Neuroscience Clinical Trials
When most people think of medical research they assume that the most advanced research comes from academic affiliations. But here at Cook Children's, we challenge that assumption. Our medical team is deeply involved in research of all kinds. We look for better ways to diagnose, treat and prevent neurological conditions ... and we look for ways to cure them.
Cook Children's is at the forefront of leading-edge clinical research and our specialists are actively participating in studies investigating a variety of conditions including epilepsy, stroke and neuromuscular disorders.
Active trials
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A Multimodal Biomarker-based Predictive Model for Infantile Spasms
Primary Investigator: Keator MD, Cynthia
IRB ID: 2022-006 (OPEN)
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A PHASE 2, TWO-STAGE, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY EFFICACY OF RLS-0071 IN NEWBORNS WITH MODERATE OR SEVERE HYPOXIC-ISCHEMIC ENCEPHALOPATHY UNDERGOING THERAPEUTIC HYPOTHERMIA WITH LONG-TERM FOLLOW-UP (STAR)
Primary Investigator: Riley MD, David
IRB ID: Premier_RLS-0071-202 (ReAlta)_Riley (OPEN)
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A Phase 3, Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
Primary Investigator: Marks MD, Warren
IRB ID: 2022-027 (OPEN)
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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults with Developmental and Epileptic Encephalopathies
Primary Investigator: Keator MD, Cynthia
IRB ID: 2025-027 (OPEN)
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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults with Dravet Syndrome
Primary Investigator: Perry MD, M. Scott
IRB ID: 2025-024 (OPEN)
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A PROSPECTIVE, LONG-TERM REGISTRY OF PATIENTS WITH A DIAGNOSIS OF SPINAL MUSCULAR ATROPHY (SMA) - (RESTORE)
Primary Investigator: Acord, Stephanie
IRB ID: 2019-052 (OPEN)
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Activa Dystonia Therapy HDE
Primary Investigator: Marks MD, Warren
IRB ID: 593 (OPEN)
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BN44620: A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED AS AN EARLY INTERVENTION IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY AFTER GENE THERAPY
Primary Investigator: Acord, Stephanie
IRB ID: 2023-078 (OPEN)
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Brain functional reorganization as a result of deep-brain stimulation therapy in children with dystonia
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2021-009 (OPEN)
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Cognitive and Neuroimaging Biosignatures of Well-being in Youth with and without Seizures
Primary Investigator: Cooper, Crystal
IRB ID: 2020-104 (OPEN)
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Combined Abnormal General Movement Assessment and High-Resolution Brain Magnetic Resonance Imaging as a Predictor of a Diagnosis of Cerebral Palsy among Patients withHypoxic-Ischemic Encephalopathy
Primary Investigator: Johnson MD, Yvette
IRB ID: CCMC_MRI and GMA_Johnson (OPEN)
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Electrophysiological Biomarkers of GABA Metabolism in Children with Genetic Epilepsy Syndromes
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-051 (OPEN)
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Electrophysiological Biomarkers of GABA metabolism in children with SCN1A+ Dravet Syndrome
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-055 (OPEN)
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EMAtS registry
Primary Investigator: Tran, Lihn
IRB ID: 2024-111 (OPEN)
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ENDEAVOR: A CLINICAL STUDY TO EVALUATE THE SAFETY AND EFFICACY OF ETX101, AN AAV9 DELIVERED GENE THERAPY IN INFANTS AND CHILDREN WITH SCN1A POSITIVE DRAVET SYNDROME
Primary Investigator: Perry MD, M. Scott
IRB ID: 2022-076 (OPEN)
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Evaluation of the outcome of surgical intervention on the upper extremity in young patients with hemipelgia
Primary Investigator: Marshall MD, James
IRB ID: 2020-098 (OPEN)
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Ictal and Interictal High-Frequency Electrophysiological Activity for the Localization of the Epileptogenic Zone in Children with Refractory Epilepsy
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2019-070 (OPEN)
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Multi-modal neuroimaging in children with cerebral palsy to assess functional and anatomical reorganization in relation to sensory and motor functions
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2019-068 (OPEN)
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Nationwide_Epilepsy Multi-Site: Comprehensive Profiling of Neurological Disorders of Somatic Etiology Accelerator Protocol
Primary Investigator: Perry MD, M. Scott
IRB ID: 2024-046 (OPEN)
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Pediatric International DBS Registry (PEDiDBS)
Primary Investigator: Marks MD, Warren
IRB ID: 2016-013 (OPEN)
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PERC_Epilepsy Genetics Database
Primary Investigator: Keator MD, Cynthia
IRB ID: 2023-074 (TEMPORARILY CLOSED TO ACCRUAL)
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Presurgical Mapping of the Eloquent Cortex in Children with Drug Resistant Epilepsy Using Videogames
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2024-028 (OPEN)
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Quantitative Assessment of Sucking for Early Diagnosis of Brain Injury in Infants at High Risk
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2019-085 (OPEN)
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Retrospective data analysis of neurophysiological data for intraoperative or epilepsy monitoring. An international, retrospective, multi-site study.
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-059 (OPEN)
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Robot-assisted hand training to induce manual functional change and cerebral neural plasticity in children with cerebral palsy
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2021-012 (OPEN)
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Sensory and Affective Processing in Children with Autism Spectrum Disorder
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2024-047 (OPEN)
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SRP-9001-401: A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice
Primary Investigator: Marks MD, Warren
IRB ID: 2023-084 (OPEN)
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STK-001-DS-301_Stoke_Dravet Syndrome
Primary Investigator: Perry MD, M. Scott
IRB ID: Stoke_STK-001-DS-301_Perry (OPEN)
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VOG, A Retrospective Evaluation of High-Flow Intracranial Arteriovenous Malformations in Neonates: Clinical Management and Outcomes in North America
Primary Investigator: Riley MD, David
IRB ID: 2024-052 (OPEN)
We're here to help.
If your child has been diagnosed, you probably have lots of questions. We can help. If you would like to schedule an appointment, refer a patient or speak to our staff, please call our offices at 682-885-2500.