PLEO-CMT-EXT
Research type
Research Study
Full title
International, multi-center, double blind 9-month follow-up extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A
IRAS ID
219114
Contact name
Mark Roberts
Contact email
Sponsor organisation
PHARNEXT SA
Eudract number
2015-002379-81
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
122505, IND Number
Duration of Study in the UK
1 years, 2 months, 31 days
Research summary
All the participants enrolled and having completed the primary study CLN-PXT3003-02 will be eligible to continue in the extension study if they choose to take part.
If participants consent to take part, those randomised to dose 1 or dose 2 in the primary study will continue in the extension study at the same dose, while those who received placebo will be assigned to one of the two active doses at entry in the extension study. In this way all participants will have the opportunity to benefit from active treatment.
It is possible that participants taking part in this study may find no benefit to them, but the information obtained from this study will help in future to improve the treatment of people with Charcot-Marie-Tooth disease type 1A.
This extension study is designed to offer participants prolonged follow-up, to investigate for up to 2 years the long-term safety and tolerability of PXT3003 (main objective), and to observe the durability of its effect.The Follow-up study will consist of an inclusion visit that will correspond to the last visit (V6) of the previous study CLN-PXT3003-02 and 3 follow-up visits at 3-month, 6-month and 9-month.
Visits will be scheduled as following:
V6 - Inclusion visit: at Day 0 (= 15 months after baseline)
V7 - 3-month visit: at Day 90 ± 10 Days (= 18 months after baseline)
V8 - 6-month visit : at Day 180 ± 10 Days (= 21 months after baseline)
V9 - 9-month visit : at Day 270 ± 10 Days (= 24 months after baseline)Pharnext is sponsor of the study.
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
17/EM/0195
Date of REC Opinion
28 Jul 2017
REC opinion
Further Information Favourable Opinion