• Daniela Varges, Henrike Manthey, Uta Heinemann, Claudia Ponto, Matthias Schmitz, Walter J Schulz-Schaeffer, Anna Krasnianski, Maren Breithaupt, Fabian Fincke, Katharina Kramer, Tim Friede, and Inga Zerr.
• Department of Neurology, National Reference Center for TSE Surveillance, University Medical Center Goettingen, Göttingen, Germany.
• J. Neurol. Neurosurg. Psychiatr. 2017 Feb 1; 88 (2): 119-125.

ObjectivesThe main objective of the present study is to study the therapeutic efficiency of doxycycline in a double-blinded randomised phase II study in a cohort of patients with sporadic Creutzfeldt-Jakob disease (sCJD).MethodsFrom the National Reference Center of TSE Surveillance in Germany, patients with probable or definite sCJD were recruited for a double-blinded randomised study with oral doxycycline (EudraCT 2006-003934-14). In addition, we analysed the data from patients with CJD who received compassionate treatment with doxycycline in a separate group. Potential factors which influence survival such as age at onset, gender, codon 129 polymorphism and cognitive functions were evaluated. The primary outcome measure was survival.ResultsGroup 1: in the double-blinded randomised phase II study, 7 patients in the treatment group were compared with 5 controls. Group 2: 55 patients with sCJD treated with oral doxycycline were analysed and compared with 33 controls by a stratified propensity score applied to a Cox proportional hazard analysis. The results of both studies were combined by means of a random-effects meta-analysis. A slight increase in survival time in the doxycycline treatment group was observed (p=0.049, HR=0.63 (95% CI 0.402 to 0.999)).ConclusionsOn the basis of our studies, a larger trial of doxycycline should be performed in persons in the earliest stages of CJD.Trial Registration NumberEudraCT 2006-003934-14; Results.Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.

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