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.

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

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.

The purpose of this study is to find out if myofascial release therapy (MFR), (a massage method that will be used in the belly area), will improve the symptoms in CHOP patients with Irritable Bowel Syndrome (IBS). Participation will last approximately 8 months which includes in person visits and remote follow-up (surveys only). The MFR intervention is comprised of 6 once a week in-person visits that will be conducted by a licensed massage therapist. This therapist will train participants in order for them to self-administer MFR therapy at home, daily. Participants will be asked to record their IBS symptoms throughout their study participation.

This study looks to assess how celiac disease affects the quality of life of patients between the ages of 13-18. We are looking to recruit 100 patients who are in their first year of diagnosis and their parents. If the above criteria apply to you, you may be eligible to take part in the study.

The goal of this study is to identify and measure anxiety symptoms in children and adolescents with food allergy. We are looking for families of children with IgE-mediated food allergies between 8 and 18 years of age to participate. The study procedures involve both the caregiver and child filling out online questionnaires on two separate occasions. If you are interested in participating, you can sign up immediately by clicking the link below: https://is.gd/FoodAllergyAnxiety If you have any questions, please contact the research coordinator, Leah Wilkey, by phone at 215-590-6198 or email at wilkeyl@email.chop.edu.

The purpose of this study is to evaluate how safe and effective the drug Qsymia (Phentermine/Topiramate of Ph/T) is for children, adolescents, and young adults with hypothalamic obesity. We will also evaluate if Ph/T helps with weight loss and changes in hunger in patients with hypothalamic obesity.

If you are determined to be eligible, then your participation will last for approximately 8 months. Participants will be asked to complete up to 5 in-person visits and 5 telephone safety check-ins. During the study you will be randomly assigned to study drug (phentermine/topiramate) or placebo for 28 weeks.

You will complete study procedures including blood tests, ECGs, mobile cardiac outpatient telemetry, body composition scan, and questionnaires. Compensation and reimbursement of limited travel expenses is available.

The DISCOVERY Study is a new, national effort to learn about the causes of type 2 diabetes in children. DISCOVERY is for children 9-14 years old who are at higher risk of developing type 2 diabetes. We want to figure out why some children get type 2 diabetes and others do not.