Prion Disease
Conditions
Keywords
Creutzfeldt-Jakob disease, inherited, sporadic, acquired, new variant CJD
Brief summary
PRION-1 aims to assess the activity and safety of Quinacrine (Mepacrine hydrochloride) in human prion disease. It also aims to establish an appropriate framework for the clinical assessment of therapeutic options for human prion disease that can be refined or expanded in the future, as new agents become available.
Detailed description
The human prion diseases have been traditionally classified into Creutzfeldt-Jakob disease (CJD), Gerstmann-Sträussler-Scheinker (GSS) disease and kuru. They can alternatively be classified into three causal categories: sporadic, acquired and inherited. The appearance of a new human prion disease, variant CJD (vCJD), in the United Kingdom from 1995 onwards, and the experimental evidence that this is caused by the same prion strain as that causing bovine spongiform encephalopathy (BSE) in cattle, has raised the possibility that a major epidemic of vCJD will occur in the United Kingdom and other countries as a result of dietary or other exposure to BSE prions. These concerns have led to intensified efforts to develop therapeutic interventions. Quinacrine has been previously used to treat other diseases such as malaria; however, it was found to have serious side effects and is no longer licensed in the United Kingdom. There is only very limited evidence from laboratory tests for the potential use of quinacrine in human prion disease, and the evidence to date for any possible clinical benefit is very scarce. The PRION-1 trial is being undertaken since there are no other drugs currently available which are considered suitable for human evaluation.
Interventions
DRUGQuinacrine
Sponsors
Medical Research Council
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Aged 12 years or more, diagnosed with any type of human prion disease.
Exclusion criteria
* In a coma, or in a pre-terminal phase of disease such that prolongation of the current quality of life would not be supported * Known sensitivity to quinacrine * Been taking any other putative anti-prion therapy for less than 8 weeks
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to death | — |
| proportion of responders, with responders defined as patients showing either clinical improvement or lack of deterioration in 3 key neurological and neuropsychiatric measures | — |
Secondary
| Measure | Time frame |
|---|---|
| Rankin score | — |
| Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog) | — |
| Glasgow coma score | — |
| Mini Mental State Examination (MMSE) | — |
| magnetic resonance imaging scan (MRI) | — |
| electro-encephalogram (EEG) | — |
| cerebrospinal fluid (CSF) | — |
| Barthel Activities of Daily Living (ADL) | — |
| Clinician's Dementia rating (CDR) | — |
Countries
United Kingdom
Outcome results
None listed