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

Abstract

BackgroundBlepharospasm is treated with botulinum toxin, but obtaining satisfactory results is sometimes challenging.ObjectiveThe aim is to conduct an exploratory trial of oral dipraglurant for blepharospasm.MethodsThis 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.ResultsDipraglurant 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.ConclusionAlthough 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.