The first Phase IIb clinical trial in Down syndrome regression disorder (DSRD). An open-label, randomized study comparing the safety and efficacy of lorazepam, intravenous immunoglobulin (IVIg) and the Janus Kinase inhibitor (JAK inhibitor), tofacitinib.
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.
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.
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.
