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

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by University of Colorado, Denver
Sponsor:
Collaborator:
Denver Health Medical Center
Information provided by (Responsible Party):
University of Colorado, Denver
ClinicalTrials.gov Identifier:
NCT02229032
First received: August 27, 2014
Last updated: September 3, 2014
Last verified: September 2014
  Purpose

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
Dravet Syndrome

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population

Resource links provided by NLM:


Further study details as provided by University of Colorado, Denver:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    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


Estimated Enrollment: 150
Study Start Date: August 2014
Estimated Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts
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

  Eligibility

Ages Eligible for Study:   up to 50 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02229032

Contacts
Contact: Edward Maa, MD 303-602-5099 edward.maa@ucdenver.edu

Locations
United States, Colorado
Denver Health Medical Center Recruiting
Denver, Colorado, United States, 80204
Contact: Edward Maa    303-602-5099    edward.maa@ucdenver.edu   
Principal Investigator: Edward Maa, MD         
Sponsors and Collaborators
University of Colorado, Denver
Denver Health Medical Center
Investigators
Principal Investigator: Edward Maa, MD Denver Health Medical Center
  More Information

No publications provided

Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT02229032     History of Changes
Other Study ID Numbers: 14-0294
Study First Received: August 27, 2014
Last Updated: September 3, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by University of Colorado, Denver:
Dravet Syndrome
cannabidiol therapy
Charlotte's Web

Additional relevant MeSH terms:
Syndrome
Disease
Pathologic Processes

ClinicalTrials.gov processed this record on October 23, 2014