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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 : Completed
First Posted : January 7, 2015
Results First Posted : March 2, 2022
Last Update Posted : March 2, 2022
Sponsor:
Collaborators:
Jazz Pharmaceuticals
Faneca 66 Foundation
Information provided by (Responsible Party):
Anne Comi, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

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.

Condition or disease Intervention/treatment Phase
Sturge-Weber Syndrome Drug: Cannabidiol Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
Study Start Date : December 2014
Actual Primary Completion Date : April 2019
Actual Study Completion Date : April 2021


Arm Intervention/treatment
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.
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




Primary Outcome Measures :
  1. Number of Seizures Per Month [ Time Frame: Measured within 56 days before baseline and 56 days before week 14 ]
    A baseline seizure frequency was recorded for each subject in a diary for eight weeks prior to investigational drug initiation and parents/caregivers documented seizures on a daily basis throughout the trial using a seizure log. For assessing the efficacy of CBD, the investigator counted the change in frequency of seizures per month. The number of seizures within 56 days of the baseline and the number of seizures within 56 days of week 14 were calculated. Higher seizure frequency indicates worse outcome. This outcome is measured as the change in number of seizures per month between the baseline and week 14 time points.


Secondary Outcome Measures :
  1. Percentage Change in Seizure Frequency at Most Recent Visit on CBD Compared With Baseline [ Time Frame: Measured at Baseline and most recent visit within 1 year ]
    The percentage change, between the seizure frequency per month reported at baseline compared to seizure frequency per month at the subject's most recent visit, on CBD was calculated. Higher positive percentage change in seizure frequency per month would indicate better outcome. Positive values indicate a decrease in seizure frequency.



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month to 45 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02332655


Locations
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United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Sponsors and Collaborators
Anne Comi, MD
Jazz Pharmaceuticals
Faneca 66 Foundation
Investigators
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Principal Investigator: Anne M Comi, MD Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
  Study Documents (Full-Text)

Documents provided by Anne Comi, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:
Additional Information:
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Responsible Party: Anne Comi, MD, Principal Investigator, Director Sturge-Weber Center, Kennedy Krieger Institute, Professor Johns Hopkins University School of Medicine, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT02332655    
Other Study ID Numbers: IRB00029264
First Posted: January 7, 2015    Key Record Dates
Results First Posted: March 2, 2022
Last Update Posted: March 2, 2022
Last Verified: February 2022
Keywords provided by Anne Comi, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:
Sturge-Weber syndrome
SWS
Drug resistant epilepsy
Additional relevant MeSH terms:
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Sturge-Weber Syndrome
Brain Stem Infarctions
Klippel-Trenaunay-Weber Syndrome
Syndrome
Disease
Pathologic Processes
Brain Infarction
Brain Ischemia
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Stroke
Vascular Diseases
Cardiovascular Diseases
Infarction
Ischemia
Necrosis
Angiomatosis
Hemangioma
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Neoplasms
Neurocutaneous Syndromes
Cannabidiol
Anticonvulsants