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ImproveCareNow (ICN)

To improve patient care and achieve the best health outcomes possible for children with IBD.

LifeSkills Mobile

5,000 transgender women are testing a LifeSkills Mobile intervention web-app to see if it helps reduce condomless sex, increase PrEP use, and decrease HIV infections over several years.

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.

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.

Prader-Willi Syndrome (PWS)

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

Pyeloplasty Database

There is currently a lack of information on critical questions surrounding laterality of ureteropelvic junction obstruction (UPJO), the difference between extrinsic and intrinsic obstruction, the utility of retrograde pyelogram, and the appropriate management for patients with concurrent UPJO and ureterovesical junction obstruction (UVJO). Therefore, we propose first a retrospective study of all pyeloplasty’s completed at CHLA for UPJO to answer some of these critical questions. The results of this study may inform future studies in this realm. We will retrospectively review all cases of pyeloplasty completed at CHLA (from 2000 through present) to identify differences in outcomes.