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
To determine if MEDI8897 reduces the amount of visits to see a medical professional due to a lower respiratory tract infection caused by RSV. We will be looking at this in healthy preterm infants entering their first RSV session.
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.
Develop non-invasive measures to identify abnormal placental function in babies with and without congenital anomalies.
To test a new experimental drug Temisirolimus - Temsirolimus (also known as Torisel®) is approved for kidney cell cancer treatment in adults - in combination with approved chemotherapy drugs - in the hopes of finding a drug combination that may be effective against leukemia and non-hodgkin’s lymphoma that has come back after initial treatment. To find the highest dose that can be given without casing severe side effects.
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.
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
To determine the safety and feasibility of autologous umbilical cord blood-derived mononuclear cells delivered into the myocardium of the right ventricle during planned Stage II Glen surgical procedure for individuals with hypoplastic left heart syndrome.
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.
