Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Status:
Active, not recruiting
Trial end date:
2022-01-23
Target enrollment:
Participant gender:
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.
Phase:
Phase 1
Details
Lead Sponsor:
University of Texas Southwestern Medical Center
Collaborators:
Children's Hospital Los Angeles Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Feinstein Institute for Medical Research University of Michigan