PXT3003 in CMT1A : the PREMIER clinical trial starts in France

The first French investigation center for the PREMIER trial has just opened: this is the CHU de la Timone in Marseille, which has just recruited the first European participant. Other French centers should also participate in this international phase III trial which started last March in the United States and will take place in Europe, Canada and Israel. 


It plans to include 350 participants with CMT1A to assess the safety and effectiveness of PXT3003, a combination of baclofen, naltrexone and sorbitol that aims to improve peripheral nerve abnormalities.


This trial follows another Phase III trial, the PLEO-CMT trial, which tested PXT 3003 in 235 participants with CMT1A, with encouraging results but was troubled by problems with the stability of PXT3003. Since then, the manufacture and packaging of the product has been improved in preparation for the PREMIER trial.


Pharnext Announces First Patient Enrolled in Europe in the PREMIER Trial, its Pivotal Phase III Clinical Study of PXT3003 in Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’). Pharnext press release (July 12, 2021)


PREMIER on Clinicaltrials.gov: NCT04762758