Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population

December 13, 2017 updated by: University of Colorado, Denver
There is tremendous curiosity about medical marijuana and the treatment of epilepsy. In a specific genetic epilepsy known as Dravet Syndrome, a mutation occurs affecting the SCN1A gene. A specific strain of marijuana known as Charlotte's Web, available in Colorado, may have activity in this catastrophic epilepsy syndrome. Anecdotal reports suggest both success and lack of response with this therapy. Genetic analysis of the differences between Dravet responders and non-responders may prove useful for identifying patients likely to be helped by this therapy, as well as shed light on the putative mechanisms by which marijuana may exert any antiepileptic effect.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

19

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Denver, Colorado, United States, 80204
        • Denver Health Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 50 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with Dravet Syndrome, ages 1-50, who are self- seeking therapy with Charlotte's Web strain of medical marijuana with the assistance of a medical marijuana doctor, but are still naïve to therapy

Description

Inclusion Criteria:

  • age 1-50 with gene confirmed Dravet Syndrome
  • uncontrolled epilepsy, having failed at least 2 prior treatments (including appropriate Anti-epileptic drugs, ketogenic diet, vagus nerve stimulation Therapy)
  • Seizure frequency should be more than 2 visually countable motor seizures per week (tonic clonic, tonic, clonic, myoclonic, or astatic)
  • care providers able to identify convulsive seizures, drop attacks, or tonic seizures, or any combination of countable seizure types
  • self- seeking therapy with Charlotte's Web strain of medical marijuana with the assistance of a medical marijuana doctor, but are still naïve to therapy
  • care provider able to complete seizure count diary
  • able to obtain oral swab sample from subject with Dravet Syndrome
  • agree to forfeit any right to results of genetic analysis

Exclusion Criteria:

  • subjects having non-epileptic seizures that are not distinguishable from epileptic seizures by the care provider
  • prisoners
  • pregnant women
  • failure to forfeit right to results of genetic analysis

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Cross-Sectional

Cohorts and Interventions

Group / Cohort
Dravet Sydrome
Patients with Dravet Syndrome who are self-seeking therapy with Charlotte's Web strain of medical marijuana with the assistance of a medical marijuana doctor, but are still naïve to therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
genetic differences between patients with Dravet Syndrome (SCN1A gene mutation) who appear to respond to high concentration cannabidiol (CBD) oil therapy versus those who do not.
Time Frame: 1 day
Patients completing three months of therapy with a seizure reduction of >50% will be designated a "Responder" and patients who discontinue therapy for any reason prior to 3 months or have a <=50% seizure reduction will be designated "Non-Responder". Between-groups genetic analysis will be performed
1 day

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Colorado, Denver

Collaborators

Denver Health Medical Center

Investigators

  • Principal Investigator: Edward Maa, MD, Denver Health Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2014

Primary Completion (Actual)

July 1, 2017

Study Completion (Actual)

July 1, 2017

Study Registration Dates

First Submitted

August 27, 2014

First Submitted That Met QC Criteria

August 28, 2014

First Posted (Estimate)

August 29, 2014

Study Record Updates

Last Update Posted (Actual)

December 15, 2017

Last Update Submitted That Met QC Criteria

December 13, 2017

Last Verified

December 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 14-0294

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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