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

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Brief Title

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

Official Title

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

Brief Summary

      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 Type

Observational


Primary Outcome

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.


Condition

Dravet Syndrome


Study Arms / Comparison Groups

 Dravet Sydrome
Description:  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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

19

Start Date

August 2014

Completion Date

July 2017

Primary Completion Date

July 2017

Eligibility Criteria

        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
      

Gender

All

Ages

N/A - 50 Years

Accepts Healthy Volunteers

No

Contacts

Edward Maa, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02229032

Organization ID

14-0294


Responsible Party

Sponsor

Study Sponsor

University of Colorado, Denver

Collaborators

 Denver Health Medical Center

Study Sponsor

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


Verification Date

December 2017