Pharnext, a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, has announced publication in PLoS One of statistical findings evaluating the clinical scale Charcot-Marie-Tooth Neuropathy Score (CMTNS) for application in Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Pharnext said that CMTNS is the first clinical scale dedicated to quantify impairment and measure disease progression in CMT patients. It has been used as the primary endpoint in most completed clinical trials for CMT1A to date. However, its ability to measure responses to treatment has not yet been demonstrated, the company said.
In the article titled: “A Rasch Analysis of the Charcot-Marie-Tooth Neuropathy Score (CMTNS) in a Cohort of Charcot-Marie-Tooth Type 1A Patients” authors Wenjia Wang et al. report that CMTNS remains particularly useful for measuring disease severity in CMT1A. However, CMTNS appears less sensitive in assessing mild forms of the disease. Therefore, the authors recommend further refinement of the scale to better assess therapeutic efficacy in all forms of CMT1A: mild, moderate and severe.
Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext, said “One of the major challenges of CMT1A trial design is selecting a clinically meaningful endpoint. The findings confirm the limitations of the CMTNS. They support Pharnext’s decision, endorsed by the European and American regulatory agencies, not to use CMTNS as the primary efficacy endpoint in our pivotal PLEO-CMT trial for patients suffering from mild to moderate forms of CMT1A, and to use the Overall Neuropathy Limitations Scale (ONLS) instead. The ONLS has been used as the primary endpoint to assess treatment efficacy in other peripheral neuropathies pivotal trials.”