| Eligibility |
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.
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