Communicable diseases - human prion disease and malaria

Lead Research Organisation: MRC Clinical Trials Unit

Abstract

We are trying to find treatments for human prion disease, of which variant CJD is one kind. The challenge is working out how to best assess possible treatments when patients are very sick and their carers feel strongly about how they are managed.||We first used a patient preference design, where patients could choose to take a drug, to not take it, or be randomised - almost all patients/carers did not want randomisation. A second study therefore clinically reviewed all UK patients taking a specific drug, and highlighted the lack of standardised management and monitoring. Now we are setting up a study for all UK patients with human prion disease - they can take any treatment they want, but we will follow them at the same intervals with standardised assessments.

Technical Summary

Human prion disease: the aim is to assess new therapies for this rapidly progressive fatal disease, and to identify the most appropriate study designs and outcome measures to make these assessments. Randomised controlled trials are difficult to perform and interpret given the small number of affected patients who are often incapacitated, necessitating consent from carers with strong views about the appropriateness of interventions. The first UK treatment study in human disease, PRION-1, was therefore designed as a patient preference trial which allowed individuals to choose to receive quinacrine or not, or to be randomised. The trial finished follow-up in March 2007, and found that randomisation was unacceptable to almost all patients/carers. Final results will be published later this year. A second study of pentosan polysulphate was designed as a clinical review of all UK patients who had received this drug which has to be given intraventricularly. It found that it had been given and tolerated at a very wide range of doses (although complications from the surgical procedures were common) and highlighted the lack of standardised management and monitoring. As a consequence, funding has recently been approved to set up a UK cohort of patients with human prion disease who will be monitored using standardised assessments and schedules, regardless of which interventions they choose to receive.

Publications

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