Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome

NCT04447846 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: IRB00204427
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

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).

Conditions

Sturge-Weber Syndrome

Keywords

Sturge-Weber Syndrome; SWS; Cognitive impairments

Outcome Measures

Primary Outcomes (1)

• List Sorting Working Memory Test

  Data on cognitive function was collected using the List Sorting Working Memory Test from the NIH Toolbox. Data was collected on working memory performance, which was transformed into a t-score from 0 to 100 where a higher t-score indicates better performance. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and after 6 months on study drug.

  Baseline, Follow-up (6 months)

Secondary Outcomes (18)

• Picture Vocabulary Test

  Baseline, Follow-up (6 months)

• Seizure Frequency

  Baseline, Follow-up (6 months)

• Migraine Severity

  Baseline, Follow-up (6 months)

• Modified House Classification Scores

  Baseline, Follow-up (6 months)

• Erhardt Developmental Prehension Assessment Scores

  Baseline, Follow-up (6 months)

Study Arms (1)

Cannabidiol/ Epidiolex

EXPERIMENTAL

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.

Drug: Cannabidiol

Interventions

Cannabidiol 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.

Also known as: Epidiolex, CBD

Cannabidiol/ Epidiolex

Eligibility Criteria

• Age: 3 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Anne Comi, MD: lead
• Jazz Pharmaceuticals: collaborator
• Faneca 66 Foundation: collaborator

Study Sites (1)

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Related Links

• Hunter Nelson Sturge-Weber Center Website

MeSH Terms

Conditions

Sturge-Weber Syndrome; Cognitive Dysfunction

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Hemangioma; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms; Neurocutaneous Syndromes; Nervous System Diseases; Angiomatosis; Vascular Diseases; Cardiovascular Diseases; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Limitations and Caveats

Due to the COVID-19 , visits transitioned from an in-person setting to a remote setting. Four subjects had baseline testing conducted in person and follow-up virtually, and 5 subjects had both baseline and follow-up testing virtually. The differences in testing environment and the context of the pandemic may have affected results. Also, due to variability in subject age and the small number of subjects, some participants were outside of assessment age norms.

Results Point of Contact

• Title: Dr. Anne Comi
• Organization: Kenndey Krieger Institute

comi@kennedykrieger.org

443-923-9127

Study Officials

• Anne M Comi, MD

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

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator, Director Sturge-Weber Center, Kennedy Krieger Institute, Professor Johns Hopkins University School of Medicine

Study Record Dates

• First Submitted: October 10, 2019
• First Posted: June 25, 2020
• Study Start: October 14, 2019
• Primary Completion: August 16, 2021
• Study Completion: December 9, 2022
• Last Updated: May 25, 2023
• Results First Posted: May 25, 2023

Record last verified: 2023-05

Locations

US (1)