Clinical Studies

Clinical research is very important because it is one of the best ways to improve patient care. Currently, hundreds of studies are taking place throughout Lurie Children's, including at the research institute, that involve many of our doctors, nurses, clinical research professionals, scientists and technicians.

Search below for a clinical study, or read more on our guide to clinical research for parents and children.

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A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD) ID: NCT01794000

The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease.

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) ID: NCT00214773

The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate...

Carboplatin as a Radiosensitizer in Treating Childhood Ependymoma ID: NCT01088035

This is a phase II study evaluating the feasibility of concurrent carboplatin given with focal radiation therapy in children age 12 months to < 21 years with newly diagnosed localized ependymoma who have no or minimal residual disease post-operatively (< 0.5 cm). The hypothesis is that utilizing carboplatin as a radiosensitizer is feasible and...

Longitudinal Study of Mitochondrial Hepatopathies (MITOHEP) ID: NCT01148550

The specific aims of this study are (1) to determine the clinical phenotypes and natural history of hepatic RC and FAO disorders, (2) to determine the correlation between genotype and phenotype, (3) to determine if circulating biomarkers reflect diagnosis and predict liver disease progression and survival with the native liver, (4) to determine...

An Open-label Phase 2 Study of UX007 (Triheptanoin) in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) ID: NCT01886378

UX007-CL201 is an open-label Phase 2 study to assess the safety and clinical effects of UX007 in subjects with LC-FAOD. Following a 4 week run in period on current therapy, subjects will cross over to daily UX007 treatment for an initial 24 week treatment period, followed by an additional 54 week extension period. Approximately 30 subjects at...