Androgen Reduction in Congenital Adrenal Hyperplasia

• Register: ClinicalTrials.gov
• Last refreshed on: 13 February 2023
• Main ID: NCT03548246
• Date of registration: 24/04/2015
• Prospective Registration: Yes
• Primary sponsor: University of Texas Southwestern Medical Center
• Public title: Androgen Reduction in Congenital Adrenal Hyperplasia ARCH
• Scientific title: A Phase 1-2 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children With Classic 21-Hydroxylase Deficiency
• Date of first enrolment: January 2023
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT03548246
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Perrin C White, MD
• Affiliation: UT Southwestern Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- Pre-pubescent girls (age 2 years [12 kg] to 8 years inclusive; skeletal age =9 years)
or boys (age 2 years [12 kg] to 9 years inclusive; skeletal age =10 years).

- Confirmed classic 21-hydroxylase deficiency evident by genotype groups A, A1 or B, or
by clinical course.

- Requirement for standard of care fludrocortisone (any dose) and =10 mg/m2/day of
hydrocortisone for at least 1 month prior to the study consent.

- Morning serum androstenedione concentrations >1.5 x ULN after 7 days of dosing with
doses of hydrocortisone required for physiologic replacement.

- Informed consent .

Exclusion Criteria:

- Evidence of central puberty: Tanner Stage >2 for breast development in girls or
testicular volume >4 mL in boys, or random LH >0.3 mIU/mL.

- Current or history of hepatitis from any etiology.

- Abnormal liver function tests (transaminases>3X ULN).

- Abnormal renal function tests (BUN or creatinine >1.5 ULN).

- Significant anemia (hemoglobin < 12 g/dl).

- Clinically significant ECG abnormality

- A history of a malabsorption syndrome.

- Evidence of active malignancy.

- Co-existent disease that may interfere with linear growth or that requires concomitant
therapy that is likely to interfere with study procedures or results.

- Treatment with potentially hepatotoxic medications, CYP2D6, strong inhibitors or
inducers of CYP3A4

- Treatment with medications to affect puberty or synthesis of sex steroids, including
gonadotropin releasing hormone agonists, aromatase inhibitors, or androgen receptor
blockers

- Treatment with growth hormone

- Known allergies, hypersensitivity, or intolerance to abiraterone acetate or its
excipients.



Age minimum: 2 Years
Age maximum: 9 Years
Gender: All

Health Condition(s) or Problem(s) studied

Congenital Adrenal Hyperplasia

Intervention(s)

Drug: Hydrocortisone

Drug: Fludrocortisone

Drug: Placebo

Drug: Abiraterone acetate

Primary Outcome(s)

Bone age advancement [Time Frame: 104 weeks]

Secondary Outcome(s)

Predicted adult height [Time Frame: 104 weeks]

Body mass index Z-score [Time Frame: 104 weeks]

Weight [Time Frame: 104 weeks]

Hydrocortisone dose required to normalize androstenedione levels [Time Frame: 104 weeks]

Number of adverse events [Time Frame: 104 weeks]

Secondary ID(s)

042015-068

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institutes of Health Clinical Center (CC)

Feinstein Institute for Medical Research

Children's Hospital Los Angeles

University of Michigan