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.
Phase of Trial: Phase III
Latest Information Update: 31 Oct 2017
At a glance
- Drugs Baclofen/naltrexone/sorbitol (Primary)
- Indications Charcot-Marie-Tooth disease
- Focus Adverse reactions
- Acronyms PLEO-CMT-FU
- Sponsors Pharnext
- 28 Oct 2017 Study design has been changed from double blind to open label.
- 18 Sep 2017 According to a Pharnext media release, the data from PLEO-CMT trial will form the basis of the submission package for market approval in the first quarter of 2019. Long-term safety data from PLEO-CMT-FU would then be submitted to regulatory authorities during their review of the marketing authorization application.
- 18 Sep 2017 According to a Pharnext media release, the company has decided to switch high dose patients patients to receive double the amount of dose 1 (2 x 5 mL) in the 9-month open label extension study (PLEO-CMT-FU). Patients from the placebo and dose 1 arms in the 15-month double blind PLEO-CMT study will continue the Phase 3 clinical trial as planned: then, these patients will have the opportunity to continue treatment with PXT3003 in the PLEO-CMT-FU extension study for 9 months.