Cure Glomerulonephropathy Network (CureGN) is a multi-year study of 2,400 children and adults with four rare glomerular (kidney) diseases at over 50 centers in the US, Canada, and Italy. The purpose of CureGN is to create a patient research network to learn about four major causes of glomerular disease : 1. Minimal Change Disease (MCD) 2. Focal Segmental Glomerulosclerosis (FSGS) 3. Membranous Nephropathy (MN) 4. IgA Nephropathy (IgAN)

The purpose of this research study is to 1) validate the use of surveys such as fatigue scales, Mitochondrial Disease scales, and quality of life scales, 2) validate clinical exams, such as muscle strength tests, and the 6 minute walk test, and 3) to define the natural history of the disease in the Mitochondrial Disease patient population.

Our team is working on a study to better understand neurological problems, including seizures and developmental delays, that occur in children with hyperinsulinism, including HI/HA syndrome, and type 1 diabetes mellitus. The study involves a one day visit to The Children’s Hospital of Philadelphia and The University of Pennsylvania for a specialized magnetic resonance imaging scan (MRI) of the brain, an electroencephalogram (EEG), blood draw, and completion of questionnaires to evaluate development and behavior. The entire study visit is expected to take about five hours. If you are interested in learning more, please contact us at HIResearch@email.chop.edu.

The purpose of this research study is to test how people with cystic fibrosis process sugar and learn more about their risk of developing cystic fibrosis-related diabetes. We will also test the body's ability to make insulin in response to sugar. 

CHOP, in collaboration with Onyx Therapeutics, Inc., is conducting a study for children and young adults up to 21 years old, with relapsed or refractory acute lymphoblastic leukemia. This clinical trial is adding the study drug, Carfilzomib, to a standard relapse chemotherapy regimen. For more information about this study, including specific eligibility criteria, please visit clinicaltrials.gov and search for "NCT02303821". To hear more about this and other available trails or get any questions answered please contact our Cancer Intake Specialist by phone at 267-426-0762 or email Oncointake@email.chop.edu.  

The main objective of this study is to see if repeat subcutaneous doses of dupilumab, the study medicine, helps to reduce eosinophilic inflammation in the stomach of participants with eosinophilic gastritis/ eosinophilic gastroenteritis compared to a placebo. A placebo is a pill or substance that is given to a patient like a drug but that has no physical effect on the patient.

This study involves taking a study drug called CUDC-907.  CUDC-907 is an oral drug that blocks certain proteins in tumor cells; these proteins may be important in the growth of some cancers. The overall goals of this study are to evaluate the side effects and benefits of CUDC-907 in patients with relapsed or treatment refractory solid tumor, brain tumor, or lymphoma, and to determine the best dose of CUDC-907 for children and adolescents.  This study will also look at the levels of CUDC-907 in the blood over time after a dose. 

The purpose of this study is to create a biorepository, which is a collection of biological samples and the data associated with them, to support our ongoing and future studies on ultra-rare pediatric neurodegenerative disorders. These include disorders associated with mitochondrial and autophagic dysfunction such as TBCK, congenital disorders of autophagy, mitochondrial disorders, and cases of neurodegeneration without known genetic diagnoses. Ultra-rare disorders affect 1 in every 50,000 kids.

Using the biological samples we collect, we intend to derive cellular disease models to study these disorders at a molecular level and screen for therapeutic targets. We also intend to look for biomarkers that may inform outcome measures in future interventions and clinical trials. By looking at the cells in these biological samples, we hope to understand how they work, what is wrong with them, and how we could fix them. The overarching aim is to yield generalizable knowledge about these disorders and translate it into better diagnostic awareness and clinical care.

Are you interested in participating in a research study about bone health in children and adolescents at high risk of suffering a broken bone? Dr. Weber at CHOP is looking for people between 5-19 years old with a genetic or a long-term medical condition affecting bone strength to participate in this study. We are also recruiting children between 5-19 years old with no medical conditions as healthy participants in the study. Participation will require a single visit at The Children’s Hospital of Philadelphia, which will include bone scans, a muscle strength evaluation by a physical therapist, blood and urine tests, and questionnaires. Eligible participants who complete the study will be compensated for their time.