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ISRCTN
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ISRCTN06722585
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ClinicalTrials.gov identifier
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NCT00104663
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Public title
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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
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Scientific title
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Acronym
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PRION-1
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Serial number at source
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G0400713
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Study hypothesis
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The PRION-1 trial is being undertaken to evaluate the activity and safety of quinacrine in human prion disease since there are no other drugs currently available which are considered suitable for human evaluation.
The primary aim of the trial is a randomised controlled comparison of immediate quinacrine treatment versus no quinacrine treatment, with the option of starting quinacrine after 24 weeks (deferred quinacrine); only patients who are willing to be randomised will enter this comparison. However it is appreciated that many patients will have a strong preference for receiving quinacrine immediately. Other patients will have a strong preference for not receiving quinacrine (for example, they may prefer to wait for future therapeutic options). These non-randomised groups of patients will be followed up in the same way as the randomised patients.
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Ethics approval
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Not provided at time of registration.
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Study design
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Randomised controlled trial
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Countries of recruitment
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United Kingdom
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Disease/condition/study domain
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Prion disease (all types)
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Participants - inclusion criteria
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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.
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Participants - exclusion criteria
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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
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Anticipated start date
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01/05/2004
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Anticipated end date
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30/04/2007
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Status of trial
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Completed |
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Patient information material
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Target number of participants
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160
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Interventions
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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 (deferred quinacrine); 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 a 3 year trial. It is planned to recruit approximately 160 patients over a period of 2 years and follow all patients for at least 1 year.
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Primary outcome measure(s)
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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]).
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Secondary outcome measure(s)
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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.
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Sources of funding
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Department of Health (A861/495)(UK)
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Trial website
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Publications
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2009 results in http://www.ncbi.nlm.nih.gov/pubmed/19278902
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Contact name
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Prof
John
Collinge
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Address
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MRC Prion Unit
Institute of Neurology
National Hospital
Box 59
Queen Square
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City/town
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London
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Zip/Postcode
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WC1N 3GB
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Country
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United Kingdom
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Sponsor
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Medical Research Council (UK)
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Address
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Second Floor
Stephenson House
158-160 North Gower St
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City/town
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London
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Zip/Postcode
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NW1 2ND
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Country
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United Kingdom
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Date applied
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24/02/2004
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Last edited
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07/09/2009
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Date ISRCTN assigned
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24/03/2004
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