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RDA Study

This study examines the relationship between social and nonsocial reward responsiveness and depression symptoms in adolescents (14-17 years old) with confirmed or suspected autism. Over time, we hope to understand mental health outcomes in teens (ages 14-17) with confirmed or suspected autism.

RDA-ID

Over a 6-month period, adolescents and their parents will participate in clinical interviews, EEG tasks, and surveys about depression, mental health, and friendships, among other topics. Over time, we hope to understand depression and mental health outcomes in autistic adolescents with intellectual disability (14-17 years old) and their parents.

Spruce Adult Study

This is a randomized, double-blind, placebo-controlled study that will evaluate the potential of tildacerfont to reduce GC burden in adult subjects with classic CAH who have LLD ≤ A4 ≤ 2.5x ULN and are on supraphysiologic doses of GC therapy (≥30 mg/day and ≤60 mg/day HCe). This will be the first study of tildacerfont to evaluate GC dose reduction. In addition, this study will characterize clinical outcomes after up to 76 weeks of treatment with tildacerfont. An optional Open-Label Extension Period will provide an open-label treatment with tildacerfont at 200 mg QD for up to 240 weeks.

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.

MRI of placental function

Develop non-invasive measures to identify abnormal placental function in babies with and without congenital anomalies.

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).