Androgen Reduction in Congenital Adrenal Hyperplasia

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.

Conditions

• Congenital Adrenal Hyperplasia

Interventions

DRUG

Abiraterone acetate

Daily oral abiraterone acetate for 2 years. The dose will be specified based on pharmacodynamic data from Phase 1.

DRUG

Placebo

Daily placebo for 2 years.

DRUG

Hydrocortisone

Hydrocortisone will be administered at a starting dose of 7-9 mg/M2/d and adjusted as necessary based on 17-hydroxyprogesterone and ACTH levels.

DRUG

Fludrocortisone

Fludrocortisone will be administered at the dose the subject was taking a study entry and adjusted as necessary to keep plasma renin in the high normal range.

Sponsors & Collaborators

• National Institutes of Health Clinical Center (CC)

  collaborator NIH

• University of Michigan

  collaborator OTHER

• Children's Hospital Los Angeles

  collaborator OTHER

• Feinstein Institute for Medical Research

  collaborator OTHER

• University of Texas Southwestern Medical Center

  lead OTHER

Principal Investigators

• Perrin C White, MD · UT Southwestern Medical Center

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Model

PARALLEL

Eligibility

Min Age

2 Years

Max Age

9 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-01-31

Primary Completion

2026-01-31

Completion

2026-01-31

FDA Drug

Yes

Countries

• United States

Study Locations