The Use of Medicinal Cannabinoids as Adjunctive Treatment for Medically Refractory Epilepsy

• Conditions: Epilepsy, Unspecified, Refractory (Medically)
• Interventions: Drug: Medical Cannabis

• Registration Number: NCT02523183
• Lead Sponsor: University of Colorado, Denver

Brief Summary

Many families of children with medically refractory epilepsy are choosing to use medicinal cannabinoids (MCBD) as an adjunctive alternative treatment option. The safety, tolerability and efficacy of these products are not known. The primary objective of this study is to determine how the use of MCBD affects children with medically refractory epilepsy in an observational study. Measures of evaluation to be used will include: laboratory values, developmental measures, seizure diaries and serial electroencephalographic (EEG) recordings.

Detailed Description

This is an observational study of a family's choice to use MCBD and the effect of this substance on medically refractory epilepsy in children. The risks associated with this use are not well known. There have recently been reports of stroke, liver dysfunction and altered anticonvulsant levels. In addition, there have been long term risks reported of decreased memory function, cognitive problems and executive function abnormalities. This study does not condone or advocate the use of the substance but merely seeks to document the short term effects of the use in this population. It should be noted that providers at Children's Hospital Colorado are not registered providers for medicinal cannabis; therefore, this will be an observational study of effects of this substance as prescribed by other physicians outside of our institution.

Study Timeline

• Study Start: 2015-07
• Study First Submitted: 2015-07-23
• Study First Submitted QC: 2015-08-11
• Study First Posted: 2015-08-14
• Status Verified: 2020-01
• Last Update Submitted: 2020-01-14
• Last Update Posted: 2020-01-18
• Primary Completion: 2020-10
• Study Completion: 2020-11

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

1. Male or Female >1 month of age

2. Documentation of a diagnosis of medically refractory epilepsy as evidenced by medical records, genetic testing and/or the following clinical features:

   • Failure to control seizures despite an appropriate trial of two anticonvulsant medications at therapeutic doses

3. Baseline seizure frequency of at least 2 per week of the any of the following types:

   • Generalized tonic-clonic
   • Clonic
   • Tonic
   • Hemiconvulsive
   • Drop attacks
   • Focal motor
   • Epileptic spasms

4. 1-3 baseline anti-seizure medications at stable doses for a minimum of 4 weeks prior to enrollment.

5. Written informed consent obtained from the patient or the patient's legal representative.

Exclusion Criteria

1. Epilepsies associated with rapidly progressing neurodegenerative diseases ex: Rasmussen encephalitis, and tumors.
2. Epilepsies associated with treatable inborn errors of metabolism
3. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
4. Non-epileptic events.
5. Current use of MCBD products (Note: Patient is eligible if currently using MCBD but will be switching to a different product).

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Subjects with medically refractory epilepsy	Medical Cannabis	Pediatric epilepsy patients who are followed at Children's Hospital Colorado with medically refractory epilepsy, and whom the family has decided to treat with medical cannabis.

Primary Outcome Measures

Name	Time	Method
Pediatric Epilepsy Side Effects Questionnaire	Baseline, 4 weeks, 8 weeks, and 12 weeks	Side effects will be evaluated using PESQ (Pediatric epilepsy Side Effects Questionnaire). This questionnaire will be administered 4 weeks, 8 weeks, and 12 weeks after baseline.
Changes from baseline in background and architecture of seizures confirmed by non-investigational EEG	Baseline, 4 weeks, 8 weeks, and 12 weeks	Prolonged EEG recordings lasting 24-48 hours (whichever is clinically indicated) will be performed in either the home setting as an ambulatory EEG or in the hospital in the Epilepsy Monitoring Unit. This decision will be made on a clinical basis. This will allow for objective quantification of seizures in children with frequent seizures that occur on a daily basis and background EEG changes for other children. Two EEGs will be done: one at baseline, and one at the end of the study.
Seizure Frequency	Baseline, 4 weeks, 8 weeks, and 12 weeks	Each subject will maintain a seizure diary. Rescue drug use and VNS (Vagus Nerve Stimulation) activations will be tracked as an adjunct to all current anti-seizure medications. Frequency of status epilepticus will be tracked as well as hospital visits for seizures or adverse events from MCBD. Clinical evaluations will be taken every 2 months throughout the duration of the study. Subjects will be monitored every 2 weeks by telephone and/or email.

Trial Locations

Locations (1)

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States