Drug Resistant EpilepsyNCT03676049
Cannabidiol for Drug Resistant Pediatric Epilepsy (Expanded Access Use)
A study testing Cannabidiol in people with Drug Resistant Epilepsy, run by John at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 to 19 years
- Conditions the study is for
- Drug Resistant Epilepsy
- What is being tested?
- Cannabidiol
- 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 5 to 19 years
- All sexes
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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.
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- Significant impaired hepatic function at screening as characterized by one of the following:
- 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
- History or presence of alcohol or substance abuse
- Use of other cannabis or CBD products
Trial locations and contacts
1 site in 1 country.
United States1 site
- Available
University of Mississippi Medical Center
Jackson, Mississippi, 39216
About this study
Official title: Cannabidiol (CBD) Cannabis Extract Oral Solution for Drug Resistant Pediatric Epilepsy (Expanded Access Use)
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.
Read the detailed description
Expanded Access Use: Restrictions to those with most significant epilepsy severity and especially neurocognitive delays (to minimize side effect profiles) remain in place. Study design, hover, is targeted at an Expanded Access use of product for children who would otherwise have no appropriate remaining treatment modality left.
Timeline: 3 months with the option to continue for up to 3 months of follow up therapy.
Procedures:
Participants: Five to ten (5-10) refractory epilepsy patients, aged 5-19 years old, all sexes, who must have failed at least 3 appropriate drugs, +/- vagal nerve stimulator, +/- dietary therapy, +/- resective surgery will be provided CBD as part of the Principal Investigator's Physician Expanded Access IND.
Written parental permission obtained from the patient's parent or legal representative must be obtained prior to beginning treatment.
Drug administration: 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. Any patients under 40kg will have adjusted dosing based on weight as follows:
For 40 kg and up:
1 ml BID, escalating to 2 ml BID, and then 3 ml BID
For 30 - 39 kg:
0.75 ml BID, escalating to 1.5 and 2.25 ml BID
For 20 - 29 kg 0.5 ml BID, escalating to 1 and 1.5 ml BID
Clinical evaluations: All subjects will be clinically evaluated at baseline, once a month for the three months of titration period and during the three months of follow up period. During the first week of the study drug initiation, patients will be called at day 3 and then at day 7 to review any changes in condition. Subsequently, patients will receive a phone call weekly and will be evaluated in their physician study visits during the titration period to review any changes in condition and determine whether an increase in dosing is required. The investigator will be available by telephone throughout the 3 months of the titration period of the study, in between the visits and throughout the 3 months in the follow up period. Patients will receive the CBD during their monthly study visits during the titration and follow up period. The first dose of CBD will be administered by the physician in the Pediatric Clinical Research Clinic.
Pregnant patients will be excluded from the study. Female 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. 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.
During the study, the patients will be constantly monitored as the investigators describe below:
Onset:
- Pediatric Neurology Physician evaluation.
- Initial lab work to include complete blood counts, comprehensive metabolic panel and baseline co-existent antiepileptic drug levels. Blood pregnancy testing will be performed on those patients with childbearing potential.
- Initial body weight and routine vital signs
- Initial baseline Electroencephalogram (EEG). Video EEG prior to enrollment if epileptic syndrome or severity is unclear. Decision and enrollment classification to be made by Dr. Ingram prior to enrollment.
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview prior to enrollment. 4 weeks/1 month:
- Pediatric Neurology Physician evaluation.
- Lab work to include complete blood counts, comprehensive metabolic panel and concomitant epilepsy medication levels. Blood testing for CBD/THC will also be performed. Additional lab draws can be performed with any symptoms to suggest anticonvulsant toxicity or concerning clinical signs/symptoms.
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 1-month study window. 8 weeks/2 months:
- Pediatric Neurology Physician evaluation.
- Lab work as indicated for 4 weeks/1 month.
- Body weight and routine vital signs
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 2-months study window. 12 weeks/3 months:
- Pediatric Neurology Physician evaluation.
- EEG at the completion of the 3-month study window.
- Lab work as indicated for 4 weeks/1 month.
- Body weight and routine vital signs
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 3-months study window.
Follow up:
Patients who are stable on therapy and happy with the results may remain on study for up to 3 more months after titration period. These patients will have 6 months total on medication and one visit/month during the follow up time for clinical evaluation:
- Complete blood count
- Comprehensive metabolic profile (including liver function tests)
- Measurement of levels of concomitant anti-epileptic drugs
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview.
- EEG (at 6 months visit)
- Conditions
- Drug Resistant Epilepsy
- Being tested
- Cannabidiol
- Keywords
- Children, Epilepsy, Cannabidiol
- Medical subject headings
- Drug Resistant Epilepsy, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CannabidiolDrug
Also called CBD
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Drug Resistant EpilepsyStudies of the same condition, open and under way.
