Efficacy of Botulinum Toxin A in Adult Subjects With Raynaud Phenomenon Secondary to Systemic Sclerosis

Phase 3 Completed 91 enrolled Assistance Publique - Hôpitaux de Paris

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This study aims to assess whether or not a single injection schedule of botulinum toxin A (BTX-A) in both hands improves Raynaud phenomenon (RP) secondary to systemic sclerosis (SSc) better than a placebo at 4, 12 and 24 weeks after the treatment. This study's hypothesis is that the number of RP attacks per week from baseline to 4 weeks after treatment is significantly lower in the group treated with BTX-A than in the control group treated by the placebo. Furthermore, BTX-A in both hands is expected to improve both symptomatic (attack frequency, digital ulcer healing) and functional (pain, hand function, quality of life) symptoms of RP secondary to SSc more than placebo.

Timeline

Start: 2018-10-15
Primary completion: 2020-07-31
Completion: 2020-07-31

Outcome

Missed primary endpoint

paper Neither the primary nor secondary end points were reached, and our results do not support any beneficial effect of palmar BTA injections to treat SSc-RP. PMID 36066501 ↗

paper P = 0.77 versus placebo PMID 36066501 ↗

Publications

Senet P, Maillard H, Diot E, Lazareth I, Blaise S, Arnault JP, Pistorius MA, Boulon C, Cogrel O, Warzocha U, Riviere S, Malloizel-Delaunay J, Servettaz A, Sassolas B, Viguier M, Monfort JB, Janique S, Vicaut E; BRASS collaborators. Efficacy and Safety of Botulinum Toxin in Adults with Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study. Arthritis Rheumatol. 2023 Mar;75(3):459-467. doi: 10.1002/art.42342. Epub 2022 Dec 14.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Botulinum toxin type A	Protein / enzyme biologic	50 iu	Other