Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome

NCT ID: NCT04447846

Last Updated: 2023-05-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-10-14
• Study Completion Date: 2022-12-09

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 phase I trial investigating the use of CBD for medically refractory seizures suggests CBD may also have a beneficial effect on cognition, mood, and behavior. The investigators hypothesize that CBD/ Epidiolex will improve SWS brain function resulting in improved cognitive function, social interactions, mood, motor function and behavior, as well as reduced migraines. This is an open-label prospective oral drug trial of Epidiolex in 10 subjects. Assessments will be done at baseline and repeated after 6 months on study drug.

Conditions

Sturge-Weber Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cannabidiol/ Epidiolex

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 24 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 cognitive impairments in patients with Sturge-Weber syndrome.

Group Type: EXPERIMENTAL

Cannabidiol

Intervention Type: DRUG

Initiation of treatment will begin with 5 mg/kg/day given in two divided doses. The dose will be increased by 5 mg/kg/day after seven days and then by 5 mg/kg/day every seven days up to a maximum dose of 20 mg/kg/day given.

Interventions

Cannabidiol

Initiation of treatment will begin with 5 mg/kg/day given in two divided doses. The dose will be increased by 5 mg/kg/day after seven days and then by 5 mg/kg/day every seven days up to a maximum dose of 20 mg/kg/day given.

Intervention Type: DRUG

Other Intervention Names

Epidiolex; CBD

Eligibility Criteria

Inclusion Criteria

• 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.
• Written informed consent obtained from the patient or the patient's legal representative must be obtained prior to beginning treatment.

Exclusion Criteria

• 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 HBV-DNA and/or positive HbsAg, quantifiable HCV-RNA)

2. Uncontrolled diabetes as defined by fasting serum glucose greater than 1.5

3. Active (acute or chronic) or uncontrolled severe infections

4. 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.

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Jazz Pharmaceuticals

INDUSTRY

Sponsor Role: collaborator

Faneca 66 Foundation

OTHER

Sponsor Role: collaborator

Anne Comi, MD

OTHER

Sponsor Role: lead

Responsible Party

Anne Comi, MD

Principal Investigator, Director Sturge-Weber Center, Kennedy Krieger Institute, Professor Johns Hopkins University School of Medicine

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Anne M Comi, MD

Role: PRINCIPAL_INVESTIGATOR

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Locations

Kennedy Krieger Institute

Baltimore, Maryland, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

https://www.kennedykrieger.org/patient-care/centers-and-programs/sturge-weber-syndrome-center

Hunter Nelson Sturge-Weber Center Website

Other Identifiers

IRB00204427

Identifier Type: -

Identifier Source: org_study_id