Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia

Autor: Francesco Saccà, Alessandro Filla, Boriana Büchner, Thomas Klopstock, Caterina Mariotti, Karen Manicom, Ludger Schöls, Lorenzo Nanetti, Wolfgang Nachbauer, Sylvia Boesch, Jennifer Müller vom Hagen, Thomas Klockgether, Heike Jacobi
Rok vydání: 2014
Předmět:
Zdroj: Movement Disorders. 29:935-939
ISSN: 0885-3185
DOI: 10.1002/mds.25836
Popis: Background Erythropoietin (EPO) derivatives have been found to increase frataxin levels in Friedreich's ataxia (FRDA) in vitro. This multicenter, double-blind, placebo-controlled, phase II clinical trial aimed to evaluate the safety and tolerability of Lu AA24493 (carbamylated EPO; CEPO). Methods Thirty-six ambulatory FRDA patients harboring >400 GAA repeats were 2:1 randomly assigned to either CEPO in a fixed dose (325 µg thrice-weekly) or placebo. Safety and tolerability were assessed up to 103 days after baseline. Secondary outcome measures of efficacy (exploration of biomarkers and ataxia ratings) were performed up to 43 days after baseline. Results All patients received six doses of study medication. Adverse events were equally distributed between CEPO and placebo. There was no evidence for immunogenicity of CEPO after multiple dosing. Biomarkers, such as frataxin, or measures for oxidative stress and ataxia ratings did not differ between CEPO and placebo. Conclusion CEPO was safe and well tolerated in a 2-week treatment phase. Secondary outcome measures remained without apparent difference between CEPO and placebo. © 2014 International Parkinson and Movement Disorder Society
Databáze: OpenAIRE
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