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

NCT ID: NCT02229032

Last Updated: 2017-12-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

19 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-08-31

Study Completion Date

2017-07-31

Brief Summary

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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.

Detailed Description

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Conditions

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Dravet Syndrome

Keywords

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Dravet Syndrome cannabidiol therapy Charlotte's Web

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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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

No interventions assigned to this group

Eligibility Criteria

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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
Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Denver Health Medical Center

OTHER

Sponsor Role collaborator

University of Colorado, Denver

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Edward Maa, MD

Role: PRINCIPAL_INVESTIGATOR

Denver Health Medical Center

Locations

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Denver Health Medical Center

Denver, Colorado, United States

Site Status

Countries

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United States

Other Identifiers

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14-0294

Identifier Type: -

Identifier Source: org_study_id