- 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
- John
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, John, University of Mississippi Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03676049
- Sponsor's protocol ID
- 2017-0259
- Status last verified
- May 2024
- First submitted
- 11 Sep 2018
- Last updated
- 9 May 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cannabidiol (CBD) Cannabis Extract Oral Solution for Drug Resistant Pediatric Epilepsy (Expanded Access Use)
- ClinicalTrials.gov ID
- NCT03676049
- Acronym / sponsor ID
- 2017-0259
- Conditions
- Drug Resistant Epilepsy
- Keywords
- Children, Epilepsy, Cannabidiol
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- JohnUniversity, hospital or other
- First posted
- 18 Sep 2018 (actual)
- 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 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.
Detailed description
Expanded Access Use: Restrictions to those with most significant epilepsy severity and especially neurocognitive delays (to minimize side effect profiles) remain in place. Study design, hover, is targeted at an Expanded Access use of product for children who would otherwise have no appropriate remaining treatment modality left.
Timeline: 3 months with the option to continue for up to 3 months of follow up therapy.
Procedures:
Participants: Five to ten (5-10) refractory epilepsy patients, aged 5-19 years old, all sexes, who must have failed at least 3 appropriate drugs, +/- vagal nerve stimulator, +/- dietary therapy, +/- resective surgery will be provided CBD as part of the Principal Investigator's Physician Expanded Access IND.
Written parental permission obtained from the patient's parent or legal representative must be obtained prior to beginning treatment.
Drug administration: 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. Any patients under 40kg will have adjusted dosing based on weight as follows:
For 40 kg and up:
1 ml BID, escalating to 2 ml BID, and then 3 ml BID
For 30 - 39 kg:
0.75 ml BID, escalating to 1.5 and 2.25 ml BID
For 20 - 29 kg 0.5 ml BID, escalating to 1 and 1.5 ml BID
Clinical evaluations: All subjects will be clinically evaluated at baseline, once a month for the three months of titration period and during the three months of follow up period. During the first week of the study drug initiation, patients will be called at day 3 and then at day 7 to review any changes in condition. Subsequently, patients will receive a phone call weekly and will be evaluated in their physician study visits during the titration period to review any changes in condition and determine whether an increase in dosing is required. The investigator will be available by telephone throughout the 3 months of the titration period of the study, in between the visits and throughout the 3 months in the follow up period. Patients will receive the CBD during their monthly study visits during the titration and follow up period. The first dose of CBD will be administered by the physician in the Pediatric Clinical Research Clinic.
Pregnant patients will be excluded from the study. Female 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. 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.
During the study, the patients will be constantly monitored as the investigators describe below:
Onset:
- Pediatric Neurology Physician evaluation.
- Initial lab work to include complete blood counts, comprehensive metabolic panel and baseline co-existent antiepileptic drug levels. Blood pregnancy testing will be performed on those patients with childbearing potential.
- Initial body weight and routine vital signs
- Initial baseline Electroencephalogram (EEG). Video EEG prior to enrollment if epileptic syndrome or severity is unclear. Decision and enrollment classification to be made by Dr. Ingram prior to enrollment.
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview prior to enrollment. 4 weeks/1 month:
- Pediatric Neurology Physician evaluation.
- Lab work to include complete blood counts, comprehensive metabolic panel and concomitant epilepsy medication levels. Blood testing for CBD/THC will also be performed. Additional lab draws can be performed with any symptoms to suggest anticonvulsant toxicity or concerning clinical signs/symptoms.
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 1-month study window. 8 weeks/2 months:
- Pediatric Neurology Physician evaluation.
- Lab work as indicated for 4 weeks/1 month.
- Body weight and routine vital signs
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 2-months study window. 12 weeks/3 months:
- Pediatric Neurology Physician evaluation.
- EEG at the completion of the 3-month study window.
- Lab work as indicated for 4 weeks/1 month.
- Body weight and routine vital signs
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 3-months study window.
Follow up:
Patients who are stable on therapy and happy with the results may remain on study for up to 3 more months after titration period. These patients will have 6 months total on medication and one visit/month during the follow up time for clinical evaluation:
- Complete blood count
- Comprehensive metabolic profile (including liver function tests)
- Measurement of levels of concomitant anti-epileptic drugs
- Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview.
- EEG (at 6 months visit)
Study design
Who may qualify
- Ages
- 5 to 19 years
- Sex
- All
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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.
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 |
|---|---|---|
| CannabidiolAlso: CBDCBD 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. | 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 9 May 2024.
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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.
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 in people with Drug Resistant Epilepsy, run by John 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 10 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 9 May 2024. 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?
Contact John B Ingram, M.D. ((601) 984-5210 ext. 4-5210, jingram@umc.edu), or a site near the patient. 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 9 May 2024.