HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 161
- 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 E will evaluate the safety and efficacy of a single study drug, SLS-005 (Trehalose injection, 90.5 mg/mL for intravenous infusion) 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 E SLS-005 - Trehalose, the participant will complete a screening visit to assess additional Regimen E eligibility criteria. Once Regimen E eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active SLS-005 or matching placebo.
Regimen E will enroll by invitation, as participants may not choose to enroll in Regimen E. 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 E.
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).
- •Current diagnosis or healthcare professional-recommended treatment (medication, exercise or diet) of diabetes mellitus
- •Master Protocol screening glucose >140 mg/dl
- •Prior treatment with IV trehalose or known hypersensitivity to trehalose
- •Current use of oral trehalose (see prohibited medication Section 5.9)
- •Inability for participant to return to site for weekly drug administration, until approved for home infusions
研究组 & 干预措施
SLS-005
SLS-005 is administered via infusion once weekly for 24 weeks.
干预措施: SLS-005 (Drug)
Matching Placebo
Matching placebo is administered via infusion once weekly for 24 weeks.
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: 24 Weeks
Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). 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.
Mortality Event Rate
时间窗: Baseline to 24 Weeks
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. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
次要结局
- Respiratory Function(Baseline to 24 Weeks)
- Muscle Strength(Baseline to 24 Weeks)
- Number of Participants That Experienced Death or Death Equivalent(Baseline to 24 Weeks)
研究者
Merit E. Cudkowicz, MD
Chief, Neurology Department
Massachusetts General Hospital