Research Studies Finder

The purpose of this study is to look at the safety and tolerability of RBX-121, a one-time gene therapy, in subjects with MPS II (Hunter Syndrome). You may be eligible for this study if you are a male between the ages of 4 months to 5 years old. Reimbursement for travel and research procedures will be provided. This study involves blood draws, interviews, urine collection, genetic testing, general anesthesia, lumbar punctures, MRI, gene therapy, lumbar puncture, ultrasound, ECG/echocardiogram and physical and neurological examinations. If you have questions or would like to learn more about the study, please contact the study team at MetabolismResearch@email.chop.edu.

Use of elexacaftor-texacaftor-ivacaftor ("ETI"), also known as Trikafta, is associated with weight gain in most people with cystic fibrosis (CF) who take this medication. However, some people gain more or less weight than is expected, and this can be either beneficial or problematic based on the individual's starting weight. The goal of this study is to compare factors between children and young adults with CF who have either had robust or minimal weight gain after one year of treatment with ETI.This observational study requires one study visit to CHOP that will last approximately 6 hours.

The goal of this study is to find out if we can develop a digital health approach that can increase sleep duration and improve growth outcomes in children.

This purpose of this study is to determine the efficacy and safety of tisagenlecleucel (also referred to as CTL019, or Kymriah) in children, adolescents and young adults with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL). The study is enrolling children and young adults up to age 25 with CD19-expressing mature B-cell NHL, including Burkitt leukemia/lymphoma, that has relapsed after one or more prior therapies or is primary refractory (not achieved a complete remission (CR) or partial response (PR) after the first line of therapy). This study involves the administration of the study drug, blood draws, assessment of lymphoma response, and chart reviews. After a process called leukapheresis, in which T cells (a type of white blood cell) are removed from the subject's blood and modified in order to target the lymphoma, they are returned back to the subject as the study drug. Reimbursement for travel may be provided. If you have questions or would like to learn more about the study, please contact the Cancer Immunotherapy Program at (215) 425-7193. The Principal Investigator is Dr. Shannon Maude.

The PRISM study is looking to understand what happens to children and young adults who get infected with SARS-CoV-2 and how their immune system responds. To do this, we are examining blood, urine, stool, saliva, and nasal swab samples, as well asinformation related to SARS-CoV-2 infection and MIS-C, collected from subjects.We will also study subjects' results onbreathing tests, echocardiograms, electrocardiograms, and a chest CT scan. Participation lasts for up to 1 year and includes in-person, telephone, and video study visits.

This study aims to support familieswith buying, cooking, and eating fresh fruits and vegetables at home! The goal of this study is to learn how fruit and vegetable vouchers and virtual cooking classes and resources affect families' experiences with home cooking and food shopping, as well as caregiver mental health.

The purpose of this research study is to learn more about Graves disease and PTC/Hypothyroidism, How it effects brain function and brain function changes after starting medical treatment.

The purpose of this study to learn more about the brain function of females with Rett Syndrome and how it compares to the brain function of typically developing females. The primary investigator of the study is Dr. Eric Marsh. For our research to be successful we need to partner with families who have a typically developing daughter between the age of 1 and 17. If your child agrees to help us with our study she will be asked to:

• Complete intelligence testing
• Receive an Evoked Potential measurement - this is a non-invasive procedure similar to an EEG.
• Complete yearly follow up visits

The purpose of this research study is to learn more about the normal function of blood vessels in healthy children and adolescents. This will help us discover differences between healthy individuals and children with lupus. Healthy control participants will be asked to make 1 visit to CHOP to complete a brief survey, a fasting blood draw and a test to measure the blood pressure in your fingers.

A study to investigate changes of symptoms and esophageal cell structure with tezepelumab compared with placebo in patients aged 12 to 80 years old with eosinophilic esophagitis. Subjects up to 21 years of age will be enrolled at CHOP.