Doxycycline in early CJD: a double blinded randomised phase II and observational study
Objectives The 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) Methods From 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. Results Group 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) Conclusions On the basis of our studies, a larger trial of doxycycline should be performed in persons in the earliest stages of CJD. Trial registration number EudraCT 2006 003934 14; Results.
Journal of Neurology, Neurosurgery Psychiatry