The pharmaceutical company Pharnext is on track to start its phase III clinical trial, PREMIER in the USA, by the end of March 2021.
The PREMIER study will test the safety and efficacy of the drug PXT3003 for the treatment of people with Charcot Marie Tooth disease type 1A (CMT1A). PXT3003 is a combination of three different drugs and aims to reduce the levels of the PMP22 protein, which is increased in people with CMT1A. The trial plans to enrol approximately 350 people with CMT1A across 50 centres worldwide. In the study participants will be randomly assigned to receive either PXT3003 or a placebo.
Data from an earlier phase III trial, called PLEO-CMT, suggests that PXT3003 is safe and effective for the treatment of people with CMT1A. However, this trial experienced manufacturing problems and treatment interruptions, resulting in missing data. This has led to the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) requesting an additional trial to test the safety and efficacy of PXT3003 for the treatment of CMT1A. This will be tested in the PREMIER study.
Pharnext is also continuing its ongoing Phase 3 extension study, CLN-PXT3003-03. This is an open label study for participants who had been enrolled in the PLEO-CMT trial. The extension study has followed people with CMT1A, who continue to receive the treatment, for over two years. This is to test how effective the treatment is over a longer period of time as well as its safety and tolerability.
If you have any research questions, please contact our Research Line on firstname.lastname@example.org