NCT02579759

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)

International, Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study Assessing in Parallel Groups the Efficacy and Safety of 2 Doses of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A Treated 15 Months

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 323 (actual)
• Sponsor: Pharnext S.C.A. · Academic / Other
• Sex: All
• Age: 16 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.

Detailed description

PXT3003 is a fixed dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol selected via a Systems Biology approach and developed by Pharnext, with the aim to limit the production of PMP22 and protect/improve axonal function in patients with CMT1A. On September 18th 2017, PXT3003 dose 2 was prematurely discontinued, due to an unexpected investigational medicinal product quality event (failed month 18 stability testing). This resulted in a large proportion of missing data that led us to reconsider the efficacy analysis that was initially planned in the protocol.The independent data safety monitoring committee did not identify any safety concern on September 5th 2017. All patients randomised to dose 2 were requested to undergo the end of study visit, and were offered to enter the extension study (CLN-PXT3003-03).

Conditions

• Charcot-Marie-Tooth Disease Type 1A

Interventions

Type	Name	Description
DRUG	PXT3003 dose 1	Liquid oral solution, 5 ml twice a day, morning and evening with food
DRUG	PXT3003 dose 2	Liquid oral solution, 5 ml twice a day, morning and evening with food
DRUG	placebo	Liquid oral solution, 5 ml twice a day, morning and evening with food

Timeline

• Start date: 2015-12-01
• Primary completion: 2018-03-01
• Completion: 2018-08-01
• First posted: 2015-10-20
• Last updated: 2020-02-27
• Results posted: 2020-02-27

Locations

30 sites across 8 countries: United States, Belgium, Canada, France, Germany, Netherlands, Spain, United Kingdom