Find research studies available to children cared for by the Division of Neurology team.
A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease. These results will be crucial to our understanding of disease presentation and progression. Providing this information about the pathophysiology will allow for effective development of clinical trials to test therapies and the future use of neuroprotective agents in children.
While recent research has demonstrated that whole genome sequencing is a powerful first-line diagnostic tool, important questions remain around its long-term impact on downstream clinical management approaches. The investigators hope to address these questions by way of chart review and survey administration in a small population of suspected leukodystrophy patients who receive whole genome sequencing as part of their clinical care.
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:
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.
The aim of this study is to determine ifNB-001 (an investigational drug that is not approved by the FDA) is effective, safe, and well tolerated intreating children and adolescents with 22q11DS and anxiety, inattention, and/or autism. The study will take place over a 21-week period with routine telemedicine check-ins, home health nurse visits, and behavioral questionnaires. Except for the first visit which will be in-person at the CHOP Philadelphia campus, all other study visits will be done at the patient's home by a study nurse.
The purpose of this study is to look at the Sodium Magnetic Resonance Imaging (MRI) findings in children with brain tumors and healthy children. An MRI is a machine which takes pictures of the brain. The type of MRI image in this study is a new type that is not yet FDA approved and the study is looking to see if the new type of MRI image is useful in looking at sodium in the brain and brain tumors. Sodium MRI (also known as 23Na-MRI) is a useful non-invasive imaging technique to assess biochemical and physiologic changes in tissues, e.g. integrity of cells and tissue viability. Sodium MRI has been beneficial in the evaluation of several neurological disorders including acute ischemic strokes, multiple sclerosis, amyotrophic lateral sclerosis, migraines, and multiple tumor types, including tumors outside of the central nervous system.
We want to study brain responses during simulated driving and are currently looking for participants 12-17 years old or those who have had brain surgery. To do so, we'll have eligible participants come to CHOP for some non-invasive (safe) brain scans and drive using a simulator. If you are interested in learning more about the study, please contact Victoria Kaufman at kaufmanv@chop.edu or 267-425-4150.
This study is a trial comparing the effects of erenumab to placebo in children ages 6-17 years old with migraine 15 days per month or more. 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.
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.