CJD Quinacrine Study (UCSF, NCT00183092)

A single-site, double-blind, placebo-controlled phase 2 trial at UCSF testing oral quinacrine in sporadic CJD. It was run by the Memory and Aging Center with NIA funding. It found no effect on survival over the 2-month randomized period, and it showed that a placebo-controlled randomized trial can be done in a rapidly fatal prion disease.

Status as of 2026-11-01. Please check the study registry for the latest position.

Key facts

  • Registered 2005-09 as NCT00183092 (org id IA0083), sponsor UCSF with the National Institute on Aging as collaborator; phase 2, randomized, quadruple masked, one site.
  • Participants had probable or definite sporadic CJD, were 18 or older, could swallow, and agreed to autopsy; genetic prion disease was an exclusion.
  • The registry lists quinacrine 100 mg three times daily against matching placebo; the paper states 300 mg daily.
  • Of 425 patients referred, 69 enrolled, 54 were randomized, and 51 were analysed (28 placebo, 23 quinacrine) after three were found to carry PRNP mutations.
  • Primary outcome, survival to month 2: 19 of 28 on placebo and 13 of 23 on quinacrine; hazard ratio 1.43 (95% CI 0.58 to 3.53), p 0.43.
  • At month 2 the quinacrine group had declined less on the Clinical Dementia Rating sum of boxes and the modified Rankin scale; no cognitive test differed. The authors flag a baseline imbalance in Rankin and MMSE scores.
  • Elevated liver tests occurred only on quinacrine; dose reduction was needed in 22% of the quinacrine and 18% of the placebo arm. After month 2, all participants could take open-label quinacrine.
  • The result was published in Neurology 2013;81(23):2015-2023, not in Lancet Neurology; the authors call it Class I evidence of no 2-month survival benefit.
  • Funders named in the paper include NIA grants, the Hillblom Foundation, Sherman Fairchild Foundation, Fight for Mike Homer Program, Rainwater Charitable Foundation and the CJD Foundation.
  • Relation to PRION-1: the UK study (Collinge, MRC Prion Unit) was a patient-preference design that became mainly observational because only 2 of 84 main-study patients accepted randomization, and it also found no adjusted survival difference.
  • Geschwind's 2009 Lancet Neurology editorial on PRION-1 cites the 2004 French (Haik et al.) and Nakajima et al. quinacrine reports as showing no clear benefit and argues future trials must be randomized, blinded and placebo controlled.

Timeline

  1. 2005to 2005-04 Trial start (paper says February 2005, registry April 2005).
  2. 2005Registered on ClinicalTrials.gov.
  3. 2009to 2009-05 Enrolment ends (registry results text says January 2009, paper says May 2009).
  4. 2009PRION-1 published; Geschwind editorial notes UCSF enrolment complete.
  5. 2012Primary and study completion per registry.
  6. 2013Results published in Neurology.
  7. 2014Results posted on ClinicalTrials.gov.

Last checked 1 November 2026

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