Pharnext completes patient enrolment in Phase lll trial of PXT3003 to treat CMT1A
French biopharmaceutical company Pharnext has completed the enrolment of patients for its new Phase lll PLEO-CMT trial of PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A).
Developed using Pharnext’s research and development (R&D) platform Pleotherapy, PXT3003 is a oral fixed-low dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol.
A total of 323 patients with mild-to-moderate CMT1A based in 30 sites across Europe, the US and Canada were enrolled in the trial, which began in December last year.
The enrolled patients have already been randomised to receive either placebo or one of two doses of PXT3003.
PLEO-CMT is a multi-centre, randomised, double blind, placebo-controlled, three-arm Phase lll study, which will compare in parallel groups the efficacy and safety of two orally administered doses of PXT3003 to placebo over a period of 15 months.
Pharnext chief medical officer René Goedkoop said: “Completing enrolment of our pivotal Phase 3 trial of PXT3003 is a significant milestone and highlights the strength of our clinical operations and management teams, as well as the support of our numerous clinical trial sites, our dedicated clinical investigators and the patient community.”
The primary endpoint of the PLEO-CMT study is to assess the change in the overall neuropathy limitation scale (ONLS) score at 12 and 15 months of treatment to measure improvement of patients’ disability with PXT3003.
Pharnext will also evaluate the additional secondary outcome measures, which includes functional and electrophysiological endpoints.
The company is further planning to carry out a nine month follow-up study, where all patients who will have completed the first 15 months will receive the active PXT3003 dose.
Results of the PLEO-CMT trial are scheduled to arrive by the second half of 2018.