NCT03894826

Voronistat in Pediatric Patients With Drug Resistant Epilepsy

A Phase 2 Clinical Trial Testing the Safety and Efficacy of Voronistat in Pediatric Patients With Drug Resistant Epilepsy

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: University of Calgary · Academic / Other
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The study evaluates the safety, tolerability, and efficacy of Vorinostat in addition to standard of care anti-epileptic drugs in pediatric patients with medically refractory epilepsy. All participants entering the treatment phase will receive Vorinostat.

Detailed description

Vorinostat is a potent inhibitor of histone deacetylases (HDAC). HDACs catalyze the removal of acetyl groups from the lysine residues of proteins, including histones and transcription factors. Valproic acid, a broad anti-epileptic drug, commonly used as first line treatment in epilepsy, has also been shown to inhibit HDAC activity . It is it is anticipated that Vorinostat in a population of pediatric patients with epilepsy will be well-tolerated, similar to that seen with valproic acid and other normally prescribed anti-epileptic drugs. Participants consenting to participate will enter a 4-week screening period to ensure eligibility. Eligible participants will then enter the 6-week treatment phase. Participants will be seen for a final Safety Follow-up visit 6 weeks after end of treatment. Participants who are enrolled and complete all study procedures will be in the study for a total of 16 weeks.

Conditions

• Refractory Epilepsy

Interventions

Type	Name	Description
DRUG	Vorinostat 100 MG	Vorinostat administered by mouth, once daily at a dose of 230 mg/m2/day for a total of 6 weeks

Timeline

• Start date: 2018-12-10
• Primary completion: 2020-04-01
• Completion: 2020-10-01
• First posted: 2019-03-29
• Last updated: 2019-03-29

Locations

1 site across 1 country: Canada