Clinical Research Study Finder

Children's Hospital of Philadelphia leads or participates in hundreds of clinical research studies and clinical trials. Many of the studies that are currently enrolling patients can be found in this database. Use this finder to learn more about the purpose of these studies and clinical trials, find out who can participate, and tell us you're interested in enrolling. You can also look for studies that offer remote participation from home!

Find a Clinical Research Study

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Neuroscience Center Biorepository

In order to understand the conditions that affect the children cared for in the Division of Neurology, we need to better understand underlying mechanisms. This study will enable us to understand how the brain and nervous system work. The goal of this study is to collect biological samples and medical information from children to create a Neuroscience Biorepository to advance future neurological research. Any patient with a neurological condition can participate. Please talk to your provider (Nurse Practitioner or Doctor) about opportunities for your children or their friends to help. 

Natural History of Homocystinuria

The goal of this study is to observe patients with Homocystinuria (CBSDH) over 3 years to learn how their disease is managed under regular circumstances. This study does not involve any investigational medications, but will provide information to researchers who are currently developing a medication to treat the disease. The study has 5 main parts, all of which are provided free of charge to participants: DXA exam, blood draws, ophthalmology exam, cognitive testing, and physical exams. To learn more about the study, please contact our team via the information listed below.

Healthy Controls EndoPAT

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.

ARPKD/HRFD Database

CHOP is partnering with Children's National Medical Center (CNMC) to create a research database of data and blood samples of people who have autosomal polycystic kidney disease (ARPKD) or another hepato/renal fibrocystic disease (HRFD). The purpose is to try to learn more about the causes of these diseases. If you are interested in participating, we will collect data from your medical record to contribute to the database at CNMC. You may also provide an optional blood sample. You may visit arpkdb.org and download the FAQs for you and your child’s doctor or call the CHOP Research Coordinator, Mohini Dutt, at 267-425-3933.

National Pediatric Cardiology Quality Improvement Collaborative

The purpose of this research study is to learn more about improving the treatment and outcomes for patients who have a diagnosis of a single ventricle, such as Hypoplastic Left Heart Syndrome (HLHS) or similar heart defects. You will be asked to fill out a questionnaire called the Ages and Stages Questionnaire about your child. It is used widely in pediatrics to check on babies’ development. We will ask you to fill this out about every three months during your child’s 15 months of life. Also, information will be collected about your child’s surgeries, clinic visits and hospital stays from diagnosis through the first year of life. The information may be used to help us understand an infant's response to surgery and medical treatment and may be used to improve care for children with single ventricles.

Trial of Erenumab in Children and Teens with Migraine less than 15 days per month

This study is a trial comparing the effects of erenumab to placebo in children ages 6-17 years old with migraine less than 15 days per month. Eligible participants will be given either erenumab or placebo, assigned randomly (like the flip of a coin), followed by an open-label phase in which all participants receive erenumab. Erenumab has been FDA-approved for use in adults to decrease the frequency of migraine. Patients may be eligible to participate if they have had migraine for at least one year, and agree to come to CHOP's main hospital for several study visits. Patients whose headache started suddenly (New Daily Persistent Headache), patients whose headache started after a concussion (Post-Traumatic Headache), and patients with severe depression are not eligible for this study.

Friedreich Ataxia Clinical Outcomes Measures

This is a multicenter natural history and clinical measure study. Our objectives are to advance clinical care, research and therapeutic approaches in FA through the development and validation of clinical outcome measures in FA, collection of quantitative serial clinical data on patients and expansion of our established research network. A secondary objective is to build a parallel DNA and RNA repository for use in large scale translational research including modifier gene studies. We wish to also enroll control subjects for a hearing test and sample collection to compare to data from FA patients.

Children's Brain Tumor Network Collection Protocol

The Children’s Brain Tumor Network Collection (CBTN) Protocol is a collaborative, multi-institutional biobanking research program dedicated to the study of childhood brain tumors. The ultimate goal of the CBTN is to improve outcomes for children with brain tumors by offering freely accessible samples and data to researchers around the world. The CBTN collects and stores specimens from pediatric patients that have abnormal cell growth in their central nervous system, such as brain and spinal cord tumors. Only specimens that are no longer needed for the care of the child are accepted. Samples can include tissue, blood, cerebrospinal fluid, and in very rare cases urine and stool. These specimens will be paired with clinical data that will be collected over time on the patient. These samples and data may be shared with researchers to find better outcomes and treatments and may be used in genetic testing. Samples may be used in growing a cell line, which is a small tumor that may be used in an animal model for research and clinical trials. Biological relatives also have an optional opportunity to participate by providing a blood or a saliva sample, or a cheek swab. Subjects and their families also have the option of receiving results back if they are found to be clinically significant to the care of the patient. Private health information will be protected by an electronic system that acts as a gatekeeper between CHOP and researchers and protects the information that can identify someone. There is no compensation for participation.  

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.


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