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
| ISRCTN | ISRCTN06722585 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN06722585 |
| ClinicalTrials.gov number | NCT00104663 |
| Secondary identifying numbers | G0400713 |
- Submission date
- 24/02/2004
- Registration date
- 24/03/2004
- Last edited
- 10/11/2010
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Prof John Collinge
Scientific
Scientific
MRC Prion Unit
Institute of Neurology
National Hospital
Box 59
Queen Square
London
WC1N 3GB
United Kingdom
Study information
| Study design | Randomised controlled trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| Scientific title | |
| Study acronym | PRION-1 |
| Study objectives | 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(s) | Not provided at time of registration. |
| Health condition(s) or problem(s) studied | 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 |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | 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 study start date | 01/05/2004 |
| Completion date | 30/04/2007 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 160 |
| Key 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. |
| Key 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 |
| Date of first enrolment | 01/05/2004 |
| Date of final enrolment | 30/04/2007 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centre
MRC Prion Unit
London
WC1N 3GB
United Kingdom
WC1N 3GB
United Kingdom
Sponsor information
Medical Research Council (UK)
Research council
Research council
Second Floor
Stephenson House
158-160 North Gower St
London
NW1 2ND
United Kingdom
"ROR" |
https://ror.org/03x94j517 |
|---|
Funders
Funder type
Government
Department of Health (A861/495)(UK)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/04/2009 | Yes | No | |
| Results article | results | 01/10/2010 | Yes | No |
