An Exploratory, Randomized, Double-Blind Clinical Trial of Dipraglurant for Blepharospasm.
blepharospasm
dipraglurant
dystonia
glutamate
trial
Journal
Movement disorders : official journal of the Movement Disorder Society
ISSN: 1531-8257
Titre abrégé: Mov Disord
Pays: United States
ID NLM: 8610688
Informations de publication
Date de publication:
03 Feb 2024
03 Feb 2024
Historique:
revised:
12
12
2023
received:
25
09
2023
accepted:
12
01
2024
medline:
4
2
2024
pubmed:
4
2
2024
entrez:
4
2
2024
Statut:
aheadofprint
Résumé
Blepharospasm is treated with botulinum toxin, but obtaining satisfactory results is sometimes challenging. The aim is to conduct an exploratory trial of oral dipraglurant for blepharospasm. 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. 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. 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.
Sections du résumé
BACKGROUND
BACKGROUND
Blepharospasm is treated with botulinum toxin, but obtaining satisfactory results is sometimes challenging.
OBJECTIVE
OBJECTIVE
The aim is to conduct an exploratory trial of oral dipraglurant for blepharospasm.
METHODS
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
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
CONCLUSIONS
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.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : NINDS NIH HHS
ID : U54 NS116025
Pays : United States
Informations de copyright
© 2024 International Parkinson and Movement Disorder Society.
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