The SYNAPSE-CMT Phase IIa clinical trial evaluated NMD670 (ignaseclant) administered orally twice daily, compared to placebo, in 81 adults with Charcot-Marie-Tooth disease (CMT1 and CMT2) over 21 days in five countries, including France. Initial results from the trial were shared in a press release:
- the primary endpoint, corresponding to improvement in distance walked in the 6-minute walk test, was not achieved by the treated group compared to placebo.
- However, signs of efficacy were observed for several secondary endpoints, including grip strength, fine motor skills of the hands and certain clinical scores (CMT-FOM and CMT-HI).
The laboratory considers that the data support the continued development of the product in a longer trial involving a larger group of patients.
