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

Gamze Kilic-Berkmen; Hodam Kim; Dongdong Chen; Cameron I Yeo; Ashok R Dinasarapu; Laura M Scorr; Woon-Hong Yeo; David A Peterson; Hilde Williams; April Ruby; Roger Mills; H A Jinnah

doi:10.1002/mds.29734

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

Mov Disord. 2024 Apr;39(4):738-745. doi: 10.1002/mds.29734. Epub 2024 Feb 3.

Authors

Gamze Kilic-Berkmen¹, Hodam Kim^{2

3}, Dongdong Chen⁴, Cameron I Yeo¹, Ashok R Dinasarapu¹, Laura M Scorr¹, Woon-Hong Yeo^{2

3

5

6}, David A Peterson⁷, Hilde Williams⁸, April Ruby⁸, Roger Mills⁸, H A Jinnah^{1

9}

Affiliations

¹ Department of Neurology, Emory University School of Medicine, Atlanta, Georgia, USA.
² Department of IEN Center for Human-Centric Interfaces and Engineering, The Institute for Electronics and Nanotechnology, Georgia Institute of Technology, Atlanta, Georgia, USA.
³ Department of George W. Woodruff School of Mechanical Engineering, Georgia Institute of Technology, Atlanta, Georgia, USA.
⁴ Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA.
⁵ Departments of Parker H. Petit Institute for Bioengineering and Biosciences, Institute for Materials, Neural Engineering Center, Institute for Robotics and Intelligent Machines, Georgia Institute of Technology, Atlanta, Georgia, USA.
⁶ Wallace H. Coulter Department of Biomedical Engineering, Georgia Tech and Emory University, Atlanta, Georgia, USA.
⁷ Department of Institute for Neural Computation, University of California in San Diego, La Jolla, California, USA.
⁸ Addex Pharmaceuticals Inc., Geneva, Switzerland.
⁹ Department of Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA.

PMID: 38310362
PMCID: PMC11045316 (available on 2025-04-01)
DOI: 10.1002/mds.29734

Abstract

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.

Keywords: blepharospasm; dipraglurant; dystonia; glutamate; trial.

Publication types

Randomized Controlled Trial
Research Support, Non-U.S. Gov't
Clinical Trial, Phase II

MeSH terms

Aged
Blepharospasm* / drug therapy
Double-Blind Method
Female
Humans
Male
Middle Aged
Treatment Outcome

Abstract

Publication types

MeSH terms

Grants and funding