Clinical trial NCT02332655: Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
Official title: Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Sturge-Weber Syndrome
Keywords: Sturge-Weber syndrome
SWS
Drug resistant epilepsy
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Sponsor class: OTHER
Collaborators: name: Jazz Pharmaceuticals
name: Faneca 66 Foundation
First posted: 2015-01-07
Enrollment: 5
Sex: ALL
Minimum age (years): 1
Maximum age (years): 45
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Number of Seizures Per Month
timeFrame: Measured within 56 days before baseline and 56 days before week 14
Secondary outcomes:
measure: Percentage Change in Seizure Frequency at Most Recent Visit on CBD Compared With Baseline
timeFrame: Measured at Baseline and most recent visit within 1 year
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
Study officials:
affiliation: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
name: Anne M Comi, MD
role: PRINCIPAL_INVESTIGATOR
References: