CMT1A Clinical Trial Update: Pharnext PREMIER trial of PXT3003

by | Feb 23, 2021 | 0 comments

The PREMIER Trial, which is being conducted in patients with mild-to-moderate CMT1A, is expected to enroll approximately 350 subjects ages 16-65 with a confirmed genetic diagnosis of CMT1A. This International multi-centered pivotal Phase III study will enroll patients at 50 sites worldwide (20 in the US and 5 in Canada) and is set to begin by the end of March 2021. 

The PREMIER Trial is a randomized, double-blind, two-arm placebo controlled study designed to confirm PXT3003 safety and efficacy in patients with CMT1A. Patients enrolled will be treated for 15 months.

As agreed with regulatory agencies, the primary efficacy endpoint is the Overall Neuropathy Limitation Score (ONLS) scale, which measures functional disability. The secondary endpoints will include the following outcome measures: 1) 10-Meter Walk Test (10mWT), 2) Quantified Muscular Testing (bilateral foot dorsiflexion dynamometry), 3) Patient Global Impression of Change (PGI-S), 4) Patient Global Impression of Change (PGI-C), 5) Charcot-Marie-Tooth Neuropathy Score, version 2 (CMTNS-v2), and 6) Quantified Muscular Testing (hand grip). Safety and tolerability will be monitored throughout the study.

For those interested in participation, the US sites will be listed and updated regularly at the website as they are activated and ready to screen and enroll patients. US sites, but also sites in Canada, Europe and Israel, will also be available and listed on a web portal dedicated to patients. This resource will also include interesting information about the trial. It will be live by the end of March 2021.

***Pharnext is also continuing to conduct its ongoing Phase III Extension Study, CLN-PXT3003-03, an open label study that has enrolled 187 patients out of the 323 with mild-to-moderate CMT1A patients that were enrolled in the first double-blind Phase III Trial (PLEO-CMT). As of today, 130 CMT1A patients are still being treated with high-dose PXT3003 and have been followed for more than 2 years in order to assess not only the safety and tolerability of PXT3003, but also its long-term efficacy. Pharnext will be reporting top-line interim data on the safety and efficacy of PXT3003 from this extension study during Q2 2021.

More information about the PREMIER Trial can be found at:


You can also join HNFs patient-registry, GRIN for clinical trials updates.

Learn more on this topic

Related Blog Posts

2024 CMT Roadshow Coming to a City Near You?

2024 CMT Roadshow Coming to a City Near You?

Do you want to make a difference in CMT research? HNF is looking for patients with a confirmed CMT diagnosis to participate in the CMT Biobank

Join the conversation

Leave a Comment


Submit a Comment

Your email address will not be published. Required fields are marked *


Join for notifications on events, campaigns, & news