Research Program

The first year of neurology training includes 8 weeks of research time that will serve as a starting point for a research project that is to be completed by graduation. The division chief, program directors, Director of Clinical Research and program faculty are available to help guide residents in their selections of mentors and projects. Opportunities for clinical research within the Division of Child Neurology include projects in epilepsy, headache, inflammatory nervous symptom disease, metabolism, neuro-genetics, neuroimaging, neuromuscular disorders and childhood stroke as well as projects from other affiliated departments.

The Research Program includes a monthly seminar series designed to teach fundamental approaches for basic and clinical research, and to provide a venue for the residents to present and discuss the findings of their projects. Residents are encouraged to prepare their projects for presentation at national meetings and for publication. 

Neurology Research Studies

Learn more about the researchers in the Division of Child Neurology and our clinical studies

Current Child Neurology Research Studies

Epilepsy:

• Neural plasticity and recovery of visual functions in patients with hemispherectomy (Patterson)

• Open-label, single-arm, multicenter, long-term follow-up study to evaluate safety and efficacy of brivaracetam used as adjunctive treatment in pediatric subjects with epilepsy (Vaisleib)

• A randomized, double-blind, placebo-controlled study of the safety and efficacy of intranasal midazolam (usl261) in the outpatient treatment of subjects with seizure clusters (Vaisleib)
• Computerized cognitive assessment of medication effects in newly diagnosed patients with epilepsy (Asato)

• Regional Approaches to Improve Access to Services for Children with Epilepsy (Asato)

• Established Status Epilepticus Treatment Trial (Sogawa)

• Prospective observational study of epileptic encephalopathy (Sogawa)

• The efficacy of Vagus Nerve Stimulator on status epilepticus (Sogawa, Sitwat)

• A study on the impact of early genetic testing in infantile onset epilepsy (Sogawa)

• Effectiveness of Ketamine vs. Pentobarbital for Refractory Status Epilepticus in Children; a retrospective multi-hospital database study (Sogawa)

• The efficacy of vagus nerve stimulator on primary generalized epilepsy (Sogawa, Sitwat)

Neuro-Muscular:

• Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-us-201 (Abdel-Hamid)

• An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy (Abdel-Hamid)

• A Phase 3 efficacy and safety study of Ataluren (PTC124) in patients with nonsense mutation dystrophy (enable the ribosome to read through premature nonsense stop signals on mRNA) (Abdel-Hamid)

• A phase 2 randomized, double-blind, placebo-controlled, multiple ascending dose study to evaluate the Safety, efficacy, pharmacokinetics and pharmacodynamics of pf-06252616 in ambulatory boys with DMD. (monoclonal antibody against myostatin which blocks muscle differentiation and growth. (Abdel-Hamid)

• A randomized, double-blind, placebo-controlled, multiple ascending subcutaneous dose to evaluate the safety, tolerability and pharmcokinetics of BMS-986089 in DMD (myostatin inhibitor) (Abdel-Hamid)

• An open label access to provide treatment with MP-104 (deflazacort) to DMD patients (Abdel-Hamid)

• A 48-Week, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with DMD (Abdel-Hamid)

Neuro-Metabolic:

• Triheptanoin/Long-Chain Fatty Acid Oxidation Disorders (Goldstein)

• 1 P NMR Evaluation of ATP Production in Individuals with Mitochondrial Disease (Goldstein)

• North American Mitochondrial Disease Consortium (NAMDC) (Goldstein)

• A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Mitochondrial Myopathy (Goldstein)

• A Phase 1/2 Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study Investigating the Safety, Tolerability, and Efficacy of Intravenous MTP-131 for the Treatment of Mitochondrial Myopathy in Subjects with Genetically Confirmed Mitochondrial Disease (Goldstein)

• An Open Label Extension Study of HGT-HIT 045 Evaluating long term Safety and Clinical Outcomes of Intrathecal Idursulfase- IT administered in conjunction with intraveneous Elaprasen in pediatric patients with Hunter Syndrome and cognitive impairment (Goldstein)

Neuro-Genetics:

• Neurogenetics Program Patient Registry: Clinical and Genetic Diagnosis, Natural History Study, Translational Research and Biorepository (Rajan, Goldstein)

• Functional characterization of GEMIN5 - Establishing an gene discovery pipeline from variants of unknown significance on next generation sequencing (Rajan, Goldstein, Pandey)

Neuro-Development:

• Effects of AFQ056 on Language Learning in Young Children with Fragile X Syndrome (FXS) (Filipink)

• Fragile X Clinical and Research Consortium (FXCRC) Registry (FORWARD)

• LEND: The UCLID CENTER at the University of Pittsburgh-LEND Training Outcomes Study (Asato, Noll)

• Demographic Factors and Sleep in Children with Autism (Elkhatib, Asato)

• Assessing Knowledge Base and Needs for Supporting Transition Education in Autism (Jindal)

Behavioral Health:

• Non-inferiority trial of Bright IDEAS Problem-Solving Skills Training (PSST) comparing face-to-face PSST to a recently developed electronic version (ePSST) for caregivers of children diagnosed with cancer (Noll)

• Dissemination science trial (R25e) to train 200 psychosocial professionals (psychologists, social workers, advanced practice nurses) who work in pediatric oncology centers how to deliver PSST (Noll)

• To assess children enrolled on the current COG high-risk ALL phase 3 trial with computerized assessment tools and standardized psychoeducational tools over time to determine if early pharmacologic or behavioral interventions mitigate psychological problems (Noll)

• Pilot feasibility trial of Bright IDEAS PSST in pediatric rehabilitation medicine

• Two site pilot feasibility trial of Bright IDEAS PSST in pediatric sickle cell disease (SCD) clinic.

Neuro-Immunology:

• Acute Demyelinating Disorders of Childhood (ADEM) (Alper)

• Acute brain stem clinical and radiological syndromes in children (Thakkar)

• Neuropsychological outcomes in immune mediated brain disorders (Thakkar)

Stroke:

• Regional Coordinating Stroke Center of Stroke Trials Network (NINDS) (Cummings)

Neuro-Radiology:

• Multimodal imaging diagnostic approach with analysis of ASL, single voxel MRS and Diffusion in patients with POLG mutations (Goldstein, Asato, Zuccoli)

• Perfusion changes in hemiplegic migraine (Munjal, Cummings, Safier, Zuccoli)

• Relationship between vasogenic edema and perfusion imaging in patients with PRES (Heredia, Zuccoli)

• Brainstem presentation of pediatric MS (Shelton, Alper, Zuccoli)

• Role of SWI imaging in abusive head trauma (Wilson, Zuccoli)

Basic Science:

• Cellular and Molecular Mechanisms of FUS-related neurodegeneration (Pandey)

• Molecular Basis of FUS/TLS-related amyotrophic lateral sclerosis (Pandey)

• Molecular library screen for suppressors of FUS proteinopathy (Pandey)

• A Drosophila model to investigate the role of FUS in ALS (Pandey)

• Changes in neonatal brain structure and gene expression associated with prenatal administration of glucocorticoids (Franks)

• Viral immunotherapy to eradicate subclinical brain metastases (Bergman)

• Targeted Recombinant Vesicular Stomatitis Virus to Treat Breast Cancer Metastases (Bergman)