonged Fridericia-corrected QT (QTcF) interval (QTcF > 450 ms (males) or > 470 ms (females)) at Master Protocol Screening
研究组 & 干预措施
Matching Placebo
Placebo Comparator: Matching Placebo
干预措施: Matching placebo (Drug)
NUZ-001
NUZ-001
干预措施: NUZ-001 (Drug)
结局指标
主要结局
Disease Progression
时间窗: Baseline to 36 Weeks
Change in disease severity over time as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
次要结局
- Respiratory Function(Baseline to 36 Weeks)
- Survival(Baseline to 36 Weeks)
研究者
Merit E. Cudkowicz, MD
Executive Director, Neurology Department
Massachusetts General Hospital