Dravet SyndromeNCT02910297
The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy
An observational study of Dravet Syndrome and Lennox Gastaut Syndrome, run by Gillette Children's Specialty Healthcare.
Trial summary
The purpose of this study is to determine if cannabidiol (CBD) obtained via the state of Minnesota reduces seizures in patients with severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome), and to measure blood levels of CBD to help determine CBD concentration-response characteristics. Read more about this study
- Who is it for?
- Ages 2 to 30 years
- Conditions the study is for
- Dravet Syndrome, Lennox Gastaut Syndrome
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Sep 2016to 31 Dec 2017
- Phase
- Observational
- 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 2 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Subjects who have registered or plan to register with the state of Minnesota for taking medical cannabis and have severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome)
Who may be able to join
- Diagnosed with Dravet Syndrome or Lennox-Gastaut Syndrome
- Patients who are planning to obtain medical cannabidiol
- Patients who are already taking medical cannabidiol and are planning to stop taking it.
Who cannot join
- Patients without a diagnosis of Dravet syndrome or Lennox-Gastaut syndrome
About this study
The purpose of this study is to determine if cannabidiol (CBD) obtained via the state of Minnesota reduces seizures in patients with severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome), and to measure blood levels of CBD to help determine CBD concentration-response characteristics.
Read the detailed description
Patients with medically refractory epilepsy who are registering to take CBD in the State of Minnesota will be offered the opportunity to participate in this study. If consent to participation is obtained, the patient (or more likely, the patient's guardian) will be asked to begin a seizure diary for at least a 7-day period prior (ideally, longer) to receiving the CBD, and to continue tracking seizures throughout the study. Upon receipt of CBD from the Minnesota dispensary, subjects will take their CBD as directed and will return to the clinic for a repeat visit (which is part of standard care) occurring 2-8 weeks after continual use of CBD. During these clinic visits, patients undergo blood draws to monitor their other prescribed AED concentrations as part of standard of care. In addition to the standard blood draw, the research participant will be asked to provide a blood sample of 5 ml to measure CBD concentrations. As most patients are instructed to take CBD two hours prior to their standard of care AEDs, clinical experience indicates that most patients are taking CBD quite early in the morning (around 6 AM). Thus, the time of sample collection will be recorded as hours post-dose, and will likely be nearer peak serum concentrations. If a study participant has an indwelling intravenous access port, they will be asked to provide an additional sample of 5 ml at least one hour after the initial sample. During the repeat visit, subjects will be asked to continue documenting seizure activity in their seizure diary while they continue their CBD treatment. Any changes to AEDs administered concurrently with CBD will also be recorded.
Subjects who are already on a stable regimen of CBD will also be eligible to participate. At a clinic visit, they will be asked to provide a blood sample of 5 ml, the time of each sample will be recorded as hours post-dose of CBD. They will also be asked to begin a seizure diary and continue tracking seizure activity. If a patient terminates their CBD treatment, they will be asked to continue tracking seizure activity after their last CBD dose until their follow-up clinic visit, to determine change in seizure activity while on and off CBD.
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome
- Keywords
- cannabidiol, Dravet Syndrome, Lennox Gastaut Syndrome
- Medical subject headings
- Epilepsies, Myoclonic, Lennox Gastaut Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in seizures
Subjects or parents/guardians of subjects will record seizure activity in seizure diaries both prior to and during cannabidiol administration
Measured: a 7-day seizure diary completed at least twice within 3 months
Secondary outcomes (1)
Cannabidiol concentration
Each subject will provide a blood sample of 5 ml to measure concentrations of cannabidiol
Measured: 1 time at a clinic visit within three months. If a patient has a port, they will be asked to provide an additional blood sample of 5 ml at least one hour after the first sample during the same clinic visit.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2016 (estimated)
- Study startSep 2016
- Primary completion31 Dec 2017
- Study completion31 Dec 2017
Why it stopped: unable to recruit enough subjects on CBD
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dravet SyndromeStudies 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
- Observational
- Observational model
- Case crossover
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gillette Children's Specialty Healthcare
- Sponsor type
- University, hospital or other
- Collaborators
- University of Minnesota
- Responsible party
- Sponsor
- Investigators
- Samuel A Roiko, Ph.D., Principal investigator, Gillette Children's Specialty Healthcare
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02910297
- Sponsor's protocol ID
- CBD-PK-1
- Status last verified
- Dec 2021
- First submitted
- 11 Aug 2016
- Last updated
- 16 Dec 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy
- ClinicalTrials.gov ID
- NCT02910297
- Acronym / sponsor ID
- CBD-PK-1
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome
- Keywords
- cannabidiol, Dravet Syndrome, Lennox Gastaut Syndrome
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: unable to recruit enough subjects on CBD
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Gillette Children's Specialty HealthcareUniversity, hospital or other
- Collaborators
- University of Minnesota
- Study start
- Sep 2016 (actual)
- Primary completion
- 31 Dec 2017 (actual)
- Study completion
- 31 Dec 2017 (actual)
- First posted
- 22 Sep 2016 (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.
