Find a Research Study
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.
Postopertive Opioids in Children
The aim is to find out how overall health and wellness is related to need for pain medication after surgery.
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.
Remodulin for the treatment of Persistent Pulmonary Hypertension of the Newborn (PPHN)
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
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.
Safety of UCB-MNC Delivered into the Myocardium in HLHS Subjects
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.
SMART (Sequential Multiple Assignment Randomized Trial) Study
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.
Spruce Adult Study
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.