Clinical trial NCT00104663: PRION-1: Quinacrine for Human Prion Disease
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Prion Disease
Keywords: Creutzfeldt-Jakob disease
inherited
sporadic
acquired
new variant CJD
Lead sponsor: Medical Research Council
Sponsor class: OTHER_GOV
First posted: 2005-03-04
Enrollment: 160
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: Inclusion Criteria:

* Aged 12 years or more, diagnosed with any type of human prion disease.

Exclusion Criteria:

* 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

Primary outcomes:
measure: Time to death
measure: 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:
measure: Mini Mental State Examination (MMSE)
measure: Clinician's Dementia rating (CDR)
measure: Rankin score
measure: Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog)
measure: Glasgow coma score
measure: Barthel Activities of Daily Living (ADL)
measure: magnetic resonance imaging scan (MRI)
measure: electro-encephalogram (EEG)
measure: cerebrospinal fluid (CSF)
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: National Prion Clinic
Study officials:
affiliation: MRC Prion Unit
name: John Collinge, MD, FRCP
role: PRINCIPAL_INVESTIGATOR
affiliation: MRC Clinical Trials Unit
name: Janet Darbyshire, MBChB, FRCP
role: STUDY_DIRECTOR
References: