MedTrace Logo

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

NCT02574910 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2025-11-04

Study results available
· View outcomes & findings →

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.

Conditions

  • Congenital Adrenal Hyperplasia

Interventions

DRUG

Abiraterone acetate

This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.

Sponsors & Collaborators

Principal Investigators

  • Perrin C White, MD · UT Southwestern Medical Center

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
9 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-08-01
Primary Completion
2023-06-03
Completion
2023-06-03
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02574910 on ClinicalTrials.gov