Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2017 Feb;88(2):119-125.
doi: 10.1136/jnnp-2016-313541. Epub 2016 Nov 2.

Doxycycline in early CJD: a double-blinded randomised phase II and observational study

Daniela Varges  1 , Henrike Manthey  1 , Uta Heinemann  1 , Claudia Ponto  1 , Matthias Schmitz  1 , Walter J Schulz-Schaeffer  2 , Anna Krasnianski  1 , Maren Breithaupt  1 , Fabian Fincke  1 , Katharina Kramer  3 , Tim Friede  3 , Inga Zerr  1
Affiliations
Clinical Trial

Doxycycline in early CJD: a double-blinded randomised phase II and observational study

Daniela Varges et al. J Neurol Neurosurg Psychiatry. 2017 Feb.

Abstract

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.

PubMed Disclaimer

Conflict of interest statement

Conflicts of Interest: None declared.

Figures

Figure 1
Figure 1
Overview clinical study. CJD, Creutzfeldt-Jakob disease; PEG, percutaneous endoscopic gastronomy.
Figure 2
Figure 2
Survival times of doxycycline treated and non-treated patients. (A) Kaplan-Meier survival times of both trials. (B) Forest plot of the combined meta-analysis. RE, randomised effects.
Figure 3
Figure 3
Influence of the codon 129 polymorphism on survival time in the observational study.
See this image and copyright information in PMC

Comment in

References

    1. Korth C, Peters PJ. Emerging pharmacotherapies for Creutzfeld-Jakob disease. Arch Neurol 2006;63:497–501. 10.1001/archneur.63.4.497 - DOI - PubMed
    1. Trevitt CR, Collinge J. A systematic review of prion therapeutics in experimental models. Brain 2006;129:2241–65. 10.1093/brain/awl150 - DOI - PubMed
    1. Stewart LA, Rydzewska LH, Keogh GF, et al. . Systematic review of therapeutic interventions in human prion diseases. Neurology 2008;70:1272–81. 10.1212/01.wnl.0000308955.25760.c2 - DOI - PubMed
    1. Zerr I. Human prion diseases: progress in clinical trials. Brain 2013;136:996–7. 10.1093/brain/awt075 - DOI - PMC - PubMed
    1. Appleby BS, Lyketsos CG. Rapidly progressive dementias and the treatment of human prion diseases. Expert Opin Pharmacother 2011;12:1–12. 10.1517/14656566.2010.514903 - DOI - PMC - PubMed

Publication types