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Abiraterone Acetate in 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).

Adrenas CAH (Congenital Adrenal Hyperplasia) Study

This is a Phase 1/2, first-in-human, open-label, dose-escalation study designed to evaluate the safety, tolerability, and efficacy of BBP-631 administered to up to 25 adult participants diagnosed with classic congenital adrenal hyperplasia (CAH) (simple virilizing or salt-wasting, Group 1) or with classic salt-wasting CAH (Group 2) due to 21-hydroxylase deficiency (21-OHD) and who are monitored for 24 weeks post-treatment. All participants who receive BBP-631 will be followed for an additional 4.5 years for safety and efficacy in a separate long term follow up protocol (Study CAH-399). In total, all participants will be followed for at least 5 years after the date of treatment with BBP-631.

Burosumab

To collect information on the long-term effects of XLH on children and adults who have participated in burosumab clinical trials, adults and children who have not participated in burosumab clinical trials, and who may or may not have been treated with burosumab during the course of the study.

CTLA (Abatacept) Prevention (TN18)

Abatacept is given as an investigational study drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See TrailNet.org website for more details.

Hydroxychloroquine (HCQ) Prevention (TN22)

Hydroxychloroquine is given as an investigational drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See website for more details https://www.trialnet.org/our-research/prevention-studies/hydroxychloroquine-hcq

MR Imaging of Perinatal Brain Injury

The collection of the research data we hope will help better screening, diagnosing procedures and treatment of brain injury in newborns and identify a connection between MR imaging and neurodevelopmental outcomes.