Prion disease (all types) Nervous System Diseases Prion disease
Conditions
Interventions
The primary arm of the trial is a randomised controlled comparison of immediate quinacrine treatment (300 mg/day) versus no quinacrine treatment, with the option of starting quinacrine after 24 weeks
only in patients willing to be randomised.
Alternatively, patients can choose to be non-randomised and either receive quinacrine treatment immediately or not receive quinacrine treatment.
PRION-1 is
Sponsors
Medical Research Council (UK)
Eligibility
Sex/Gender
All
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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. | — |
Countries
United Kingdom
Outcome results
None listed