Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome

Active, not recruiting

Phase 1/2 Results N/A

Trial Description

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 Description

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.

Conditions

Interventions

  • Cannabidiol Drug
    Other Names: Epidiolex; CBD
    Intervention Desc: Initiation of treatment will begin with 2mg/kg/day. The dose will be increased by 3 mg/kg/day after seven days and then by 5 mg/kg/day every seven days up to a maximum dose of 25 mg/kg/day given. The dose of concomitant antiepileptic drugs will remain unchanged during the first 12 weeks of CBD treatment (or until 8 weeks after steady state at final dose), unless symptoms of toxicity and/or significant changes in blood levels are observed.
    ARM 1: Kind: Experimental
    Label: Cannabidiol
    Description: All subjects will receive the experimental Epidiolex (cannabidiol) oral solution to be taken at home twice a day, and will be treated on an outpatient basis. The drug will be taken for 48 weeks unless the subject chooses to participate in the extension phase of the study, in which case the subject will continue to receive the drug for one additional year or until the drug is approved for clinical use for the treatment of epilepsy in patients with Sturge-Weber syndrome.

Trial Design

  • Masking: Open Label
  • Purpose: Treatment
  • Endpoint: Safety/Efficacy Study
  • Intervention: Single Group Assignment

Outcomes

Type Measure Time Frame Safety Issue
Primary Change in seizure frequency 1 year No
Secondary Change in average seizure duration by seizure type 1 year No
Secondary Change in the number of episodes of status epilepticus, defined as convulsive seizure lasting longer than 10 minutes 1 year No
Secondary Change in the number of uses of rescue medication 1 year Yes
Secondary Change in the number of ER visits/ hospitalizations 1 year Yes
Secondary Change in the number of episodes of status epilepticus, defined as convulsive seizure lasting longer than 10 minutes 1 year No

Sponsors