An ongoing Phase 3 clinical trial program is investigating the drug PXT3003 as an experimental treatment for Charcot-Marie-Tooth disease type 1A in adults. Pharnext SA says that they expect to be able to provide top-line results from the first part of the trial around October 2018. The full article can be read here, at Business Wire.
About Charcot-Marie-Tooth Disease Type 1A (CMT1A)
CMT1A is a type of Charcot-Marie-Tooth disease (CMT). CMT is a group of inherited conditions that affect a person’s peripheral nerves. Peripheral nerves are involved in controlling muscles and connecting sensory information, such as what objects feel like, to the brain.
Patients with the CMT1A form of CMT typically experience damage to the peripheral nerves, and may also have symptoms such as wasting of the lower leg muscles, sensory loss, and hand weakness, amongst others.
CMT1A is caused by an alteration to the PMP22 gene that causes an extra copy of it. According to the original article on Business Wire, an estimated 125,000 people in the US and Europe are affected by CMT1A.
About PleodrugTM PXT3003
PXT3003 is a combination of naltrexone, baclofen, and sorbitol, which is being investigated as an experimental treatment for adults with CMT1A. It has been granted Orphan Drug Designation in the US and Europe.
A Phase 2 clinical trial of PXT3003 has produced promising results for its effectiveness as a treatment for CMT1A, and, so far, it appears to be safe. An ongoing Phase 3 trial is investigating the drug further.
The Phase 3 Clinical Program
The Phase 3 clinical program comprises of two studies: PLEO-CMT and PLEO-CMT-FU.
PLEO-CMT is a pivotal, randomised, double-blind Phase 3 trial that is taking place at multiple locations over 15 months. The effects of the drug are being compared to a placebo group, and patients, who have mild to moderate CMT1A, are being assessed using ONLS scoring, amongst other measures. The top-line results of this study are expected to be released around October 2018.
The second study, PLEO-CMT-FU, is an extension study open to patients who completed the PLEO-CMT trial. Its aim is to learn more about the long-term safety and tolerability of the drug.
