Condition category
Nervous System Diseases
Date applied
24/02/2004
Date assigned
24/03/2004
Last edited
10/11/2010
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Prof John Collinge

ORCID ID

Contact details

MRC Prion Unit
Institute of Neurology
National Hospital
Box 59
Queen Square
London
WC1N 3GB
United Kingdom

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 measures

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, 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.

Overall trial start date

01/05/2004

Overall trial end date

30/04/2007

Reason abandoned

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

Sponsor information

Organisation

Medical Research Council (UK)

Sponsor details

Second Floor
Stephenson House
158-160 North Gower St
London
NW1 2ND
United Kingdom

Sponsor type

Research council

Website

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

Results - basic reporting

Publication summary

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

  1. 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.

  2. 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.

Additional files

Editorial Notes