Neurosciences research projects

Brain Injury / Neurocritical Care

Accuracy of perceptions of functional outcome of pediatric neurocritical care patients with severe traumatic brain injury (sTBI).

• A multidisciplinary study where the goal is to assess how accurate the medical predications are for children hospitalized for severe traumatic brain injuries. Dr. Raquel Farias-Moeller is comparing the medical predications for these children's outcomes from providers and physicians in different specialties.

Neuro critical care COVID-19 manifestations pediatric study

• A medical chart review study to determine the frequency and presence of neurological symptoms and diagnoses in children hospitalized with confirmed or suspected COVID-19.

Duchenne Muscular Dystrophy

A double-blind, placebo-controlled, multi-center study with an open-label extension to evaluate the efficacy and safety of SRP-4045 and SRP-4053 in patients with Duchenne Muscular Dystrophy (Sarepta Therapeutics, 4045-301)

• A clinical trial to evaluate the effects of the drugs casimersen (SRP-4045) and golodirsen (SRP-4053) in children diagnosed with Duchenne Muscular Dystrophy (DMD).

Long-term, open-label extension study for patients with Duchenne Muscular Dystrophy enrolled in clinical trials evaluating casimersen or golodirsen (Sarepta Therapeutics, 4045-302)

• A clinical trial to evaluate the long-term effects of the drugs casimersen (SRP-4045) and golodirsen (SRP-4053) in children diagnosed with Duchenne Muscular Dystrophy (DMD).

A multi-national, randomized, double-blind, placebo-controlled systemic gene delivery study to evaluate the safety, tolerability, and efficacy of SRP-9001 in subjects with Duchenne Muscular Dystrophy who are aged 4-7 years (Sarepta Therapeutics, SRP-9001-301)

• A clinical trial to evaluate the effects of a micro-dystrophin gene therapy drug (SRP-9001) in children diagnosed with Duchenne Muscular Dystrophy (DMD).

Open-label extension of the HOPE-2 Duchenne Muscular Dystrophy trial (Capricor, Inc., HOPE-2-OLE)

• A clinical trial to evaluate the long-term effects of a treatment using cardiosphere-derived (heart) cells in children diagnosed with Duchenne Muscular Dystrophy (DMD)

Epilepsy

Retrospective evaluation of utility of testing in pediatric patients presenting to the emergency room for first time seizure

• A project where the goal is to review medical records on brain scans (CT and MRI scans) and blood test results to determine how effective these types of tests are in evaluating children with first-time seizures with an unknown cause.

Incidence of epilepsy in a critical care neurology consult service

• A project where medical records are reviewed to look at the frequency of seizures (epilepsy) in children who have survived a neurological critical care illness or trauma. The goal is to justify the need for neurology critical care follow-up clinics to evaluate these children long term after the initial incident or trauma.

Cerebral oxygen saturation and cytochrome oxidase redox state in children with epilepsy: A pilot study – multi-channel NIRS for epilepsy seizure detection

• A study to learn if the use of a type of measurement called infrared spectroscopy (NIRS) can detect changes in the level of oxygen in the blood of children diagnosed with seizures.

A multi-centered examination of pediatric epilepsy surgery evaluation in the US ("PERC/Surgical/Cook Children's")

• A multi-department study to compare how different pediatric epilepsy centers across the country evaluate children as potential candidates for surgery to eliminate seizures. The goal is to collect data which may help future researchers compare centers and potentially standardize the surgery evaluation process.

General Neurology

A prospective study of acute flaccid myelitis (AFM) to define natural history, risk factors, and pathogenetic mechanisms. (DMID Protocol 19-0005)

• A study funded by the National Institute of Health (NIH) to collect data and blood, nose swab, mouth swab, and stool samples on children diagnosed with a condition called Acute Flaccid Myelitis (AFM).

A multi-site retrospective case series of electromyography (EMG) findings in acute flaccid myelitis (AFM)

• A study to build a dataset to allow doctors to better understand the EMG findings in children diagnosed with AFM and to compare those findings to the outcomes in children diagnosed with AFM.

A Randomized, Double-Blind Double-Dummy, Placebo-Controlled, 3-Arm, Parallel-Group Study in Pediatric Subjects Aged 10 Through 17 Years to Evaluate the Efficacy and Safety of BG00012 and BIIB017 for the Treatment of Relapsing-Remitting Multiple Sclerosis

• A clinical trial to evaluate the effectiveness of BG00012 (dimethyl fumarate; Tecfidera®) and BIIB017 (peginterferon beta-1a; Plegridy®) in children and teens with relapsing-remitting multiple sclerosis.

Photodynamic therapy for benign dermal neurofibromas phase II

• A study to learn more about the effects of a drug called Levulan Kerastick which is applied on the skin of patients diagnosed with a condition called Type 1 neurofibromas.

International Pediatric Stroke Study Protocol (IPSS): Towards the Establishment of Standards of Practice and the Initiation of Multi-Center, Multi-National Clinical Trials for Neonates and Children with Stroke

• A study to build a database and network of centers to with the goal of gathering standardized data of the diagnosis, risk factors, outcomes, and therapies of children with stroke.

