NCT02967679已完成2 期
SERENDEM Study: MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
MedDay Pharmaceuticals SA1 个研究点 分布在 1 个国家目标入组 15 人开始时间: 2016年12月5日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Length of Motor Nerve Potential
研究概览
简要总结
The single-center, open-label Phase II study has the objective of assess the effect of MD1003 on motor and sensory conduction in patients suffering from demyelinating polyneuropathies in 15 subjects.
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
- 20 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female aged between 20 and 85 years.
- •Patients fulfilling one of the following diagnosis:
- •Five patients with chronic inflammatory demyelinating polyneuropathy on both clinical and neurophysiological grounds.
- •Five patients with proven genetic diagnosis of CMT1a or CMT1b
- •Five patients with anti-MAG polyneuropathy.
- •Electrophysiological parameters worsening for the past 3 years
- •Available EMG record, performed during the past 6 months to assess variability of NCV parameters
- •Signed and dated written informed consent to participate in the study in accordance with local regulations
- •Likely to be able to participate in all scheduled evaluation and complete all required study procedures,
- •In the opinion of the investigator, the patient will be compliant and have a high probability of completing the study.
- •Both male and female subjects who are not either surgically sterile (tubal ligation/obstruction or removal of ovaries or uterus) or post-menopausal (no spontaneous menstrual periods for at least one year confirmed by a negative hormone panel) must commit to using TWO highly effective method of birth control for the duration of the study and for two months after the treatment termination.
排除标准
- •Any general chronic handicapping disease other than peripheral neuropathy
- •Impossibility to perform the 10 meters walking test
- •Impossibility to assess electrophysiological parameters
- •Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or cancer,
- •Patients with hypersensitivity to MD1003 excipients (lactose)
- •Laboratory tests out of normal range according to the reference laboratory values. Deviations may be accepted if considered by the investigator as not clinically significant with regards to the study continuation,
- •Patients with history or presence of alcohol abuse or drug addiction,
- •Patients likely to be non-compliant to the study procedures or for whom a long-term follow-up seems to be difficult to achieve.
- •Any new medication for neuropathy initiated less than 3 months prior to inclusion. For CIDP patients, relapse in the past 3 months before inclusion.
- •Not easily contactable by the investigator in case of emergency or not capable to call the investigator
- •Subjects without effective contraception
研究组 & 干预措施
MD1003
Experimental
MD1003 100mg capsules, 1 capsule tid for 48 weeks
干预措施: MD1003 (Drug)
结局指标
主要结局
Length of Motor Nerve Potential
时间窗: 48 weeks
Absolute change from baseline at W48.
Distal Latency (Msec)
时间窗: 48 weeks
Absolute change from baseline at Week 48.
F Wave Latency (Msec)
时间窗: 48 weeks
Absolute change from baseline at Week 48.
Motor Nerve Conduction Velocity (m/Sec)
时间窗: 48 weeks
Absolute change from baseline at Week 48.
次要结局
- ONLS (Overall Neuropathy Limitations Scale)(48 weeks)
- Change From Baseline at Week 48 for Timed 10-meter Walk Test(48 weeks)
- INCAT Sensory Sum Score (ISS)(48 weeks)
- Absolute Change From Baseline at Week 48 for Medical Research Council (MRC) Subscore (Total Muscle) and Total Score(48 weeks)
- Strength-duration Time Constant (ms)(48 weeks)
- Posturography Score(48 weeks)
- 6-minute Walk Test(48 weeks)
- Excitability Testing: Supernormality (%)(48 weeks)
- Rheobase (mA)(48 weeks)
- Refractoriness (%)(48 weeks)
- Minimum Absolute Refractory Period (ms).(48 weeks)
- Maximum Absolute Refractory Period (ms).(Week 48)
研究者
研究点 (1)
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