Prion DiseaseNCT00104663
PRION-1: Quinacrine for Human Prion Disease
A study testing Quinacrine in people with Prion Disease, run by Medical Research Council at 1 site in United Kingdom.
Trial summary
PRION-1 aims to assess the activity and safety of Quinacrine (Mepacrine hydrochloride) in human prion disease. It also aims to establish an appropriate framework for the clinical assessment of therapeutic options for human prion disease that can be refined or expanded in the future, as new agents become available. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Prion Disease
- What is being tested?
- Quinacrine
- How many people
- 160 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jun 2004to Mar 2007
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged 12 years or more, diagnosed with any type of human prion disease.
Who cannot join
- In a coma, or in a pre-terminal phase of disease such that prolongation of the current quality of life would not be supported
- Known sensitivity to quinacrine
- Been taking any other putative anti-prion therapy for less than 8 weeks
Trial locations and contacts
1 site in 1 country.
About this study
Official title: PRION-1: Quinacrine for Human Prion Disease. A Partially Randomized Patient Preference Trial to Evaluate the Activity and Safety of Quinacrine in Human Prion Disease
PRION-1 aims to assess the activity and safety of Quinacrine (Mepacrine hydrochloride) in human prion disease. It also aims to establish an appropriate framework for the clinical assessment of therapeutic options for human prion disease that can be refined or expanded in the future, as new agents become available.
Read the detailed description
The human prion diseases have been traditionally classified into Creutzfeldt-Jakob disease (CJD), Gerstmann-Sträussler-Scheinker (GSS) disease and kuru. They can alternatively be classified into three causal categories: sporadic, acquired and inherited. The appearance of a new human prion disease, variant CJD (vCJD), in the United Kingdom from 1995 onwards, and the experimental evidence that this is caused by the same prion strain as that causing bovine spongiform encephalopathy (BSE) in cattle, has raised the possibility that a major epidemic of vCJD will occur in the United Kingdom and other countries as a result of dietary or other exposure to BSE prions. These concerns have led to intensified efforts to develop therapeutic interventions.
Quinacrine has been previously used to treat other diseases such as malaria; however, it was found to have serious side effects and is no longer licensed in the United Kingdom. There is only very limited evidence from laboratory tests for the potential use of quinacrine in human prion disease, and the evidence to date for any possible clinical benefit is very scarce. The PRION-1 trial is being undertaken since there are no other drugs currently available which are considered suitable for human evaluation.
- Conditions
- Prion Disease
- Being tested
- Quinacrine
- Keywords
- Creutzfeldt-Jakob disease, inherited, sporadic, acquired, new variant CJD
- Medical subject headings
- Prion Diseases, Creutzfeldt-Jakob Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuinacrineDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to death
proportion of responders, with "responders" defined as patients showing either clinical improvement or lack of deterioration in 3 key neurological and neuropsychiatric measures
Secondary outcomes (9)
Mini Mental State Examination (MMSE)
Clinician's Dementia rating (CDR)
Rankin score
Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog)
Glasgow coma score
Barthel Activities of Daily Living (ADL)
magnetic resonance imaging scan (MRI)
electro-encephalogram (EEG)
cerebrospinal fluid (CSF)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2005 (estimated)
- Study startJun 2004
- Study completionMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prion DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 160 participants
Sponsor and investigators
- Lead sponsor
- Medical Research Council
- Sponsor type
- Other government
- Investigators
- John Collinge, MD, FRCP, Principal investigator, MRC Prion UnitJanet Darbyshire, MBChB, FRCP, Study director, MRC Clinical Trials Unit
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00104663
- Sponsor's protocol ID
- Version 1.1
- Other IDs
- Grant ID:71361
- Status last verified
- Apr 2015
- First submitted
- 3 Mar 2005
- Last updated
- 7 Apr 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PRION-1: Quinacrine for Human Prion Disease. A Partially Randomized Patient Preference Trial to Evaluate the Activity and Safety of Quinacrine in Human Prion Disease
- ClinicalTrials.gov ID
- NCT00104663
- Acronym / sponsor ID
- Version 1.1
- Conditions
- Prion Disease
- Keywords
- Creutzfeldt-Jakob disease, inherited, sporadic, acquired, new variant CJD
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 160 participants
- Lead sponsor
- Medical Research CouncilOther government
- Study start
- Jun 2004
- Study completion
- Mar 2007
- First posted
- 4 Mar 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
PRION-1 aims to assess the activity and safety of Quinacrine (Mepacrine hydrochloride) in human prion disease. It also aims to establish an appropriate framework for the clinical assessment of therapeutic options for human prion disease that can be refined or expanded in the future, as new agents become available.
Detailed description
The human prion diseases have been traditionally classified into Creutzfeldt-Jakob disease (CJD), Gerstmann-Sträussler-Scheinker (GSS) disease and kuru. They can alternatively be classified into three causal categories: sporadic, acquired and inherited. The appearance of a new human prion disease, variant CJD (vCJD), in the United Kingdom from 1995 onwards, and the experimental evidence that this is caused by the same prion strain as that causing bovine spongiform encephalopathy (BSE) in cattle, has raised the possibility that a major epidemic of vCJD will occur in the United Kingdom and other countries as a result of dietary or other exposure to BSE prions. These concerns have led to intensified efforts to develop therapeutic interventions.
Quinacrine has been previously used to treat other diseases such as malaria; however, it was found to have serious side effects and is no longer licensed in the United Kingdom. There is only very limited evidence from laboratory tests for the potential use of quinacrine in human prion disease, and the evidence to date for any possible clinical benefit is very scarce. The PRION-1 trial is being undertaken since there are no other drugs currently available which are considered suitable for human evaluation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Aged 12 years or more, diagnosed with any type of human prion disease.
Key exclusion criteria (3 listed)
- In a coma, or in a pre-terminal phase of disease such that prolongation of the current quality of life would not be supported
- Known sensitivity to quinacrine
- Been taking any other putative anti-prion therapy for less than 8 weeks
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Quinacrine | Drug | – |
Primary outcome
Time to death
proportion of responders, with "responders" defined as patients showing either clinical improvement or lack of deterioration in 3 key neurological and neuropsychiatric measures
9 secondary outcomes
Mini Mental State Examination (MMSE)
Clinician's Dementia rating (CDR)
Rankin score
Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog)
Glasgow coma score
Barthel Activities of Daily Living (ADL)
magnetic resonance imaging scan (MRI)
electro-encephalogram (EEG)
cerebrospinal fluid (CSF)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Apr 2015.
- MRC Clinical Trials UnitLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Collinge, MD, FRCP principal investigator
MRC Prion Unit
Janet Darbyshire, MBChB, FRCP study director
MRC Clinical Trials Unit
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Quinacrine in people with Prion Disease, run by Medical Research Council at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to death. The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Apr 2015.