Neuromuscular research

Neuromuscular and neuromuscular testing study data bank

• A local Children's Wisconsin-based data bank study where the goal is to gather data on children seen, tested, or examined by a neuromuscular provider or physician. The purpose is to collect data for use in future research, to allow for quality improvement, and to allow the study team to contact families if children potentially qualify for clinical trials.

Muscular Dystrophy Association's neuromuscular observational research (MOVR) data hub protocol (MDA, #MDACS1)

• A data bank study funded by the Muscular Dystrophy Association (MDA) to collect data on children diagnosed with different types of neuromuscular diseases to better understand health outcomes, provide data to help develop new therapies and drugs, and to better understand neuromuscular diseases.

POMGnT1 muscle-eye-brain databank and registry

• A Children's Wisconsin-based data registry to collect medical records and contact information for children who have been diagnosed with a rare disorder known as POMGnT1 muscle-eye-brain disease.

Retrospective review of inpatient pediatric rhabdomyolysis without renal failure

• A medical chart review study to review the system for evaluating and managing patients with a condition called rhabdomyolysis (breakdown of muscle tissue) without kidney issues. The goal is to examine the current method of evaluating these children and to propose a new method or system of evaluation that is as equally safe, effective, and costs equal or less than the current system.

Spinal Muscular Atrophy

Spinal Muscular Atrophy tissue bank

• A local Children's Wisconsin-based data bank study where the goal is to gather data on children diagnosed with Spinal Muscular Atrophy, including blood and spinal fluid samples (collected at the same time as when ordered for care and treatment purposes). The data and blood/spinal fluid samples are stored for use for future researchers and research studies.

A prospective, long-term registry of patients with a Diagnosis of Spinal Muscular Atrophy (AveXis, Inc., AVXS-101-RG-001)

• A data registry funded by the pharmaceutical company AveXis, Inc. to collect data on children diagnosed with Spinal Muscular Atrophy to better understand health outcomes in children treated with different drugs or therapies, especially as new drugs and therapies are approved and available.

Our neuropsychology department has ongoing multidisciplinary clinical research activities examining cognitive outcomes within the following patient populations:  

Comprehensive Epilepsy Program

We work collaboratively with Neurology, Neurosurgery and Radiology to examine the cognitive outcomes associated with various epilepsy disorders and surgical interventions. View the publications from the Comprehensive Epilepsy Program.

Multidisciplinary Brain Tumor Program

In collaboration with our Neuro-oncology and Neurosurgery partners, we have examined the cognitive, emotional and functional outcomes of children treated for various brain tumors. View the publications from the Multidisciplinary Brain Tumor Program.

Spina Bifida Program

Children with myelomeningocele often demonstrate a cognitive profile of strengths and weaknesses that may impact their academic functioning. Development of a standard of care for evaluation and measurement of cognitive functioning in this population may allow for more prompt intervention. The MCW/CW Neuropsychology Team is leading a multi-site collaborative group to evaluate care standards 

Additionally, within our own CW program, we are examining cognitive and functional outcomes of children with Spina Bifida across the age range, from those in early childhood to late teens beginning to transition into adulthood. We additionally are examining how issues of equity and access to care impact outcomes across this age span. View the publications from the Spina Bifida Program.

DREAM Clinic

In the DREAM (Developmentally Ready: Engagement and Achievement of Milestones) Clinic, we work with partners in Neurology, Neonatology and Physical Medicine & Rehabilitation to examine developmental outcomes in our youngest patients who have graduated from our Neonatal Intensive Care Unity. We are exploring issues related to health equity and access to care as well as a model of early follow-up and intervention to optimize developmental outcomes. View the publications from the DREAM Clinic.

Tracking Outcomes of Cardiac Neurodevelopment (TOuCaN) Program

Children born with congenital heart disease (CHD) have increased risk of neurodevelopmental problems. The Tracking Outcomes of Cardiac Neurodevelopment (TOuCaN) Program was established to evaluate and monitor the cognitive development of children with CHD from preschool-age through young adulthood. We follow recommended guidelines put forth by the American Heart Association and the American Academy of Pediatrics regarding routine neuropsychological evaluations.  Along with our partners in the Herma Heart Institute and HOPE (Healthy Outcomes Post-ICU Engagement) Clinic, we are a member of Cardiac Neurodevelopmental Outcome Collaborative, a multidisciplinary, multinational organization working together to describe outcomes and to establish and implement best clinical practice. Members our TOuCAN Program are researching neurodevelopment broadly including exploring attention regulation, social functioning, adaptive functioning and longitudinal cognitive outcomes.  

Pediatric and Infant Neuropsychological Testing (PINT) Clinic

Our neuropsychology team works with 200+ new preschool-aged patients with a variety of developmental and medical concerns on a yearly basis. We have the opportunity to follow the development of many of these children over the years into adolescence and even adulthood. We are examining the outcomes of these patients to identify the most important factors related to good outcomes in order to identify what interventions will be most helpful to our new preschool patients.

