International, multi-center, open-label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

Pharnext0 个研究点目标入组 290 人开始时间: 2017年1月19日最近更新: 2017年10月30日

适应症

试验速览

暂无简介。

•Male or female patients with all of the following inclusion criteria will be considered for randomization:
•1.Patient previously randomized to the initial PLEO-CMT study (protocol CLN-PXT3003-02) and having completed 15 months of double-blind treatment in that study, including all procedures required at the Study Termination visit (V6) or patient under prematurely discontinued from CLN-PXT-3003-02 (due to sponsor decision on September 18th, 2017) must have performed an early V6.
•2. Patients who completed V6 assessments within the 4 weeks prior extension study or patients who have completed a new baseline visit (VB) if the V6 assessments were not completed in the 4 weeks prior study entry.
•3. Patient providing a signed written informed consent to participate in the extension study and willing and able to comply with all study procedures and scheduled visits. All minor patients who were included in the primary study will have reached their majority, so only adult patients will be included in the extension study.
•4. Female patients must agree to continue using an approved method of birth control throughout the extension 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 285
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 5

•Patients with any of the following criteria will be excluded from entry:
•1.Presenting any clinically significant change in health status that, in the opinion of the Investigator, would prevent the subject from participating in this study or successfully completing this study.
•2.Any unauthorized concomitant treatments, in the 4 weeks preceding study entry, as study CLN-PXT3003-02.

发起方