HEALEY ALS Platform Trial - Regimen D Pridopidine
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 163
- 试验地点
- 1
- 主要终点
- Disease Progression as Assessed by the ALSFRS-R Total Score
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 D will evaluate the safety and efficacy of a single study drug, pridopidine, 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 D Pridopidine, the participant will complete a screening visit to assess additional Regimen D eligibility criteria. Once Regimen D eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active pridopidine or matching placebo.
Regimen D will enroll by invitation, as participants may not choose to enroll in Regimen D. 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 D.
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).
- •Participants with a confirmed prolonged Fridericia-corrected QT (QTcF) interval (defined as a QTcF interval of >450 ms for men and >470 ms for women).
- •Participants with clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, or asymptomatic sustained ventricular tachycardia, or presence of left bundle branch block.
- •Participants with known history of long QT syndrome or a first degree relative with this condition.
- •Participants using prohibited medications within the 4 weeks prior to the Regimen Specific Screening Visit, as detailed in section 5.
- •Participants using the following medications at the time of the Regimen Specific Screening Visit:
- •Nuedexta - at a dosage higher than 20 mg dextromethorphan + 10 mg quinidine BID
- •Citalopram - at a dosage higher than 20 mg/day
- •Escitalopram - at a dosage higher than 10 mg/day
- •Participants with a known allergy to any ingredient of the study intervention (pridopidine, silicified microcrystalline cellulose, and magnesium stearate).
研究组 & 干预措施
Pridopidine
Pridopidine is administered orally twice daily for 24 weeks.
干预措施: Pridopidine (Drug)
Matching Placebo
Matching placebo is administered orally twice daily 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 Total Score
时间窗: Baseline to 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
- Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline(Baseline to 24 Weeks)
- Bulbar Function in All Randomized Participants(Baseline to 24 Weeks)
- Respiratory Function(Baseline to 24 Weeks)
- Bulbar Function in Participants With Rapid Pre-baseline Progression(Baseline to 24 Weeks)
- Time to Bulbar Decline(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