NEPTUNE (Nephrotic Syndrome Study Network)
To advance research on Minimal Change Disease (MCD), Focal and Segmental Glomerulosclerosis (FSGS), and Membranous Nephropathy (MN) that define nephrotic syndrome.
To advance research on Minimal Change Disease (MCD), Focal and Segmental Glomerulosclerosis (FSGS), and Membranous Nephropathy (MN) that define nephrotic syndrome.
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.
To find out which of two commonly used IV fluids given in the Emergency Department for sepsis is most effective.
Two main purposes of the study: To see if serious bacterial infections occur less than once a year in each study subject. This will determine if study drug is effective. Also to find out if study drug is safe and tolerable for study subjects.
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.
To find out the effects of tenofovir alafenamide (TAF) on children and adolescents with chronic hepatitis B.
Carfilzomib (also known as Kyprolis®) is approved to be used in certain adult patients with relapsed or refractory multiple myeloma (myeloma that returns after a successful course of treatment or myeloma that does not respond to treatment). Multiple myeloma is a type of blood cancer. Carfilzomib is not approved to treat ALL. It has not been given in combination with the other drugs used in this study. This study is being done to find out if carfilzomib can be safely given before and during treatment with standard chemotherapy drugs.
The goals of this study are to improve survival rates in children and young adults with relapsed AML through the combination of DEC and VOR.
This study aims to evaluate the side effects (good and bad) of giving Vincristine Suflate Liposome Infection (also known as Marqibo®) - a drug approved for leukemia treatment in adults but that is experimental in children - with other standard chemotherapy drugs and to measure how effective the combination is at treating children and young adults with leukemia that has come back after initial treatment.
The primary aim of this registry will be to identify genes and/or biologic and environmental factors that either cause these tumors or increase one's risk for developing them. Ultimately, research using this registry may result in improved diagnosis, more effective treatments and possibly prevention.