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 double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in children and young adults with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) followed by long-term open-label treatment.

Primary Investigator: M. Scott Perry

IRB ID: 2018-090 (OPEN)

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A Learning Healthcare System for Pediatric Epilepsy

Primary Investigator: M. Scott Perry

IRB ID: 2019-064 (OPEN)

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A Long-term Observational Study Evaluating Eteplirsen in Patients with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice

Primary Investigator: Warren A. Marks

IRB ID: 2019-007 (OPEN)

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A Phase 2, Prospective, Interventional, Open-Label, Multi-Site, Extension Study to Assess the Long-Term Safety and Tolerability of TAK-935 as Adjunctive Therapy in Patients with RareEpilepsy

Primary Investigator: M. Scott Perry

IRB ID: 2019-029 (OPEN)

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

Primary Investigator: Warren A. Marks

IRB ID: 2019-052 (OPEN)

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Activa� Dystonia Therapy

Primary Investigator: Warren A. Marks

IRB ID: 593 (OPEN)

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An Expanded Access Protocol for Use of Triheptanoin in Patients with Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

Primary Investigator: Adrian Lacy

IRB ID: 2019-027 (OPEN)

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An Observational Study to Investigate Cognition and Quality of Life in Children and Adolescents with Dravet Syndrome

Primary Investigator: M. Scott Perry

IRB ID: 2019-032 (OPEN)

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An Open-Label Extension Study Of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy

Primary Investigator: Warren A. Marks

IRB ID: 2019-084 (OPEN)

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Cognitive and Neuroimaging Biosignatures of Well-being in Youth with and without Seizures

Primary Investigator: Crystal Cooper

IRB ID: 2020-104 (OPEN)

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Comprehensive Epilepsy Center Surgical Database Project

Primary Investigator: M. Scott Perry

IRB ID: 2010-068 (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: Christos Papadelis PhD

IRB ID: 2019-070 (OPEN)

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MOVR Data Hub

Primary Investigator: Warren A. Marks

IRB ID: 2019-055 (OPEN)

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Multi Center iMRI Neurosurgery Database

Primary Investigator: John Honeycutt

IRB ID: 2013-025 (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: Christos Papadelis PhD

IRB ID: 2019-068 (OPEN)

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Observational study in infants and children with SCN1A+ Dravet Syndrome

Primary Investigator: M. Scott Perry

IRB ID: 2020-056 (OPEN)

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Parental Attitudes in Neonatal Clinical Trial Enrollment:Decision-Making Preferences and Reasoning among Participants and Non-Participants

Primary Investigator: David Riley

IRB ID: 2017-016 (OPEN)

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Pediatric International DBS (PEDiDBS) Registry

Primary Investigator: Warren A. Marks

IRB ID: 2016-013 (OPEN)

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Phase I Dravet Syndrome study in Children and Adolescents

Primary Investigator: M. Scott Perry

IRB ID: 2020-012 (OPEN)

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Quantitative Assessment of Sucking for Early Diagnosis of Brain Injury in Infants at High Risk

Primary Investigator: Christos Papadelis PhD

IRB ID: 2019-085 (OPEN)

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The �HEAL� Study, High-Dose Erythropoietin for Asphyxia and Encephalopathy (A randomized, placebo-controlled, double-masked 500-subject clinical trial of erythropoietin for the treatment of neonatal hypoxic-ischemic encephalopathy)

Primary Investigator: David Riley

IRB ID: 2017-010 (OPEN)

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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.