Single-Dose Study of a Corticotropin-Releasing Factor Receptor-1 Antagonist in Women With 21-Hydroxylase Deficiency.

ABSTRACT:

Context

Treatment of 21-hydroxylase deficiency (21OHD) is difficult to optimize. Normalization of excessive ACTH and adrenal steroid production commonly requires supraphysiologic doses of glucocorticoids.

Objectives

We evaluated the safety and tolerability of the selective corticotropin releasing factor type 1 (CRF1) receptor antagonist NBI-77860 in women with classic 21OHD and tested the hypothesis that CRF1 receptor blockade decreases early-morning ACTH and 17α-hydroxyprogesterone (17OHP) in these patients.

Participants

The study enrolled eight classic 21OHD females, ages 18-58 years, seen at a single tertiary referral university setting.

Design

This was a phase Ib, single-blind, placebo-controlled, fixed-sequence, single-dose trial. During three treatment pe

SUBMITTER: Turcu AF 

PROVIDER: S-EPMC4803170 | biostudies-literature | 2016 Mar

REPOSITORIES: biostudies-literature