Pediatrics Testotoxicosis Study [Bicalutamide Anastrozole Treatment for Testotoxicosis] - BATT

Inclusion Criteria

• - Provision of written informed consent of parent/legal guardian and subject assent (as needed by local requirements)
• - Male aged 2 years and over
• - Diagnosis of testotoxicosis based on the following:
• - Clinical features of Progressive sexual precocity documented by Tanner staging and evidence of symmetrical testicular enlargement
• - Clinical features of significantly advanced bone age (defined as bone age of at least 12 months beyond chronological age)
• - Pubertal levels of serum testosterone
• - Prepubertal levels of serum gonadotropins
• - Lack of an increase in serum gonadotropin levels following GnRH stimulation
• - Other pathology excluded by:
• - Undetectable plasma b human chorionic gonadotropin (bHCG). Samples with values below the LOQ will be reported as “<10 IU/L” which in the clinical setting equate to ‘undetectable’.
• - Normal levels of 17-hydroxyprogesterone (17-OHP)
• - Normal levels of dehydroepiandrosterone sulphate (DHEAS)
• - Naive to anti androgen receptor therapy:
• (Note: Ketoconazole and Spironolactone are considered acceptable as is prior use of anastrozole or other aromatase inhibitors)
• - A documented reliable height measurement taken > 6 months prior to study enrollment. Additionally for subjects who have previously received ketoconazole or spironolactone treatment, a documented reliable height measurement taken immediately prior to beginning this treatment.
• (Note: for subjects who received such previous treatment only a single assessment is needed if it was taken immediately prior to beginning treatment and > 6 months prior to study entry)
• - Subjects should be free of endocrine or other effects of previous treatment for testotoxicosis prior to study entry: to ensure this there should be 15 days or 4 drug half lives (whichever is the longer) washout period from prior medication for testotoxicosis.