Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.
LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN.
BMC Neurol. 2019. 10;19(1):224.
The primary goal of this study is to hasten drug development for FSHD by validating two novel clinical outcome assessments (COAs). An 18-month longitudinal study in 220 genetically confirmed and clinically affected participants will be performed.
MFM-D1 will be used as an outcome measure.
Keywords: Biomarkers; Clinical trial; Electrical impedance Myography; Facioscapulohumeral muscular dystrophy; Functional testing; Muscular dystrophy; Outcome measures.
Articles in scientific journals
Analysis of Different Device Interactions in a Virtual Reality Task in Individuals With Duchenne Muscular Dystrophy-A Randomized Controlled Trial
de Freitas BL, da Silva TD, Crocetta TB, Massetti T, de Araújo LV, Coe S, Dawes H, Caromano FA, Monteiro CBM. Front Neurol. 2019 Jan 29;10:24. The aim of the study was to compare performance of patients with Duchenne Muscular Dystrophy to perform a virtual task using...
Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy.
Nagy S, Schädelin S, Hafner P, Bonati U, Scherrer D, Ebi S, Schmidt S, Orsini AL, Bieri O, Fischer D. Muscle Nerve. 2020. 61(1), 63-68. In this retrospective study, the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD...
Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophies.
Jain MS, Meilleur K, Kim E, Norato G, Waite M, Nelson L, McGuire M, Duong T, Keller K, Lott DJ, Glanzman A, Rose K, Main M, Fiorini C, Chrismer I, Linton M, Punjabi M, Elliott J, Tounkara F, Vasavada R, Logaraj R, Winkert J, Donkervoort S, Leach M, Dastgir J, Hynan L,...
Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial
Hafner P, Bonati U, Klein A, Rubino D, Gocheva V, Schmidt S, Schroeder J, Bernert G, Laugel V, Steinlin M, Capone A, Gloor M, Bieri O, Hemkens LG, Speich B, Zumbrunn T, Gueven N, Fischer D. JAMA Netw Open. 2019. 2;2(10):e1914171. Results of a single-center randomized...
Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial.
Nagy S, Hafner P, Schmidt S, Rubino-Nacht D, Schädelin S, Bieri O, Fischer D. Trials. 2019. 20(1), 637. Protocol of a multicenter, randomized, double-blind, placebo-controlled phase III trial study aiming to demonstrate safety and efficacy of tamoxifen over placebo in...
Adult MTM1-related myopathy carriers: Classification based on deep phenotyping
Cocanougher BT, Flynn L, Yun P, Jain M, Waite M, Vasavada R, Wittenbach JD, de Chastonay S, Chhibber S, Innes AM, MacLaren L, Mozaffar T, Arai AE, Donkervoort S, Bönnemann CG, Foley AR. Neurology. 2019. 93 (16), e1535-e1542. Results of a cohort study performed at the...