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

Dravet Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02229032
• Other study ID #: 14-0294
• Status: Completed
• Phase: N/A
• First received: August 27, 2014
• Last updated: December 13, 2017
• Start date: August 2014
• Est. completion date: July 2017

Study information

• Verified date: December 2017
• Source: University of Colorado, Denver
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: July 2017
• Est. primary completion date: July 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 50 Years

Eligibility

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

Related Conditions & MeSH terms

• Dravet Syndrome
• Epilepsies, Myoclonic

Locations

Country	Name	City	State
United States	Denver Health Medical Center	Denver	Colorado

Sponsors (2)

Lead Sponsor	Collaborator
University of Colorado, Denver	Denver Health Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	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