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.

Inflammatory Bowel Disease (IBD) can sometimes add stress to your life and that may affect your physical symptoms of pain and stool habits. In this study, we want to know if practicing mindfulness via an online Mindfulness-based stress reduction (MBSR) course taught by a trained teacher can help with your IBD. If you are a patient with IBD, who is between 12-19 years of age, you might be eligible to participate in an 8 week long MBSR group course online. You will answer a series of questionnaires and submit stool samples 3 times over the course of the study, and you will also participate in 1 focus group to discuss your experience in the study. You will be compensated for your participation.

CHOP, in collaboration with Dana-Farber Cancer Institute, is conducting a study for children, adolescents, and young adults up to 31 years old, with relapsed or refractory acute lymphoblastic leukemia. This clinical trial is adding the study drugs ribociclib and everolimus to a standard relapse chemotherapy regimen. For more information about this study, including specific eligibility criteria, please visit clinicaltrials.gov. 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.

This study will evaluate the safety and tolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with a neurocognitive deficit that are at least 4 months old. This study involves administration of the RGX-111, general anesthesia, lumbar puncture, blood draws, MRI, ultrasound, electrocardiogram, echocardiogram, hearing test, and chart review. If you have questions or would like to learn more about the study, please contact the Principal Investigator, Dr. Ficicioglu at (215) 590-3376. 

The Congenital Hyperinsulinism Center at the Children’s Hospital of Philadelphia is working on a research study to better understand how people with hyperinsulinism may have different blood sugar responses to certain tests (like fasting or drinking a high-protein shake) when compared to people without hyperinsulinism. This study will involve a screening visit where we will ask you about your medical history and any known episodes of low blood sugar. Depending on your responses during the interview, you will be asked to complete up to five tests at our outpatient research center. These tests include fasting, drinking a high-protein shake, drinking a high-sugar drink, eating a regular meal, and exercising on a stationary bike. We will collect blood samples from an IV throughout the tests to measure certain blood levels like glucose and insulin. You will be compensated for some or all of your travel costs and you will receive payment for your time. If you are interested in learning more, please contact us at HIResearch@chop.edu.

The Children’s Hospital of Philadelphia is seeking healthy adolescents between the ages of 12-13, who are currently in the 7^th grade, to participate in the Sleep and Growth Study 2. The purpose of this research is to learn more about the changes in sleep and body size in adolescents as they transition from middle to high school. Participants will be asked to visit CHOP so that we can measure their bone density. Following this visit, participants will wear a sleep tracker over a 2-week period and speak to a team member by phone on three occasions about the types of food eaten in the previous 24 hours. If you and your child agree to participate, the study will involve 3 visits over approximately 2 years. All participants are compensated for their time and effort. For more information and study details, please call 215-590-2383, ext. 1, or email s_grow@chop.edu. 

The Craniofacial Pathologies biobank is a research study collecting and storing reamining surgical specimens from the cranium and face from diagnosed patients at CHOP with a congenital craniofacial anomaly. The main purpose of this longitudinal study is to help researchers discover the causes of craniofacial pathologies that may lead to new treatments. 

The purpose of this study is to look at the safety and tolerability (how well you will react) of the study drug (HM15136) and to determine if it is effective for the treatment of Congenital Hyperinsulinism (HI). HM15136 is an experimental drug which is not approved by the FDA for the treatment of HI. HM15136 is stable and has a long effect in the body, only requiring it to be given (by injection) once a week.  HM15136 is designed to act like glucagon, a hormone produced by the pancreas that helps the body maintain normal blood sugar levels by increasing the glucose produced in the liver and breaking down glycogen (a form of stored glucose) into the usable glucose form.

Participation lasts for up to 22 weeks and will involve up to 13 study visits. Study procedures include taking the study drug for 8 weeks, having electrocardiograms, wearing a continuous glucose monitor (CGM) and using a handheld glucose meter, having blood and urine tests, and completing an electronic diary.

The main risks of this study include: nausea, vomiting, diarrhea, abdominal pain, injection site reactions (swelling, itching, bleeding, or pain), loss of appetite, weight loss, high blood sugar. You may benefit if the study drug proves to be more effective than your current treatment plan in treating low blood sugar.