Cannabidiol for Pediatric Epilepsy (Compassionate Use)
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|ClinicalTrials.gov Identifier: NCT02556008|
Expanded Access Status : No longer available
First Posted : September 22, 2015
Last Update Posted : May 3, 2017
|First Submitted Date||September 18, 2015|
|First Posted Date||September 22, 2015|
|Last Update Posted Date||May 3, 2017|
|Brief Title||Cannabidiol for Pediatric Epilepsy (Compassionate Use)|
|Brief Summary||This is an open-label observational study of pure CBD for the treatment for 25 children with intractable epilepsy. As pure CBD is not FDA approved, the investigators are conducting this study via the FDA expanded access mechanism on a compassionate use basis. The target patient population is children with severe refractory epilepsy who have exhausted all other reasonable avenues of treatment. These are patients for whom the risks of a relatively untested product are outweighed by the potential benefit. Using seizure-diaries maintained on a routine clinical basis, seizure frequency will be assessed four weeks prior to initiation of CBD, one month after CBD initiation, and at least every 3 months thereafter. CBD will be administered as an adjunct to all current anti-epileptic therapies.|
The investigators hope to gain an understanding of the utility of pure CBD use for the treatment of drug resistant epilepsy in this open-label dose-finding observational study. A baseline seizure frequency will be recorded for each subject in a diary for four weeks prior to investigational drug initiation and parents/caregivers will document seizures on a daily basis throughout the study period. Investigational drug (CBD) will be administered as an adjunct to all current anti-epileptic drugs. Once at their maintenance daily dose they will be evaluated one month after achieving the final steady state dose and every three months thereafter.
Visits will consist of neurological exam and seizure diary review. Baseline visit will take 1 hour and each visit thereafter will take approximately 30 minutes.
Safety and Tolerability Measurements:
Data on safety and tolerability of pure CBD is limited. According to the GW Pharma CBD Investigator's Brochure (Appendix G), side effects reported as greater than placebo with highly purified CBD in one clinical trial were conjunctival hemorrhage, change in vision, diarrhea, flatulence, gastric reflux, joint pain, muscle pain, difficulty concentrating, abnormal mood and trouble sleeping.
The patients will be closely monitored for side effects during the titration and treatment period and the dose and/or frequency may be adjusted as appropriate.
CBD is an inhibitor of CYP 2C19, CYP 2C9, and other cytochrome P450s belonging to the 2C and 3A subfamilies. The effects of CBD administered concomitantly with anti-epileptic drugs that are metabolized by this enzyme system are unknown. Anti-epileptic drug doses will be adjusted as needed based on signs and symptoms of toxicity and / or changes in drug levels.
Measurement of changes in seizure frequency:
All seizure types will be classified before study entry. Seizure type and frequency will be monitored during baseline and treatment as recorded in a patient diary. For assessing the efficacy of CBD, the investigator will count only change in the frequency of seizures (ie, tonic seizures, clonic seizures, tonic-clonic seizures lasting more than 3 seconds, atonic seizures, myoclonic seizures including myoclonic absences). In addition, parents/caregivers will report:
Data Safety Monitoring:
The Principal Investigator will review all data relating to safety and tolerability throughout the study, after every third patient has been treated, to monitor study conduct and assess patient safety.
Subject may have a reduction in seizures. This study will help the researchers learn more about the effectiveness and safety of CBD in the treatment of drug-resistant epilepsy. Hopefully this information will help in the treatment of future patients with this condition.
|Study Type||Expanded Access|
|Intervention||Drug: cannabidiol (CBD)
Treatment will begin with 2 mg/kg/day given in two divided doses. The dose will be increased by 3 mg/kg/day after seven days and then by 5 mg/kg/day every seven days up to a maximum dose of 25 mg/kg/day given.
Other Name: Epidiolex
|Publications *||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Expanded Access Status||No longer available|
|Contacts||Contact information is only displayed when the study is recruiting subjects|
|Listed Location Countries||United States|
|Removed Location Countries|
|Responsible Party||Shaun Hussain, MD, University of California, Los Angeles|
|Study Sponsor||University of California, Los Angeles|
|PRS Account||University of California, Los Angeles|
|Verification Date||May 2017|