Clinical Trial: Verapamil as Therapy for Children and Young Adults With Dravet Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Verapamil as Adjunctive Seizure Therapy for Children and Young Adults With Dravet Syndrome

Brief Summary: This study will assess how well the drug verapamil can improve control of seizures and dysautonomia symptoms in children and young adults diagnosed with Dravet syndrome. The safety of verapamil when given with all concomitant medications will also be assessed.

Detailed Summary:

Dravet syndrome (DS) is a devastating form of pediatric seizure disorder (epilepsy), often related to abnormalities of one of the genes that controls sodium channel function in the brain (SCN1A). Most children with DS experience continued seizures even with optimal treatment of currently available anti-seizure therapies [1]. Many of these seizures are prolonged, and can be life threatening.

This pilot study will assess the efficacy of verapamil in improving control of seizures in children and young adults DS. This will be done by adding verapamil as open label adjunctive therapy to medications already being given. Investigators will assess the effect of verapamil therapy on seizure control and on signs of autonomic dysfunction observable to the parents/guardians. Signs of autonomic function include body temperature regulation, sweating, heart rate, pupil size, and flushing of the skin. Iannetti, et al reported treating 2 children with clinical DS (one with an SCN1A mutation) with verapamil as adjunctive therapy [2]. Both children had a positive clinical response persisting for a number of months. No adverse effects were noted. We have treated an additional 4 children with DS with verapamil. There have been no significant adverse effects; 3 of 4 have experienced improved seizure control for months also.

Verapamil has been shown to affect autonomic tone in patients with cardiac disorders (eg. high blood pressure, heart attack). It alters the balance between parts of the autonomic nervous system's function (called sympathetic and parasympathetic function) with a shift toward decreased sympathetic tone and increased parasympathetic (vagus nerve) tone [8, 9, 10]. Verapamil is used as an effective agent to treat certain types of autonomic headaches in both adults and children. In cluster headaches, autonomic symptoms (tearing, n
Sponsor: Beverly S. Wical, M.D.

Current Primary Outcome: Seizure Frequency [ Time Frame: Baseline, 8 weeks, 12 weeks, 16 weeks, 24 weeks, and 35 weeks ]

The primary study endpoint is GTCS seizure frequency. A reduction in the number and severity of seizures is anticipated.


Original Primary Outcome: Same as current

Current Secondary Outcome: Frequency of myoclonic/absence/atypical absence seizures [ Time Frame: Baseline, 8 weeks, 12 weeks, 16 weeks, 24 weeks, and 35 weeks ]

We anticipate a reduction in myoclonic, absence, and atypical absence seizures as well.


Original Secondary Outcome: Same as current

Information By: Gillette Children's Specialty Healthcare

Dates:
Date Received: May 24, 2012
Date Started: April 2012
Date Completion:
Last Updated: March 24, 2015
Last Verified: March 2015