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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02229032
Recruitment Status : Completed
First Posted : August 29, 2014
Last Update Posted : December 15, 2017
Sponsor:
Collaborator:
Denver Health Medical Center
Information provided by (Responsible Party):
University of Colorado, Denver

Study Description
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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.

Condition or disease
Dravet Syndrome

Study Design
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Study Type : Observational
Actual Enrollment : 19 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population
Actual Study Start Date : August 2014
Actual Primary Completion Date : July 2017
Actual Study Completion Date : July 2017

Resource links provided by the National Library of Medicine


Groups and Cohorts
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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


Outcome Measures
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Primary Outcome Measures :
  1. 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


Biospecimen Retention:   Samples With DNA
saliva for DNA analysis

Eligibility Criteria
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Ages Eligible for Study:   up to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02229032


Locations
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United States, Colorado
Denver Health Medical Center
Denver, Colorado, United States, 80204
Sponsors and Collaborators
University of Colorado, Denver
Denver Health Medical Center
Investigators
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Principal Investigator: Edward Maa, MD Denver Health Medical Center
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT02229032    
Other Study ID Numbers: 14-0294
First Posted: August 29, 2014    Key Record Dates
Last Update Posted: December 15, 2017
Last Verified: December 2017
Keywords provided by University of Colorado, Denver:
Dravet Syndrome
cannabidiol therapy
Charlotte's Web
Additional relevant MeSH terms:
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Epilepsies, Myoclonic
Epilepsy, Generalized
Epilepsy
Brain Diseases
 Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes