Cannabidiol for Pediatric Epilepsy (Compassionate Use)
| Status: | Available |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 1 - 17 |
| Updated: | 4/21/2016 |
| Contact: | Ruby G Escalante |
| Email: | rubyescalante@mednet.ucla.edu |
| Phone: | 310-206-5586 |
Cannabidiol for the Treatment of Pediatric Epilepsy (Expanded Access/Compassionate Use Protocol)
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.
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.
Study Overview:
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:
- Change in average seizure severity by seizure type, defined as an increase or decrease
in seizure duration or intensity
- Change in the number of episodes of status epilepticus, defined as convulsive seizure
lasting longer than 10 minutes
- Change in the number of uses of rescue medications
- Change in the number of ER visits/ hospitalizations
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.
Benefits:
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.
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:
- Change in average seizure severity by seizure type, defined as an increase or decrease
in seizure duration or intensity
- Change in the number of episodes of status epilepticus, defined as convulsive seizure
lasting longer than 10 minutes
- Change in the number of uses of rescue medications
- Change in the number of ER visits/ hospitalizations
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.
Benefits:
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.
Inclusion Criteria:
1. Age criteria between the ages of 1 and 17 years.
2. Documentation of a diagnosis of drug resistant epilepsy as evidenced by failure to
control seizures despite appropriate trial of two or more AEDs at therapeutic doses.
Documentation must include the diagnosis of epilepsy type or epilepsy syndrome, as
well as the underlying cause, when known.
3. Between 1-3 baseline anti-epileptic drugs at stable doses for a minimum of 4 weeks
prior to enrollment. Vagus nerve stimulator (VNS), ketogenic diet and modified Atkins
diet do not count toward this limit.
4. VNS must be on stable settings for a minimum of 3 months.
5. If on ketogenic diet, must be on stable ratio for a minimum of 3 months
6. Written informed consent obtained from the patient or the patient's legal
representative must be obtained prior to beginning treatment.
Exclusion Criteria:
1. Use of any "community acquired" cannabidiol product over the last 3 months.
2. Use of any investigational treatments over the last 3 months.
3. Patients with a history of non-compliance to medical regimens or who are considered
potentially unreliable or will not be able to complete entire study.
We found this trial at
1
site
Los Angeles, California 90095
310-825-4321
Principal Investigator: Shaun A Hussain, MD
Phone: 310-206-5586
University of California at Los Angeles The University of California, Los Angeles (UCLA) is an...
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