An Exploratory, Randomized, Double-Blind Clinical Trial of Dipraglurant for Blepharospasm.

[BACKGROUND] Blepharospasm is treated with botulinum toxin, but obtaining satisfactory results is sometimes challenging.

[OBJECTIVE] The aim is to conduct an exploratory trial of oral dipraglurant for blepharospasm.

[METHODS] This study was an exploratory, phase 2a, randomized, double-blind, placebo-controlled trial of 15 participants who were assigned to receive a placebo or dipraglurant (50 or 100 mg) and assessed over 2 days, 1 and 2 hours following dosing. Outcome measures included multiple scales rated by clinicians or participants, digital video, and a wearable sensor.

[RESULTS] Dipraglurant was well tolerated, with no obvious impact on any of the measurement outcomes. Power analyses suggested fewer subjects would be required for studies using a within-subject versus independent group design, especially for certain measures. Some outcome measures appeared more suitable than others.

[CONCLUSION] Although dipraglurant appeared well tolerated, it did not produce a trend for clinical benefit. The results provide valuable information for planning further trials in blepharospasm. © 2024 International Parkinson and Movement Disorder Society.