title: Quinacrine treatment trial for sporadic Creutzfeldt-Jakob disease.
abstract: OBJECTIVE
  To determine whether oral quinacrine increases survival in sporadic Creutzfeldt-Jakob disease (sCJD).
  
  
  METHODS
  This NIH/National Institute on Aging-funded, double-blinded, placebo-controlled, stratified randomization treatment trial was conducted at the University of California, San Francisco from February 2005 through May 2009 (ClinicalTrials.gov, NCT00183092). Subjects were randomized (50:50) to quinacrine (300 mg daily) or placebo with inpatient evaluations at baseline, and planned for months 2, 6, and 12. Subjects returning for their month-2 visit were offered open-label quinacrine. The primary outcome was survival from randomization to month 2.
  
  
  RESULTS
  Of 425 patients referred, 69 subjects enrolled, 54 subjects were randomized to active drug or placebo, and 51 subjects with sCJD were included in survival analyses. Survival for the randomized portion of the trial (first 2 months) showed no significant difference between the 2 groups (log-rank statistic, p = 0.43; Cox proportional relative hazard = 1.43, quinacrine compared with placebo, 95% confidence interval = 0.58, 3.53). The quinacrine-treated group, however, declined less on 2 of 3 functional scales, the modified Rankin and Clinical Dementia Rating, than the placebo group during the first 2 months.
  
  
  CONCLUSIONS
  This interventional study provides Class I evidence that oral quinacrine at 300 mg per day does not improve 2-month survival of patients with sCJD, compared with placebo. Importantly, this study shows that double-blinded, placebo-controlled, randomized treatment trials are possible in prion disease. Furthermore, the quantitative data collected on the course of sCJD will be useful for future trials.
  
  
  METHODS
  This study provides Class I evidence that quinacrine does not improve survival for people with sCJD when given orally at a dose of 300 mg per day for 2 months.
authors.affiliation: From the Departments of Neurology (M.D.G., A.L.K., K.S.W., A.H., G.D., B.J.R., D.Y.J., C.C.T.-C., R.F., P.G., J.N.T., H.Q.C., S.A.F., J.L.D., K.L.P., S.B.P., B.L.M.), Biostatistics (J.M.N.), Pathology (S.J.D.), and Clinical Pharmacy (R.F.), and Institute for Neurodegenerative Diseases (S.J.D., S.B.P.), University of California, San Francisco.
authors.forename: Michael D
authors.initials: MD
authors.lastname: Geschwind
authors.forename: Amy L
authors.initials: AL
authors.lastname: Kuo
authors.forename: Katherine S
authors.initials: KS
authors.lastname: Wong
authors.forename: Aissa
authors.initials: A
authors.lastname: Haman
authors.forename: Gillian
authors.initials: G
authors.lastname: Devereux
authors.forename: Benjamin J
authors.initials: BJ
authors.lastname: Raudabaugh
authors.forename: David Y
authors.initials: DY
authors.lastname: Johnson
authors.forename: Charles C
authors.initials: CC
authors.lastname: Torres-Chae
authors.forename: Ron
authors.initials: R
authors.lastname: Finley
authors.forename: Paul
authors.initials: P
authors.lastname: Garcia
authors.forename: Julie N
authors.initials: JN
authors.lastname: Thai
authors.forename: Hugo Q
authors.initials: HQ
authors.lastname: Cheng
authors.forename: John M
authors.initials: JM
authors.lastname: Neuhaus
authors.forename: Sven A
authors.initials: SA
authors.lastname: Forner
authors.forename: Jacque L
authors.initials: JL
authors.lastname: Duncan
authors.forename: Katherine L
authors.initials: KL
authors.lastname: Possin
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Dearmond
authors.forename: Stanley B
authors.initials: SB
authors.lastname: Prusiner
authors.forename: Bruce L
authors.initials: BL
authors.lastname: Miller
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D007562:Creutzfeldt-Jakob Syndrome / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality*; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D011796:Quinacrine / Q000008:administration & dosage*; D015996:Survival Rate / Q000639:trends; D016896:Treatment Outcome
chemical_list: D011796:Quinacrine