Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Stopped: The study has been affected significantly by the COVID pandemic, drug supply/drug amendment, low recruitment interest, and FDA approval of another drug with the same indication, so this study has not met its primary endpoint data requirements.
United States4 participantsStarted 2017-08-01
Plain-language 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.
Who can participate
Age range2 Years – 9 Years
SexALL
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Inclusion criteria
✓. Pre-pubescent girls (age 2 years \[12 kg minimum\] to 8 years inclusive; skeletal age \<10 years) or boys (age 2 years \[12 kg\] to 9 years inclusive; skeletal age \<11 years).
✓. Confirmed classic 21-hydroxylase deficiency evident by genotype groups A, A1 or B or clinical course (e.g., adrenal crisis with documented hyperkalemia and hyponatremia, at diagnosis or during a later evaluation; ambiguous genitalia in females). Documentation of one or both parents' genotypes may be required to confirm the subject's genotype.
✓. 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 Upper limit normal (ULN) after 7 days of dosing with doses of hydrocortisone required for physiologic replacement.
✓. At least one parent (or other legally acceptable representative) must sign the informed consent form before the performance of any study procedures, but both parents must sign if both have parental rights. Children who are capable of providing assent (typically 10 years of age and older) must sign an assent form before the performance of any study procedures
Exclusion criteria
✕
What they're measuring
1
Normalization of Serum Androstenedione Level
Timeframe: 7 days
Trial details
NCT IDNCT02574910
SponsorUniversity of Texas Southwestern Medical Center
. Evidence of central puberty: Tanner Stage \>2 for breast development in girls or testicular volume \>4 mL in boys, or random luteinizing hormone (LH) \>0.3 milli-international units (mIU)/mL. Subjects with pubic and/or axillary hair as the only sign of puberty onset will be allowed.
✕. Current or history of hepatitis from any etiology, including history of active viral hepatitis A, B, or C.
✕. Patients with baseline hepatic impairment are excluded from this trial. To be eligible for this protocol, patients must meet all of the following criteria:
✕. Abnormalities of liver function developing during the study
✕. Abnormal renal function tests, defined as blood urea nitrogen (BUN) or creatinine \>1.5 ULN for age.
✕. Significant anemia (hemoglobin \< 12 g/dl). If documented to be due to iron deficiency, subjects may be rescreened 3 months after this has been treated.
✕. Clinically significant abnormality in the 12-lead electrocardiogram (ECG)