A Multicenter, Double Blind, Placebo Controlled Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Mallinckrodt
- 入组人数
- 143
- 试验地点
- 69
- 主要终点
- Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
研究概览
简要总结
About 213 people with ALS will participate in this study. There will be locations in North and South America.
During the first part, participants will be randomly assigned to a group (like by flipping a coin). Out of every 3:
- 2 will get the study drug
- 1 will get a look-alike with no drug in it (placebo)
During the second part, everyone will get the study drug.
Participation will help doctors find out if Acthar can help or slow down the symptoms of ALS better than placebo.
详细描述
This is a multicenter, multiple dose study to examine the effect of Acthar on functional decline in adult participants with ALS. Approximately 213 participants will be enrolled.
Following a screening period of up to 28 days, participants with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ 2 years prior to the Screening Visit will be randomized on a 2:1 basis to receive subcutaneous (SC) Acthar 0.2 mL (16 Units [U]) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3-week taper.
Participants who complete the 36 week double-blind treatment period are eligible to enter an Open Label Extension phase in which all participants will receive Acthar 0.2 mL (16 U) daily.
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
- Double (Participant, Investigator)
盲法说明
The Treatment Period is defined as Randomized (2:1, Arm A: Arm B), Double-blind (with care provider and outcomes assessor also blinded).
The Extension Period has no masking, because all who participate receive open label study drug.
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is 18-75 years of age at Screening
- •Has ALS symptom onset within 2 years prior to Screening
- •Has forced vital capacity (FVC) no higher than 60% at screening
- •If taking riluzole, is on a stable dose for 4 weeks before Screening
排除标准
- •Has tracheostomy, diaphragm pacing, or an ongoing need for assisted ventilation of any type
- •Has used any medication within a time period not allowed per protocol
- •Has history of Type 1 or Type 2 diabetes mellitus, or any clinically significant infection
- •Used edaravone less than 1 week before Screening
- •Received any stem cell replacement therapy
- •Used steroids within a time period not allowed per protocol
研究组 & 干预措施
Arm A: Treatment Period Acthar
Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar), daily for up to 36 weeks.
Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.
干预措施: Acthar (Drug)
Arm B: Treatment Period Placebo
Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (Matching Placebo), daily for up to 36 weeks.
Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.
干预措施: Placebo (Drug)
Arm C: Extension Period Acthar-Acthar
Participants who receive Acthar during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
干预措施: Acthar (Drug)
Arm D: Extension Period Placebo-Acthar
Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
干预措施: Acthar (Drug)
Arm D: Extension Period Placebo-Acthar
Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
干预措施: Placebo (Drug)
结局指标
主要结局
Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
时间窗: Baseline, Week 36
The ALSFRS-R is a validated questionnaire-based scale used extensively as a primary outcome measure in ALS clinical trials and is considered a predictor of survival. ALSFRS-R is a 12-item scale that measures 4 domains relevant for ALS (gross motor, fine motor, bulbar and respiratory) A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.
Number of Participants Experiencing an Adverse Event During the Treatment Period
时间窗: by the end of the treatment period (within 36 Weeks)
Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study Clinically significant changes in safety measures are recorded as adverse events.
Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period
时间窗: by the time of database lock (within 84 weeks)
Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events.
次要结局
- Treatment Period: Spirometry (%)(Baseline, Week 36)
- Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R(Baseline, Week 36)
- Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R(Baseline, Week 84)