Journal article
Randomized placebo-controlled study of lovastatin in children with neurofibromatosis type 1
JM Payne, B Barton, NJ Ullrich, A Cantor, SJC Hearps, G Cutter, T Rosser, KS Walsh, GA Gioia, PL Wolters, J Tonsgard, E Schorry, D Viskochil, L Klesse, M Fisher, DH Gutmann, AJ Silva, SJ Hunter, C Rey-Casserly, NL Cantor Show all
Neurology | LIPPINCOTT WILLIAMS & WILKINS | Published : 2016
Abstract
Objective: To assess the efficacy of lovastatin on visuospatial learning and attention for treating cognitive and behavioral deficits in children with neurofibromatosis type 1 (NF1). Methods: A multicenter, international, randomized, double-blind, placebo-controlled trial was conducted between July 2009 and May 2014 as part of the NF Clinical Trials Consortium. Children with NF1 aged 8-15 years were screened for visuospatial learning or attention deficits (n = 272); 146 children demonstrated deficits at baseline and were randomly assigned to lovastatin (n = 74; 40 mg/d) or placebo (n = 70). Treatment was administered once daily for 16 weeks. Primary outcomes were total errors on the Cambridg..
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Awarded by US Army Medical Research and Materiel Command, Office of the Congressionally Directed Medical Research Programs, Department of Defense Neurofibromatosis Research Program
Funding Acknowledgements
This work was supported by the US Army Medical Research and Materiel Command, Office of the Congressionally Directed Medical Research Programs, Department of Defense Neurofibromatosis Research Program, grant W81XWH-05-1-0615. The funding source reviewed and approved the protocol. It had no role in the conception and design of the study; collection, management, analysis, or interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication. The work was also partially funded by Sally Gottesman and Rachel B. Tiven.