Research Studies

Division of Neurology Research Studies

Find research studies available to children cared for by the Division of Neurology team.
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Clinical trial
Children (6)
Adults (2)
One-Time Visit Studies (2)
Healthy Controls (1)
MRI/Imaging Studies (1)

Clinical Trial Phases
Phase II (1)
Phase III (2)
N/A (3)

Recruitment Status
Recruiting (5)
Non-recruiting (1)

Related Specialties and Programs
Division of Neurology (6)
Department of Pediatrics (1)
Neuroscience Center (1)

Specialty
General Pediatrics (1)
Neurology (6)

AxD Outcomes
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. 

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, MRI/Imaging Studies

LeukoSEQ: First-Line Whole Genome Sequencing in Leukodystrophies
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.

Phase: N/A

Actively recruiting: Yes

Category: Children

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. 

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, Healthy Controls, One-Time Visit Studies

Safety and Efficacy of NB-001 in 22q11DS Children with Anxiety, Inattention, and/or Autism
The aim of this study is to determine if NB-001 (an investigational drug that is not approved by the FDA) is effective, safe, and well tolerated in treating 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.  

Phase: Phase II

Actively recruiting: No

Category: Children, One-Time Visit Studies

Trial of Erenumab for Preventive Treatment of Pediatric Migraine 15 days Per Month or More
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.

Phase: Phase III

Actively recruiting: Yes

Category: Children

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.

Phase: Phase III

Actively recruiting: Yes

Category: Children