Creutzfeldt-Jakob Disease Clinical Trial
Official title:
Novel Therapeutics For Prion Diseases: A Randomized, Double-blinded, Placebo-controlled Study of the Efficacy of Quinacrine in the Treatment of Sporadic Creutzfeldt-Jakob Disease
The purpose of this clinical trial is to determine the effectiveness of the medication quinacrine on survival in sporadic Creutzfeldt-Jakob disease (sCJD).
Creutzfeldt-Jakob disease (CJD)is a rapidly progressive, invariably fatal and untreatable
neurodegenerative disease with a mean duration of about eight months. Beyond the
debilitating cognitive and motor deficits that accompany CJD, the difficulty in treating
behavioral and mood disturbances and the rapidity of its course compound its tragedy. Recent
results from experiments show that, at physiological concentrations, the anti-malarial drug
quinacrine permanently clears abnormal prion proteins from cell culture. The demonstrated
efficacy of quinacrine in cell culture, its relative safety and well known side-effects in
the clinical setting, and the universal fatality of CJD justify quinacrine as an immediate
candidate for the treatment of CJD.
The purpose of this clinical trial is to determine the efficacy of the medication quinacrine
on survival in sporadic CJD (sCJD). This will be accomplished by bringing approximately 60
patients with probable or definite sCJD over approximately three years to UCSF for
evaluation and initiation of a randomized, double-blinded, placebo-controlled (delayed
treatment start) treatment study of quinacrine. Each patient will have a 50:50 chance of
being placed on quinacrine or placebo upon study enrollment; however, all patients will be
offered quinacrine after two months. Prior to study enrollment, patients will have a
comprehensive clinical assessment to confirm the diagnosis of sCJD. Participants will come
to UCSF for initial evaluation, potential study enrollment and, if possible, return to UCSF
for follow-up at two and twelve months. Patients will receive telephone follow-up (every 2
weeks for the first two months and monthly thereafter) and local blood and testing to
monitor for possible medication toxicity.
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Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment