PRION-1: Quinacrine for Human Prion Disease

Prion Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00104663
• Other study ID #: Version 1.1
• Secondary ID: Grant ID:71361
• Status: Completed
• Phase: N/A
• First received: March 3, 2005
• Last updated: April 6, 2015
• Start date: June 2004
• Est. completion date: March 2007

Study information

• Verified date: April 2015
• Source: Medical Research Council
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 160
• Est. completion date: March 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Prion Disease
• Prion Diseases

Intervention

Drug:
• Quinacrine

Locations

Country	Name	City	State
United Kingdom	National Prion Clinic	London

Sponsors (1)

Lead Sponsor	Collaborator
Medical Research Council

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to death
Primary	proportion of responders, with "responders" defined as patients showing either clinical improvement or lack of deterioration in 3 key neurological and neuropsychiatric measures
Secondary	Mini Mental State Examination (MMSE)
Secondary	Clinician's Dementia rating (CDR)
Secondary	Rankin score
Secondary	Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog)
Secondary	Glasgow coma score
Secondary	Barthel Activities of Daily Living (ADL)
Secondary	magnetic resonance imaging scan (MRI)
Secondary	electro-encephalogram (EEG)
Secondary	cerebrospinal fluid (CSF)