Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

NCT02024932 ↗

Phase 2 Completed 37 enrolled Novartis Pharmaceuticals

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).

Timeline

Start: 2014-02-04
Primary completion: 2016-04-13
Completion: 2016-04-13

Outcome

Met primary endpoint

registry p = 0.0164; Geo-mean ratio 1.037 (90% CI 1.009 to 1.065); ANCOVA NCT02024932 ↗

paper A significant difference in change in TMV was observed in the BVS857 group versus the placebo group (geometric-mean ratio 1·04 [90% CI 1·01-1·07]; p=0·02). PMID 30337273 ↗

Publications

Grunseich C, Miller R, Swan T, Glass DJ, El Mouelhi M, Fornaro M, Petricoul O, Vostiar I, Roubenoff R, Meriggioli MN, Kokkinis A, Guber RD, Budron MS, Vissing J, Soraru G, Mozaffar T, Ludolph A, Kissel JT, Fischbeck KH; BVS857 study group. Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial. Lancet Neurol. 2018 Dec;17(12):1043-1052. doi: 10.1016/S1474-4422(18)30320-X. Epub 2018 Oct 15.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	BVS857	Unknown	0.01 mg/kg	Intravenous
Subject	BVS857	Unknown	0.03 mg/kg	Intravenous
Subject	BVS857	Unknown	0.06 mg/kg	Intravenous
Subject	BVS857	Unknown	0.1 mg/kg	Intravenous