An Open-label, Non-randomized Trial to Investigate the Efficacy and Safety of Early Versus Delayed Start of Arimoclomol in Patients With Sporadic Inclusion Body Myositis Who Have Completed the IBM4809 Trial
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 121
- 试验地点
- 12
- 主要终点
- Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
研究概览
简要总结
A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)
详细描述
This was planned to be a 40-month open-label extension trial of the 20-month randomized, double-blind, placebo-controlled IBM4809 trial. The open-label trial was terminated early by the sponsor as a consequence of the results of IBM4809 which did not meet any of its efficacy endpoints. Therefore, the actual average duration of open-label treatment was approximately 28 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 45 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
- •Patient has completed the IBM4809 trial on treatment with Investigational Medicinal Product (IMP).
排除标准
- •Known or suspected allergy or intolerance to arimoclomol or its constituents.
- •Exposure to any other investigational treatment within 30 days or <5 half-lives of the baseline visit or taking part or planning to take part in another interventional trial.
- •Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
- •Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants up to 3 months after last dose. Premenopausal women must have a negative pregnancy test prior to dosing with trial medication. Acceptable methods of birth control are:
- •Hormonal methods associated with inhibition of ovulation such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the patient's usual menstrual cycle period) before arimoclomol administration.
- •Total abstinence from sexual intercourse since the last menses before arimoclomol administration. (The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence [calendar, symptothermal, post-ovulation] methods are not acceptable methods of contraception).
- •Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
- •Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
- •Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.
研究组 & 干预措施
Arimoclomol
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
干预措施: Arimoclomol (Drug)
结局指标
主要结局
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
时间窗: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
The Inclusion Body Myositis Functional Rating Scale (IBMFRS) includes 10 measures (swallowing, handwriting, cutting food and handling utensils, fine motor tasks, dressing, hygiene, turning in bed and adjusting covers, changing position from sitting to standing, walking, and climbing stairs), each graded on a Likert scale from 0 (being unable to perform) to 4 (normal). The sum of the 10 items gives a value between 0 and 40. A higher score represents less functional limitation. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.
次要结局
- Change in Modified Timed Up and Go (mTUG)(Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).)
- Change in Hand Grip Strength(Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).)
- Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD)(Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).)
- Change in the 36-Item Short Form Health Survey (SF-36)(Change from Baseline in IBM-OLE to Early Termination Visit)
- Change in Quadriceps Muscle Strength(Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).)
- Number of Falls and Near Falls(Baseline to Early Termination Visit)