Cannabidiol for Drug Resistant Pediatric Epilepsy (Expanded Access Use)
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ClinicalTrials.gov Identifier: NCT03676049
Expanded Access Status :
Temporarily not available
This is a treatment study under an approved Expanded Use IND protocol for using Cannabidiol (CBD) Extract. CBD will be used for the treatment of 5-10 children with drug resistant epilepsy. The CBD used in this study is prepared at the University of Mississippi under approval of the National Institute on Drug Abuse (NIDA) for its preparation and FDA approval under an expanded access mechanism on a compassionate use basis. The target patient population is who would otherwise have no appropriate remaining treatment modality left. These are patients for whom the risks of a relatively untested product are outweighed by the potential benefit. Using seizure-diaries to register seizure frequency, drug log and questionnaire to measure parent/patient quality of life and side effects will be assessed in each visit. Visits are: baseline, 4, 8, and 12 weeks visit. A 24 weeks visit (6 months) will be performed if the patient is stable on therapy during the 3 initial months and want to continue on the study for 3 more months. CBD will be administered as an adjunct to all current anti-epileptic therapies.
CBD will be administered as an adjunct to all current antiepileptic drugs. A dosing titration period will start with 100 mg/day, with 50mg BID dosing, and will be titrated monthly as tolerated based on clinical response, up to 300mg/day. This is based on a targeted dosing of 2.5 ---5---7.5 mg/kg/d.
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Layout table for eligibility information
Ages Eligible for Study:
5 Years to 19 Years (Child, Adult)
Sexes Eligible for Study:
Patients meeting the following criteria will be considered eligible for this study:
Age 5-19 years old
Documentation of a diagnosis of drug resistance epilepsy as evidenced by failure to control seizures despite appropriate trial of three or more anti-epileptic drugs at therapeutic doses. Some of these drugs include Brivaracetam, Carbamazepine, Clobazam, Clonazepam, Diazepam, Divalproex Sodium, Eslicarbazepine Acetate, Ethosuximide, Felbamate, Gabapentin, Lacosamide, Lamotrigine, Levetiracetam, Lorazepam, Oxcarbazepine, Perampanel, Phenobarbital, Phenytoin, Pregabalin, Rufinamide, Valproic Acid, Vigabatrin, Zonisamide. Documentation must include the diagnosis of epilepsy type or epilepsy syndrome (if possible), as well as the underlying case, when known.
Between 1-3 baseline anti-epileptic drugs at stable doses for a minimum of 4 weeks prior to enrollment. Vagus nerve stimulator, and dietary therapies do not count toward this parameter, but are not bases for exclusion.
Vagus nerve stimulator must be on stable settings for a minimum of 3 months.
A history of significant epilepsy severity and neurocognitive delays, specifically a baseline history of being nonverbal and non-ambulatory without assistance
The patient may not enter the study if ANY of the following apply:
Patients with resectable epilepsy or syndromes where dietary or other therapy is first line will not be enrolled prior to attempting those therapies with failure OR parental refusal of said therapies.
Treatment with any artisanal preparation containing or possible containing CBD during the month before initiation of the study drug.
Allergy to CBD or any cannabinoid.
Unable to comply with study visits/requirements.
CBD is contraindicated in pregnancy and during breast-feeding. Female subjects who are pregnant or breastfeeding will be excluded from treatment with CBD. Patients of childbearing potential will be screened for pregnancy prior to enrollment and during the study if a potential for pregnancy arise. A blood pregnancy test will be used. Additionally, for patients of childbearing potential, enrollment criteria will require the use of highly effective contraception (e.g. double barrier, abstinence, or IUD) for the duration of treatment with CBD. Therefore, parent/guardian of female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if the child become pregnant during the study.
Significant impaired hepatic function at screening as characterized by one of the following:
i. Alanine aminotransferase [ALT] >5 × upper limit of normal (ULN) and total bilirubin (TBL) >2 × ULN ii. ALT or Aspartate aminotransferase (AST) >3 × ULN and either TBL >2 × ULN or international normalized ratio (INR) >1.5