PRION-1: Quinacrine for human prion disease. A partially randomised patient preference trial to evaluate the activity and safety of quinacrine in human prion disease
Completed
- Conditions
- Prion disease (all types)Nervous System DiseasesPrion disease
- Registration Number
- ISRCTN06722585
- Lead Sponsor
- Medical Research Council (UK)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 160
Inclusion Criteria
Eligible patients will be adults or children aged 12 years or more diagnosed with any type of human prion disease, and without clinical or laboratory abnormalities contraindicating use of quinacrine.
Exclusion Criteria
1. In a coma, or in a pre-terminalphase of disease such that prolongation of the current quality of life would not be supported
2. Have known hypersensitivity to quinacrine
3. Have been taking any other putative anti-prion therapy for less than 8 weeks
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The primary efficacy endpoints are mortality and the proportion of responders overall and at 24 weeks. Response is defined as lack of deterioration in three key neurological and neuropsychiatric measures (standardised neurological exam, a measure of global functioning, and Brief Psychiatric Rating Scale [BPRS]).
- Secondary Outcome Measures
Name Time Method A series of secondary neurological and neuropsychiatric measures (Mini Mental State Examination [MMSE], Clinician's Dementia Rating [CDR], Rankin score, Alzheimer?s Disease Assessment Scale ? Cognitive [ADAS-Cog], Glasgow coma score and Barthel Activities of Daily Living [ADL]), and neurological investigations including magnetic resonance imaging scan (MRI), electro-encephalogram (EEG) and cerebro-spinal fluid (CSF) sampling will also be carried out.