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

A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children with Duchenne Muscular Dystrophy including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B

Primary Investigator: Marks MD, Warren

IRB ID: 2023-003 (OPEN)

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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 (BEYOND)

Primary Investigator: Marks MD, Warren

IRB ID: Sarepta_SRP-9001-401_Marks (OPEN)

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A Multimodal Biomarker-based Predictive Model for Infantile Spasm

Primary Investigator: Keator MD, Cynthia

IRB ID: 2022-006 (OPEN)

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A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-921352 as Adjunctive Therapy in Subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

Primary Investigator: Perry MD, M. Scott

IRB ID: 2020-071 (OPEN)

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A Prospective, Long Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy

Primary Investigator: Marks MD, Warren

IRB ID: 2019-052 (OPEN)

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A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

Primary Investigator: Perry MD, M. Scott

IRB ID: 2022-021 (OPEN)

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A Randomized, Double-Blind, Placebo- Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

Primary Investigator: Acord, Stephanie

IRB ID: 2022-074 (OPEN)

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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)

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Activa® Dystonia Therapy

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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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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Electrophysiological Biomarkers of GABA Metabolism in Children with Genetic Epilepsy Syndromes

Primary Investigator: Papadelis, PhD, Christos

IRB ID: 2022-055 (OPEN)

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Electrophysiological Biomarkers of GABA metabolism in children with SCN1A+ Dravet Syndrome

Primary Investigator: Papadelis, PhD, Christos

IRB ID: 2022-051 (OPEN)

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Epilepsy Genetics Database

Primary Investigator: Keator MD, Cynthia

IRB ID: 2023-074 (OPEN)

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Evaluation of the outcome of surgical intervention on the upper extremity in young patients with hemiplegia

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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Long-term Antipsychotic Pediatric Safety Trial (LAPS)

Primary Investigator: Bailey PhD, Laurie

IRB ID: 2019-078 (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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Neuromuscular Observational Research (MOVR) Data Hub Protocol

Primary Investigator: Marks MD, Warren

IRB ID: 2019-055 (OPEN)

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Pediatric International DBS (PEDiDBS) Registry

Primary Investigator: Marks MD, Warren

IRB ID: 2016-013 (OPEN)

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Phase 3 trial for patients with Dyskinetic Cerebral Palsy

Primary Investigator: Marks MD, Warren

IRB ID: 2022-027 (OPEN)

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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

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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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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Sensory and Affective Processing in Children with Autism Spectrum Disorder

Primary Investigator: Papadelis, PhD, Christos

IRB ID: 2024-047 (OPEN)

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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 682-303-2650.