Phase 2 Multi-center Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

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Abstract

This is a multicenter, single-blind, multiple dose study of ATR- 101 with a placebo washout component. The Screening Period will be followed by a placebo, Baseline Lead-in Period. Subjects will then receive the lowest dose of ATR-101 for 14 days followed by a single-blind placebo Washout Period of 14 days. Safety and efficacy assessments will occur at the end of ATR-101 treatment and following the Washout Period. If the primary outcome measure (17-OHP 2x ULN) is not achieved, the subject will proceed to the next higher dose level. ATR-101 treatment will stop once the primary outcome measure is met. A total of 5 ATR-101 dose levels are possible. Ten patients were enrolled; nine completed the study. Data analysis is underway