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Clinical Trial
.2018 Jul 1;84(1):46-54.
doi: 10.1016/j.biopsych.2018.01.008. Epub 2018 Jan 31.

Combined Analysis of Mifepristone for Psychotic Depression: Plasma Levels Associated With Clinical Response

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Clinical Trial

Combined Analysis of Mifepristone for Psychotic Depression: Plasma Levels Associated With Clinical Response

Thaddeus S Block et al. Biol Psychiatry..
Free article

Abstract

Background: Patients with psychotic depression exhibit elevated cortisol levels. Competitively antagonizing cortisol at the glucocorticoid receptor with mifepristone demonstrated therapeutic benefit in early studies of patients with psychotic depression. We present a combined analysis of all controlled phase 2 and 3 studies to report antipsychotic differences between treatment with mifepristone or placebo and to evaluate the relative contributions to response of attaining an a priori-defined, high mifepristone plasma level and markers of glucocorticoid receptor antagonism (increases in adrenocorticotropin hormone and cortisol) with treatment.

Methods: Data from five similarly designed double-blind phase 2 or 3 studies evaluating the efficacy and safety of 7-day treatment with mifepristone for the psychotic symptoms of psychotic depression were pooled for analysis (mifepristone n = 833; placebo n = 627). Clinical assessments were performed at baseline and on days 7, 14, 28, 42, and 56. Mifepristone, adrenocorticotropin hormone, and cortisol samples were collected at baseline and day 7.

Results: Combined results demonstrated meaningful efficacy (p < .004) for mifepristone in reducing psychotic symptoms with wide safety margins. Patients in the a priori-defined, high mifepristone plasma level group (≥1637 ng/mL) demonstrated a more significant treatment effect over placebo (p = .0004). A number needed to treat of 7 and 48 was observed in the high and low mifepristone plasma level groups, respectively. Adverse events were similar in mifepristone- and placebo-treated patients.

Conclusions: A high mifepristone plasma level carried the strongest association with response, followed by changes in adrenocorticotropin hormone and cortisol. Therapeutic plasma levels of mifepristone were most likely to be achieved with the 1200 mg/day dose.

Trial registration: ClinicalTrials.govNCT00130676NCT00146523NCT00128479NCT00637494.

Keywords: Combined analysis; Glucocorticoid receptor; HPA axis; Mifepristone; Plasma level; Psychotic depression.

Copyright © 2018 Society of Biological Psychiatry. Published by Elsevier Inc. All rights reserved.

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