Current Clinical Trials

Le Bonheur’s Neuroscience Institute is committed to discovering the best ways to care for kids with neurologic conditions. Our goal in pediatric neurological research is to find the best methods for treating disorders of the brain and nervous system in children. Below are our current clinical trials. If you have a patient that might benefit from these novel interventions, please contact us at neuroscience@lebonheur.org or Le Bonheur Connect at 901-287-PEDS or 1-866-870-5570.

Ganaxolone

• Diagnosis: status epilepticus (SE)
• 12 years and older
• Evaluate the safety and efficacy of intravenous ganaxolone to decrease or cease seizure activity in patients with SE
• Raise/Marinus Pharmaceuticals

EpiWatch

• Diagnosis: epilepsy
• 5 years and older
• Evaluate the efficacy of seizure detection using a mobile application, EpiWatch, on the Apple Watch for patients admitted to the epilepsy monitoring unit (EMU)
• EpiWatch/Johns Hopkins University

Risk factor study

• Diagnosis: stroke
• 28 days to 18 years
• Study the relationship between infection and increased risk factors for children experiencing acute ischemic stroke
• The Vascular Effects of Infection in Pediatric Stroke (VIPS II)/NIH: National Institute of Neurological Disorders and Stroke

Antisense oligonucleotide (STK-001)

• Diagnosis: Dravet syndrome
• 2 to 18 years
• Investigate the safety of single dose antisense oligonucleotide (STK-001) in children and adolescents with Dravet syndrome
• Monarch/Stroke Therapeutics

Human tissue study

• Diagnosis: epilepsy surgery and brain tumor surgery
• 7 to 11 years
• Utilize extra tissue samples obtained during brain surgery to examine if human brain specimens have neuropathology that correlates with related cell culture and animal models of human disease
• Investigator-initiated

Natural history study

• Diagnosis: Dravet syndrome
• 6 to 60 months
• Natural history study designed to describe the seizure, neurodevelopmental and behavioral characteristics of Dravet syndrome. Data collected from this study will serve as an external control to a clinical trial examining a gene therapy investigational drug that aims to improve the seizure burden and neurodevelopmental outcomes in patients with SCN1A-positive Dravet syndrome.
• Envision/Encoded Therapeutics

Privigen

• Diagnosis: pediatric chronic inflammatory demyelinating polyneuropathy (CIDP)
• 2 to 16 years
• Investigate the safety and efficacy of IV privigen, immune globulin in the treatment of pediatric CIDP
• IgPro10_4001/CSL Behring

EPX-100

• Diagnosis: Dravet syndrome
• 2 to 17 years
• Evluate the efficacy of EPX-100 (clemizole hydrochloride) and change in seizure frequency in patients with Dravet syndrome experiencing uncontrolled seizures
• EPX-100/Epygenic Therapeutics