This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of NBI-74788 versus placebo administered bid with breakfast and the evening meal (doses separated by approximately 12 hours) for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency.
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 trial to help determine which of two gold standard treatments, Medication or Cognitive Behavioral Therapy (CBT), or their combination, is most effective when treating anxiety disorders in children and adolescents. Treatment of every child with an anxiety disorder begins with the question of which treatment to start first, and then what to do if that initial treatment is not as helpful as it should be. This study will help patients, families, and clinicians determine which treatment sequences are most helpful for which specific patients.
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.
Study early development in infants who have an older sibling with autism.
To find out the effects of a chemical in Stevia on the symptoms of NAFLD.
Understand the experiences of patients and parents in treatment decision making for children with Neuromuscular Scoliosis (NMS).
To better understand the effects of nutrition in early pregnancy on infant growth and development.
The purpose of the study is to learn about how to improve access to care during adolescence/young adulthood and to see if Virtual Team Clinic is an acceptable form of care. Virtual Team Clinic is a new approach to provide care to (middle/high) school aged patients living with Type 1 Diabetes and their families. This new approach consists of Virtual Team Clinic appointments (primarily telemedicine, and/or in person as necessary). The study also incorporates group visits/medical appointments with a multidisciplinary care team (e.g., RD, RN, Social Worker, etc.). Each group will have a special theme and learning experience. You will learn how to better take care of your diabetes, get support from others, and hopefully have fun.
To explore the possibilities of providing weight management services to teenagers through technology.
