Clinical Trial: Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome

Brief Summary: The purpose of this study is to determine the tolerability and optimal dose of cannabidiol (CBD) as an simultaneous treatment in children and young adults with Sturge-Weber syndrome (SWS) and drug resistant epilepsy.

Detailed Summary: We hope to gain an understanding of the utility of pure CBD used for the treatment of medically refractory epilepsy in SWS in this open-label, safety dose-finding, study. Recent evidence suggests that CBD has multiple, beneficial, effects in patients (such as those with SWS that undergo neurological deterioration) suffering from medically refractory seizures. We hypothesize that CBD will reduce seizure frequency in children and young adults with SWS and will therefore help stabilize and improve their neurologic status.This trial is part of an expanded access program, available through a partnership with GW Pharmaceutical, which has been sanctioned by the FDA to study the safety and efficacy of Epidiolex (cannabidiol/CBD) in participants with SWS and medically refractory seizures.
Sponsor: Anne Comi, MD

Current Primary Outcome: Change in seizure frequency [ Time Frame: 1 year ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in average seizure duration by seizure type [ Time Frame: 1 year ]
  • Change in the number of episodes of status epilepticus, defined as convulsive seizure lasting longer than 10 minutes [ Time Frame: 1 year ]
  • Change in the number of uses of rescue medication [ Time Frame: 1 year ]
  • Change in the number of ER visits/ hospitalizations [ Time Frame: 1 year ]


Original Secondary Outcome: Same as current

Information By: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Dates:
Date Received: January 5, 2015
Date Started: December 2014
Date Completion: January 2018
Last Updated: January 23, 2017
Last Verified: January 2017