Abiraterone Acetate in Children with Classic 21-Hydroxylase Deficiency

IRB
CHLA-17-00283
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

The purpose of this study is to (1) determine the minimum effective dose of abiraterone acetate that normalizes androstenedione levels in prepubertal children with CAH secondary to 21-hydroxylase deficiency (Phase 1 trial), and (2) assess the utility of abiraterone acetate in prepubertal children with CAH as adjunctive therapy to minimize excessive androgen secretion and allow more physiological glucocorticoid replacement (Phase 2 trial).

Study Details
Clinical Trials Government Identifier
NCT03548246
Keywords
abiraterone acetate 21-hydroxylase deficiency, CAH, androstenedione, androgen, pre-pubescent
Eligibility
Males or females 2-9 years of age diagnosed with CAH
Does this study also recruit healthy volunteers?
No
Enrollment Status
Open
Coordinator Contact
Norma Martinez
Contact Email
nzmartinez@chla.usc.edu