The effectiveness of advanced MR imaging techniques for grading pediatric brain tumors

• A retrospective study evaluating the effectiveness of advanced magnetic resonance imaging techniques for distinguishing low grade from high grade pediatric brain tumors. Our aim is to determine if this type of imaging can be used to distinguish high grade from low grade pediatric tumors. Knowing tumor grade is important to help us minimize surgical interventions, optimize surgical biopsy sites, provide a more accurate therapeutic plan, improve determination of prognosis, and may help to counsel parents more effectively.

Reducing radiation exposure of imaging tests for diagnosing children with cancer

• A project that is designed to reduce the radiation exposure of imaging tests for children with cancer.

Topical photodynamic therapy (PDT) with Levulan® Kerastick® for benign dermal neurofibromas phase II

• A project involving the photosensitization of cutaneous neurofibromas using the Levulan® Kerastick® Topical Solution (Dusa), plus illumination with red light. Ultrasound and pathological response to this treatment is being assessed in this trial.

NIRS visual motor and language mapping validation study

• A study where brain mapping is performed with a novel noninvasive technique called Near Infrared Spectroscopy (NIRS). The results obtained from this technique are then compared with the current standard of care for non-invasive brain mapping for motor and language function, such as a functional MRI (fMRI).

Incidence of neonatal encephalopathy in the NICU

• A project examining whether the use of an EMR smartphrase that outlines guidelines for therapeutic hyptothermia (TH) use in patients with hypoxic-ischemic encephalopathy (HIE) during admission documentation changes the frequency of TH use in cases that don't meet criteria for it and whether this impacts long term neurodevelopment outcomes.

Determining variation in treatment approach and neurologic outcomes for infants with post-hemorrhagic hydrocephalus

• A retrospective cohort study of premature infants with post-hemorrhagic hydrocephalus at Children's. The aim of this project is to identify factors that impact the short term and long term outcomes of these infants. This data will inform local practice guidelines for the care of premature infants with PHH.

Congenital

Chiari malformation retrospective review

• A retrospective review examining presentation and outcomes of pediatric patients undergoing surgery for Chiari I malformation. In particular, this study evaluates whether various MRI measurements are predictive of surgical outcome.

Transfusion in craniosynostosis surgery

• A retrospective review examining the use of blood transfusions and outcomes in pediatric patients undergoing surgery for craniosynostosis.

Epilepsy

Predicting response to vagal nerve stimulation (VNS)

• A multi-institutional collaboration with University of Toronto/SickKids that seeks to better identify patients with epilepsy who are more likely to respond to vagal nerve stimulation (VNS) therapy

Laser ablation surgery for treatment of epilepsy and brain tumors

• A multi-institutional collaboration examining the use of MRI-guided stereotactic laser ablation for the treatment of refractory epilepsy and brain tumors.

Stereotactic electroencephalography series (SEEG series)

• A multi-institutional collaboration with University of Alabama at Birmingham/Children's of Alabama examining the use of stereo-encephalography (sEEG) depth electrode placement to better localize seizure onset in pediatric patients with refractory epilepsy.

Awake craniotomy in the pediatric population

• A review of the experience at Children's Wisconsin with brain surgery for tumors or epilepsy in children who are awake during their surgery. Awake brain surgery allows for real-time assessment of brain function during the procedure to minimize risk of functional loss.

Multiple hippocampal transections in pediatric epilepsy

• A review of our experience with an innovative memory-sparing surgical technique for treating hippocampal epilepsy in children.

Surgical results in the treatment of electrical status epilepticus in sleep

• An examination of our experience managing this relatively rare form of epilepsy with attention paid to factors associated with success/failure.

Outcomes in pediatric epilepsy surgery and intracranial electrocorticography

• This study evaluates the likelihood of successful capture of electroclinical seizures during long term intracranial electroencephalography.

Epilepsy surgery outcomes data bank

• An ongoing database comprised of patients undergoing epilepsy surgery at Children's Wisconsin which aims to help assess the outcomes of pediatric epilepsy surgery patients. 

Head & spine trauma

Neck pain in pediatric patients with mild traumatic brain injury

• A retrospective review to determine the incidence of neck pain and impact on duration of symptoms in pediatric patients with mild traumatic brain injury (mTBI).

Vacuum-assisted minimally-invasive elevation of depressed skull fractures in infants

• An examination of our experience with a minimally invasive non-surgical technique of elevating depressed skull fractures in babies.

Pediatric spine registry

• A multi-institutional collaborative registry with the Pediatric Spine Foundation that collects patient data on children with spine and chest wall disorders.

Hydrocephalus

HCRNq

• Participation in the quality improvement projects of the Hydrocephalus Clinical Research Network (HCRN) to improve diagnosis, treatment, and outcomes of hydrocephalus patients

COVID-19 impact on families and children with hydrocephalus

• A multi-institutional collaboration with University of Alabama at Birmingham/Children's of Alabama examining the impact of COVID-19 on rates of operations for hydrocephalus and factors contributing to rate variability.

Spina Bifida

Spinal cord untethering in spina bifida patients with scoliosis

• A retrospective study evaluating the role of spinal cord untethering prior to thoracolumbar fusion in children with spina bifida and scoliosis.