Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
This study is ongoing, but not recruiting participants.
GW Pharmaceuticals Ltd.
Faneca 66 Foundation
Information provided by (Responsible Party):
Anne Comi, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
First received: January 5, 2015
Last updated: January 23, 2017
Last verified: January 2017
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.
||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
||Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
Primary Outcome Measures:
- Change in seizure frequency [ Time Frame: 1 year ]
Secondary Outcome Measures:
- 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 ]
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||December 2017 (Final data collection date for primary outcome measure)
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.
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.
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.
|Ages Eligible for Study:
||1 Month to 45 Years (Child, Adult)
|Sexes Eligible for Study:
|Accepts Healthy Volunteers:
Inclusion Criteria: Participants with Sturge-Weber syndrome brain involvement as defined on neuroimaging (n=10 subjects, male and female, ages 1 month to 45 years of age) and the following:
- Documentation of a diagnosis of drug resistant epilepsy as evidenced by failure to control seizures despite appropriate trial of two or more AEDs at therapeutic doses. Drug resistant epilepsy for this study is defined as: At least 1 reported quantifiable (no cluster or innumerable) defined seizure with motor signs per month for at least 3 months prior to initial visit and during the period between Visit 1 (Screening Visit) and Visit 2 (Baseline Visit), as per data captured in daily seizure diaries. These can be focal seizures, focal seizures with impaired consciousness, myoclonic seizures, generalized, and secondarily generalized seizures.
- Between 1-5 baseline anti-epileptic drugs at stable doses for a minimum of 4 weeks prior to enrollment. Vagus nerve stimulator (VNS), ketogenic diet and modified Atkins diet do not count toward this limit.
- VNS must be on stable settings for a minimum of 3 months prior to enrollment.
- If on ketogenic or Atkins diet, must be on stable ratio for a minimum of 3 months prior to enrollment.
- Previous subjects who failed at any point to meet continuation criteria and withdrew early may be considered for re-enrollment under a new subject ID as long as the above inclusion criteria are met. The determination of whether to re-enroll will be made by the PI and sponsor on a case-by-case basis. Re-enrollment can occur no earlier than 4 weeks after the final, post-weaning follow-up visit under the old subject ID.
Written informed consent obtained from the patient or the patient's legal representative must be obtained prior to beginning treatment.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02332655
|Kennedy Krieger Institute
|Baltimore, Maryland, United States, 21205 |
Anne Comi, MD
GW Pharmaceuticals Ltd.
Faneca 66 Foundation
||Anne M Comi, MD
||Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
||Anne Comi, MD, Principal Investigator, Director Sturge-Weber Center, Kennedy Krieger Institute,Associate Professor Johns Hopkins University School of Medicine, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
History of Changes
|Other Study ID Numbers:
|Study First Received:
||January 5, 2015
||January 23, 2017
Keywords provided by Anne Comi, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:
Drug resistant epilepsy
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on August 18, 2017
Brain Stem Infarctions
Central Nervous System Diseases
Nervous System Diseases
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type