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.
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Contributors DV was involved in study concept and design, and editing the manuscript. HM, CP, WJS-S, AK, MB and FF were involved in acquisition of data. UH was involved in study concept and acquisition of data. MS was involved in critical revision of the manuscript. KK and TF were involved in statistical analyses and critical revision of the manuscript. IZ was involved in study concept and supervision, and funding.
Funding This study was supported by the Federal Ministry of Education and Research (grant no. 01KG0905) and by the Robert Koch-Institute through funds from the Federal Ministry of Health (grant no. 1369-341). Some of the work by KK has received funding from FP HEALTH 2013 ‘Innovative methodology for small populations research’ (InSPiRe) grant no. 602144.
Competing interests None declared.
Patient consent Obtained.
Ethics approval Ethikkommitee University of Goettingen.
Provenance and peer review Not commissioned; externally peer reviewed.
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