NCT02332655

Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Anne Comi, MD · Academic / Other
• Sex: All
• Age: 1 Month – 45 Years
• Healthy volunteers: Not accepted

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 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

• Sturge-Weber Syndrome

Interventions

Type	Name	Description
DRUG	Cannabidiol	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.

Timeline

• Start date: 2014-12-01
• Primary completion: 2019-04-01
• Completion: 2021-04-01
• First posted: 2015-01-07
• Last updated: 2022-03-02
• Results posted: 2022-03-02

Locations

1 site across 1 country: United States