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NEAT (Neuroimaging: Emotions and Appetite Testing) Study

The NEAT Study is about obesity, appetite, emotions, and how the brain develops in children. The study team is looking for volunteer youth to participate who are either healthy or have a clinical condition called Congenital Adrenal Hyperplasia (CAH).

Neurocrine Ped Study

This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered twice daily (bid) with breakfast and evening meals for28 weeks in approximately 81 pediatric subjects with classic congenital adrenal hyperplasia (CAH) due to21-hydroxylase deficiency. Eligible subjects will be randomly assigned in a 2:1 ratio (active:placebo) to either crinecerfont (25 mg bid via oral solution for subjects 10 to <20 kg, 50 mg bid via oral solution for subjects 20 to <55 kg, or 100 mg bid via oral capsules for subjects ≥55 kg) or matching placebo (oral solution placebo for subjects <55 kg and oral capsule placebo for subjects ≥55 kg). Dose assignment from Day 1 to Week 28 will be based on the subject’s weight at Day 1. After the 28-week placebo-controlled treatment period, there will be a 24-week, open-label treatment period, during which all subjects will receive crinecerfont at doses based on their Week 28 body weight.

Ophthalmologic disorders and eye tracking in children with autism spectrum disorder (ASD)

We are conducting a research study about visual problems in children with Autism Spectrum Disorder (ASD). We believe that many children with autism may have undiagnosed eye problems because they cannot explain when they have trouble seeing and may not undergo routine eye exams. We hope to develop a method to screen for visual problems in using a new, non-invasive technology called eye tracking. We will use a camera to record your child’s eye movements as they watch video clips for 15-20 minutes.

Pathway to Prevention

1. To identify family members of people with type 1 diabetes that may be at risk for developing type 1 diabetes. 2. To monitor the metabolic and immunologic status of at-risk individuals. Eligible individuals will be offered enrollment in type 1 diabetes prevention studies. See website for more details https://www.trialnet.org/

Perceptions of Long Acting Reversible Contraception (LARC) among Adolescent and Young Adults wth chronic illness

This is an observational study that employs qualitative methods (13 focus groups and 22 individual interviews) targeting 130 sexually active and non-sexually active adolescent and young adult (AYA) females, living with chronic illness or disability. The study is designed to explore the extent to which their experiences living with chronic illness and engagement in ongoing medical management has an impact on their contraception decision making and perception of LARC as a viable option.

Phase I Temsirolimus

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.

Prader-Willi Syndrome (PWS)

This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.