HEALEY ALS Platform Trial - Regimen G DNL343
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 249
- 试验地点
- 1
- 主要终点
- Disease Progression as Assessed by the ALSFRS-R-Slope
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 G will evaluate the safety and efficacy of a single study drug, DNL343, 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 G DNL343, the participant will complete a screening visit to assess additional Regimen G eligibility criteria. Once Regimen G eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active DNL343 or matching placebo.
Regimen G will enroll by invitation, as participants may not choose to enroll in Regimen G. 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 G.
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 100 Years（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).
- •Diagnosis of epilepsy or seizure within 6 months of randomization
- •Hypersensitivity to DNL343 or any of the excipients contained within the DNL343 drug product
- •The concomitant use of prescription or over-the-counter (OTC) medications that are inducers of certain cytochrome P450 enzymes, substrates of certain cytochrome P450 enzymes, or substrates of certain drug transporters.
研究组 & 干预措施
DNL343
干预措施: DNL343 (Drug)
Matching Placebo
干预措施: Matching Placebo (Drug)
结局指标
主要结局
Disease Progression as Assessed by the ALSFRS-R-Slope
时间窗: Baseline to 24 Weeks
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4(normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Participants with higher scores have more physical function. Note that only participants who survived to their Week 24 visit contribute to the estimate.
Mortality Event Rate
时间窗: Baseline to 24 weeks
The Mortality Rate, presented as mean deaths per month, was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times. Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row.
次要结局
- ALSFRS-R Total Score(Baseline to 24 Weeks)
- Combined Assessment of Function and Survival (CAFS)(Baseline to 24 Weeks)
- Respiratory Function(Baseline to 24 Weeks)
- Upper Limb Muscle Strength(24 weeks)
- Number of Participants With Death or Permanent Assisted Ventilation (PAV)(Baseline to 24 weeks)
- Number of Participants Who Experience Death(Baseline to 24 weeks)
- Disease Progression Biomarker(Baseline to 24 weeks)
研究者
Merit E. Cudkowicz, MD
Chief, Neurology Department
Massachusetts General Hospital