SERENDEM study: MD1003 in patients suffering from demyelinating neuropathies, an open label pilot study

MEDDAY PHARMACEUTICALS0 个研究点目标入组 15 人开始时间: 2016年3月2日最近更新: 2020年10月19日

适应症

试验速览

暂无简介。

•- 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
•- 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.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 10
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 5

•- 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
•- Pregnant woman or of childbearing potential without effective contraception

发起方

MEDDAY PHARMACEUTICALS