Additional identifiers
EudraCT number
ClinicalTrials.gov number
NCT00104663
Protocol/serial number
G0400713
Study information
Scientific title
Acronym
PRION-1
Study hypothesis
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.
Ethics approval
Not provided at time of registration.
Study design
Randomised controlled trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
Prion disease (all types)
Intervention
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.
Intervention type
Drug
Phase
Not Specified
Drug names
Quinacrine
Primary outcome measure
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
A series of secondary neurological and neuropsychiatric measures (Mini Mental State Examination [MMSE], Clinician's Dementia Rating [CDR], Rankin score, Alzheimers 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.
Overall trial start date
01/05/2004
Overall trial end date
30/04/2007
Reason abandoned (if study stopped)
Eligibility
Participant 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.
Participant type
Patient
Age group
Adult
Gender
Both
Target number of participants
160
Participant 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
Recruitment start date
01/05/2004
Recruitment end date
30/04/2007
Locations
Countries of recruitment
United Kingdom
Trial participating centre
MRC Prion Unit
London
WC1N 3GB
United Kingdom
Funders
Funder type
Government
Funder name
Department of Health (A861/495)(UK)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list
1. 2009 results in http://www.ncbi.nlm.nih.gov/pubmed/19278902
2. 2010 results in http://www.ncbi.nlm.nih.gov/pubmed/20881162
Publication citations
-
Results
Collinge J, Gorham M, Hudson F, Kennedy A, Keogh G, Pal S, Rossor M, Rudge P, Siddique D, Spyer M, Thomas D, Walker S, Webb T, Wroe S, Darbyshire J, Safety and efficacy of quinacrine in human prion disease (PRION-1 study): a patient-preference trial., Lancet Neurol, 2009, 8, 4, 334-344, doi: 10.1016/S1474-4422(09)70049-3.
-
Results
Siddique D, Hyare H, Wroe S, Webb T, Macfarlane R, Rudge P, Collinge J, Powell C, Brandner S, So PW, Walker S, Mead S, Yousry T, Thornton JS, Magnetization transfer ratio may be a surrogate of spongiform change in human prion diseases., Brain, 2010, 133, 10, 3058-3068, doi: 10.1093/brain/awq243.