An Exploratory, Randomized, Double-Blind, Crossover Study to Compare the Efficacy and Safety of BIIB074 Versus Placebo in the Treatment of Primary Inherited Erythromelalgia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Biogen
- 入组人数
- 8
- 试验地点
- 2
- 主要终点
- Weekly average severity of paroxysms
研究概览
简要总结
The primary objective of the study is to investigate the efficacy of repeat oral dosing of BIIB074 on paroxysmal pain in participants with Primary Inherited Erythromelalgia (EM). The secondary objective of the study is to investigate the efficacy of repeat oral dosing of BIIB074 on varying additional aspects of pain in participants with EM; and to investigate the safety and tolerability of repeat oral dosing of BIIB074 in participants with EM.
详细描述
This study was previously posted by Convergence Pharmaceuticals, Ltd., which has been acquired by Biogen.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
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
- •A diagnosis of primary inherited EM with family history of EM made at least 3 months from initial diagnosis.
- •Failed at least one prior treatment for EM (defined as an inadequate response or intolerance to treatment).
- •Approved concomitant medications must have been stable for at least 4 weeks prior to day
排除标准
- •Positive screening Hepatitis B surface antigen or positive Hepatitis C antibody result.
- •Received nerve blocks and/or steroid injections for neuropathic pain within 4 weeks prior to Day
- •Males whose partner is pregnant.
- •Failed at least one prior treatment for EM (defined as an inadequate response or intolerance to treatment).
- •NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
研究组 & 干预措施
Randomized Group 1
After two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period
干预措施: BIIB074 (Drug)
Randomized Group 1
After two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period
干预措施: Placebo (Drug)
Randomized Group 2
After two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period
干预措施: BIIB074 (Drug)
Randomized Group 2
After two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period
干预措施: Placebo (Drug)
结局指标
主要结局
Weekly average severity of paroxysms
时间窗: Day 1 to Week 12
11-point Pain Intensity Numerical Rating Scale (PI-NRS) is used to assess EM paroxysmal pain. PI-NRS is an 11-point pain intensity numerical rating scale, where 0=no pain and 10=worst possible pain. Weekly average is defined as the total of severity scores during a week divided by the total number of paroxysms during that week.
次要结局
- Weekly average and weekly maximum duration of paroxysms(Day 1 to Week 12)
- Patient Global Impression of Change (PGIC) score(Day 1 to Week 12)
- Columbia-Suicide Severity Rating Scale (C-SSRS) assessment(Up to Week 13)
- Number of participants with clinically significant vital sign abnormalities(Up to Week 13)
- Weekly average and maximum number of paroxysms(Day 1 to Week 12)
- Weekly maximum severity of paroxysms(Day 1 to Week 12)
- Weekly average and weekly maximum of daily background pain(Day 1 to Week 12)
- Weekly average and weekly maximum number of pain-mitigating activities(Day 1 to Week 12)
- Weekly average and weekly maximum duration of pain-mitigating activities(Day 1 to Week 12)
- Use of rescue medication(Day 1 to Week 13)
- Weekly average and weekly maximum of the daily sleep interference scale(Day 1 to Week 12)
- Weekly average and weekly maximum number of awakenings at night due to EM pain(Day 1 to Week 12)
- Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs)(Up to Week 13)
- Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities(Up to Week 13)
- Number of participants with clinically significant laboratory safety test abnormalities(Up to Week 13)