Status:
COMPLETED
Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome
Lead Sponsor:
Anne Comi, MD
Collaborating Sponsors:
Jazz Pharmaceuticals
Faneca 66 Foundation
Conditions:
Sturge-Weber Syndrome
Eligibility:
All Genders
3-50 years
Phase:
PHASE2
Brief Summary
The purpose of this study is to better understand the utility of cannabidiol (CBD/ Epidiolex) for improving the treatment of cognitive impairments in Sturge-Weber syndrome (SWS).
Detailed Description
The investigators hope to gain an understanding of the utility of pharmaceutical grade CBD used for the treatment of cognitive impairments in SWS in this open-label study. Anecdotal evidence from a ph...
Eligibility Criteria
Inclusion
- Participants with Sturge-Weber syndrome brain involvement as defined on neuroimaging (n=10 subjects, male and female, ages 3 to 50 years of age) and the following:
- Cognitive impairment defined as a cognitive neuroscore greater than or equal to 2 at screening.
- Anti-epileptic, mood or behavioral drugs (if on) at stable doses for a minimum of 4 weeks prior to enrollment.
- If present, VNS must be on stable setting 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 fail at any point to meet continuation criteria and withdraw early may be considered for re-enrollment under 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
- Patients with any severe and/or uncontrolled medical conditions at randomization such as:
- Liver disease such as cirrhosis, decompensated liver disease, and chronic hepatitis (i.e. quantifiable HBV-DNA and/or positive HbsAg, quantifiable HCV-RNA)
- Uncontrolled diabetes as defined by fasting serum glucose greater than 1.5
- Active (acute or chronic) or uncontrolled severe infections
- Active, bleeding diathesis
- Patients who have 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 start or discontinue a seizure, mood or behavioral medication in the 4 weeks leading up to screening.
- Prior treatment with any investigational drug or use of any other cannabis product 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. This includes those in foster care, or those unable to keep follow-up appointments, maintain close contact with the Principal Investigator, or complete all necessary studies to maintain safety.
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of the female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
Key Trial Info
Start Date :
October 14 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 9 2022
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT04447846
Start Date
October 14 2019
End Date
December 9 2022
Last Update
May 25 2023
Active Locations (1)
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1
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205