The purpose of this study is to determine if cannabidiol (CBD) obtained via the state of Minnesota reduces seizures in patients with severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome), and to measure blood levels of CBD to help determine CBD concentration-response characteristics.
Detailed description
Patients with medically refractory epilepsy who are registering to take CBD in the State of Minnesota will be offered the opportunity to participate in this study. If consent to participation is obtained, the patient (or more likely, the patient's guardian) will be asked to begin a seizure diary for at least a 7-day period prior (ideally, longer) to receiving the CBD, and to continue tracking seizures throughout the study. Upon receipt of CBD from the Minnesota dispensary, subjects will take their CBD as directed and will return to the clinic for a repeat visit (which is part of standard care) occurring 2-8 weeks after continual use of CBD. During these clinic visits, patients undergo blood draws to monitor their other prescribed AED concentrations as part of standard of care. In addition to the standard blood draw, the research participant will be asked to provide a blood sample of 5 ml to measure CBD concentrations. As most patients are instructed to take CBD two hours prior to their standard of care AEDs, clinical experience indicates that most patients are taking CBD quite early in the morning (around 6 AM). Thus, the time of sample collection will be recorded as hours post-dose, and will likely be nearer peak serum concentrations. If a study participant has an indwelling intravenous access port, they will be asked to provide an additional sample of 5 ml at least one hour after the initial sample. During the repeat visit, subjects will be asked to continue documenting seizure activity in their seizure diary while they continue their CBD treatment. Any changes to AEDs administered concurrently with CBD will also be recorded.
Subjects who are already on a stable regimen of CBD will also be eligible to participate. At a clinic visit, they will be asked to provide a blood sample of 5 ml, the time of each sample will be recorded as hours post-dose of CBD. They will also be asked to begin a seizure diary and continue tracking seizure activity. If a patient terminates their CBD treatment, they will be asked to continue tracking seizure activity after their last CBD dose until their follow-up clinic visit, to determine change in seizure activity while on and off CBD.
Study design
- Observational model
- Case crossover
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Subjects who have registered or plan to register with the state of Minnesota for taking medical cannabis and have severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome)
Key inclusion criteria (3 listed)
- Diagnosed with Dravet Syndrome or Lennox-Gastaut Syndrome
- Patients who are planning to obtain medical cannabidiol
- Patients who are already taking medical cannabidiol and are planning to stop taking it.
Key exclusion criteria (1 listed)
- Patients without a diagnosis of Dravet syndrome or Lennox-Gastaut syndrome
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Reduction in seizures
Time frame: a 7-day seizure diary completed at least twice within 3 months
Subjects or parents/guardians of subjects will record seizure activity in seizure diaries both prior to and during cannabidiol administration
1 secondary outcome
Cannabidiol concentration
Time frame: 1 time at a clinic visit within three months. If a patient has a port, they will be asked to provide an additional blood sample of 5 ml at least one hour after the first sample during the same clinic visit.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Dec 2021.
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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.
Samuel A Roiko, Ph.D. principal investigator
Gillette Children's Specialty Healthcare
Questions about this trial
What is this clinical trial studying?
An observational study of Dravet Syndrome and Lennox Gastaut Syndrome, run by Gillette Children's Specialty Healthcare.
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 3 inclusion and 1 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: withdrawn, as of the sponsor's last update on 16 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
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.
What does the primary outcome measure?
Reduction in seizures (time frame: a 7-day seizure diary completed at least twice within 3 months). It is what the study was designed to measure, not a finding.
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 16 Dec 2021.