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Research Studies Finder

Research Studies Finder

Use this finder to find a study or trial that is the right fit for your child. Also, filter results by phase — which are the specific steps researchers take throughout the trial to find answers, solutions and cures.

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Showing 1 - 10 of 46 results

RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

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.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Children

Weight Trajectory with ETI

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.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, One-Time Visit Studies

SLEEPY 2.0

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.

Phase: N/A

Actively recruiting: Yes

Category: Children

Neurophysiological Biomarkers in Rett Syndrome

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
All participants are compensated for their time and travel.

Phase: N/A

Actively recruiting: Yes

Category: Children, Healthy Controls

Brains Keep Changing

We want to study how brain function and structure change in childhood. We will compare brain measures in children with and without autism spectrum disorder (ASD) who are between the ages of 10-13 years old.

Phase: Pilot

Actively recruiting: Yes

Category: Children, Healthy Controls, MRI/Imaging Studies

CROSSING

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.

Phase: Phase III

Actively recruiting: Yes

Category: Adults, Children

Myofascial release therapy in IBS

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.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

Tara-002 in Lymphatic Malformations

This study is for people with growing abnormal lymph vessels called lymphatic malformations (LMs). This study has 3 parts and involves taking a study drug called TARA-002. The purpose of this study is to find out if the study drug is safe and effective (can help) people with LMs. Participants receive the study drug by injection and will complete other research assessments throughout the study.

Phase: Phase II

Actively recruiting: Yes

Category: Children

Neurometabolic Profile of Tobacco Smoking in HIV-Infected Individuals

HIV infection can cause harm to the brain and negatively affect memory, learning, and decision making in daily activities. Smoking may affect the ability of medications to reduce HIV in the bloodstream (viral load), which may affect long-term survival and brain health. This study will look for changes in the brain that might have occurred due to HIV infection or smoking.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Healthy Controls, MRI/Imaging Studies, One-Time Visit Studies

Sleep and Growth Study 2

The Childrens Hospital of Philadelphia is seekinghealthy adolescents between the ages of 12-13, who are currently in the 7thgrade,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 emails_grow@chop.edu.

Phase: Phase I

Actively recruiting: No

Category: Children, Healthy Controls

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