drugset
drugset / Trial / NCT02574910

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

NCT02574910 ↗

Phase 1 Terminated 4 enrolled University of Texas Southwestern Medical Center Children's Hospital Los Angeles · collabEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · collabUniversity of Michigan · collab
Non-randomizedSequentialOpen-labelTreatment

Summary

Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height. Additionally, these children require supraphysiologic doses of hydrocortisone to suppress secretion of adrenal androgen precursors, and this treatment can retard linear growth. This study seeks to use oral abiraterone acetate (Zytiga)as an adjunct to approved CAH therapy (oral hydrocortisone and fludrocortisone) for pre-pubescent children with classic 21-hydroxylase deficiency in order to reduce daily requirement of hydrocortisone. In this Phase 1 study, the investigators will determine the minimum effective dose of abiraterone acetate that normalizes androstenedione levels during the 7-day Treatment Period.

Timeline

Start
2017-08-01
Primary completion
2023-06-03
Completion
2023-06-03

Publications

Drugs

EvaluationDrugModalityDoseRoute
Subject Abiraterone acetate Small molecule 1 mg/kg Oral
Subject Abiraterone acetate Small molecule 2 mg/kg Oral
Subject Abiraterone acetate Small molecule 4 mg/kg Oral

Indications

classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency