Refractory Childhood EpilepsyNCT02556008
Cannabidiol for Pediatric Epilepsy (Compassionate Use)
A study testing cannabidiol (CBD) in people with Refractory Childhood Epilepsy, run by University of California, Los Angeles at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Refractory Childhood Epilepsy
- What is being tested?
- cannabidiol (CBD)
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- Child
Who may be able to join
- Age criteria between the ages of 1 and 17 years.
- 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.
- 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.
- VNS must be on stable settings for a minimum of 3 months.
- If on ketogenic diet, must be on stable ratio for a minimum of 3 months
- Written informed consent obtained from the patient or the patient's legal representative must be obtained prior to beginning treatment.
Who cannot join
- Use of any "community acquired" cannabidiol product over the last 3 months.
- Use of any investigational treatments over the last 3 months.
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, Los Angeles
Los Angeles, California, 90095
About this study
Official title: 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.
Read the detailed description
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.
- Conditions
- Refractory Childhood Epilepsy
- Being tested
- cannabidiol (CBD)
- Keywords
- Children, Epilepsy, Cannabidiol
- Medical subject headings
- Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cannabidiol (CBD)Drug
Also called Epidiolex
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Childhood EpilepsyStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02556008
- Sponsor's protocol ID
- SH001
- Status last verified
- May 2017
- First submitted
- 18 Sep 2015
- Last updated
- 22 Jan 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cannabidiol for the Treatment of Pediatric Epilepsy (Expanded Access/Compassionate Use Protocol)
- ClinicalTrials.gov ID
- NCT02556008
- Acronym / sponsor ID
- SH001
- Conditions
- Refractory Childhood Epilepsy
- Keywords
- Children, Epilepsy, Cannabidiol
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- First posted
- 22 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Age groups
- Child
Key inclusion criteria (6 listed)
- Age criteria between the ages of 1 and 17 years.
- 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.
- 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.
- VNS must be on stable settings for a minimum of 3 months.
- If on ketogenic diet, must be on stable ratio for a minimum of 3 months
Key exclusion criteria (3 listed)
- Use of any "community acquired" cannabidiol product over the last 3 months.
- Use of any investigational treatments over the last 3 months.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| cannabidiol (CBD)Also: EpidiolexTreatment 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. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing cannabidiol (CBD) in people with Refractory Childhood Epilepsy, run by University of California, Los Angeles at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jan 2026.