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Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02332655
Recruitment Status : Active, not recruiting
First Posted : January 7, 2015
Last Update Posted : January 8, 2019
Sponsor:
Collaborators:
GW Pharmaceuticals Ltd.
Faneca 66 Foundation
Information provided by (Responsible Party):
Anne Comi, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Tracking Information
First Submitted Date  ICMJE January 5, 2015
First Posted Date  ICMJE January 7, 2015
Last Update Posted Date January 8, 2019
Study Start Date  ICMJE December 2014
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 11, 2017)
Change in seizure frequency [ Time Frame: 1 year ]
Seizure Frequency between visits and per week will be calculated
Original Primary Outcome Measures  ICMJE
 (submitted: January 5, 2015)
Change in seizure frequency [ Time Frame: 1 year ]
Change History Complete list of historical versions of study NCT02332655 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 11, 2017)
  • Change in average seizure duration by seizure type [ Time Frame: 1 year ]
    Average seizure duration in seconds and minutes for each seizure type experienced by subject.
  • Change in the number of episodes of status epilepticus, defined as convulsive seizure lasting longer than 10 minutes [ Time Frame: 1 year ]
    Any episodes of status epilepticus are noted and changes in the number experienced by subject over the 1 year time frame are calculated.
  • Change in the number of uses of rescue medication [ Time Frame: 1 year ]
    Each time a rescue medication is used for a seizure between visits the data is noted and changes in frequency used over the 1 year time period will be calculated.
  • Change in the number of ER visits/ hospitalizations [ Time Frame: 1 year ]
    Number of ER visits or hospitalizations will be recorded and changes in frequency will be calculated.
Original Secondary Outcome Measures  ICMJE
 (submitted: January 5, 2015)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
Official Title  ICMJE 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 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sturge-Weber Syndrome
Intervention  ICMJE 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.

Other Names:
  • Epidiolex
  • CBD
Study Arms  ICMJE Experimental: Cannabidiol
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.
Intervention: Drug: Cannabidiol
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: January 5, 2015)
10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2019
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Exclusion Criteria:

  • Patients with seizures secondary to metabolic, toxic, infectious or psychogenic disorder or drug abuse or current seizures related to an acute medical illness.
  • Presence of only non-motor partial seizures (without limb or facial movements, eye deviation or head turning)
  • Patients who require rescue medication during the Baseline phase for more than 6 days.
  • Patients with any severe and/or uncontrolled medical conditions at randomization such as:

    1. liver disease such as cirrhosis, decompensated liver disease, and chronic hepatitis [i.e. quantifiable hepatitis B virus (HBV)-DNA and/or positive HbsAg, quantifiable hepatitis C virus (HCV)-RNA]
    2. Uncontrolled diabetes as defined by fasting serum glucose > 1.5
    3. Active (acute or chronic) or uncontrolled severe infections.
    4. Patients with an active, bleeding diathesis.
  • Patients who have had a major surgery or significant traumatic injury within 4 weeks of study entry. Patients who have not recovered from the side effects of any major surgery (defined as requiring general anesthesia), or patients that may require major surgery during the course of the study.
  • Patients who change the dose of the AEDs during 4 weeks before screening or during the baseline period.
  • Prior treatment with any investigational drug within the preceding 4 weeks prior to study entry.
  • Patients with a history of non-compliance to medical regimens or who are considered potentially unreliable or will not be able to complete the entire study. Those in foster care, unable to keep follow-up appointments, maintain close contact with Principal Investigator, or complete all necessary studies to maintain safety.
  • Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotropin (hCG) laboratory test.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month to 45 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02332655
Other Study ID Numbers  ICMJE IRB00029264
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Anne Comi, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Study Sponsor  ICMJE Anne Comi, MD
Collaborators  ICMJE
  • GW Pharmaceuticals Ltd.
  • Faneca 66 Foundation
Investigators  ICMJE
Principal Investigator: Anne M Comi, MD Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
PRS Account Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP