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