Study the Efficacy of Epidiolex for Typical Absence SeizuresPhase 4NCT04899050
Epidiolex in Typical Absence Seizures
A Phase 4 study testing Cannabidiol (Epidiolex) in people with Study the Efficacy of Epidiolex for Typical Absence Seizures, run by University of South Florida at 1 site in United States.
Trial summary
This is a Pilot study, open-label study consisting of a screening period of up to 4 weeks, a 4-week dose-titration treatment period to dose of up to 20 mg/kg/day BID of CBD (Epidiolex), and a 30 day safety follow-up period following the last dose of study medication. Read more about this study
- Who is it for?
- Ages 6 years and olderHealthy volunteers can take part
- Conditions the study is for
- Study the Efficacy of Epidiolex for Typical Absence Seizures
- What is being tested?
- Cannabidiol (Epidiolex)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jul 2021to 27 May 2023
- Phase
- Phase 4
- 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 6 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Participants are eligible to be included in the study only if all of the following criteria apply:
- Participant is willing to sign the informed consent form (ICF) indicating that the subject has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side effects, risks, and discomforts.
- Participant is male or female 6 years of age and older, with diagnosis of uncontrolled Genetic (Idiopathic) Generalized Epilepsy with Typical Absence Seizures.
- Clinical diagnosis of GGE (including, but not limited to, CAE, JAE, juvenile myoclonic epilepsy, or Jeavons syndrome) with absence seizures consistent with the International League against Epilepsy Revised Classification of Seizures (2017).
- Absence seizures, by history on average once per hour, persisting despite standard of care (SOC) treatment, defined as treatment with at least 2 AEDs appropriate for the patient's epilepsy syndrome. SOC failure, per investigator discretion, will be defined as insufficient clinical response or intolerable side effects, which precludes use of the appropriate AED.
- Observation of at least 3 instances of generalized discharges of approximately 2.5 - 4 Hz lasting >2 seconds via 24-hr ambulatory EEG.
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- On stable doses of one or more antiepileptic medication(s) for at least 30 days. If a subject is not on medication, adequate documentation justifying lack of therapy may be acceptable for the subject after Greenwich Biosciences review. Ketogenic, modified Atkins diet (MAD), or low glycemic diet with stable carbohydrate ratio for at least 30 days before screening is an acceptable antiepileptic therapy. Vagal nerve stimulation at stable settings (for at least 30 days before screening), without use of the magnet, is also acceptable.
- Subjects with reproductive capability, including all males and WOCBP, must agree to practice continuous abstinence or adequate contraception methods (appropriate double barrier method or oral, patch, implant, or injectable contraception) from as soon as feasible during screening period until at least 30 days after the last dose (i.e., intermittent abstinence based on "rhythm," temperature monitoring, or other means of timing is not acceptable). WOCBP include any woman who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, and/or bilateral oophorectomy) or is not postmenopausal. Postmenopausal is defined as amenorrhea for ≥12 consecutive months without another cause, and a documented serum follicle stimulating hormone (FSH) level ≥35 mIU/mL.
- Participants who are able and willing to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
Who cannot join
- Participants will be excluded from the study if any of the following conditions apply:
- Participant who has history of surgical intervention for treatment of epilepsy.
- Additional seizure (clinical and electrographic) types, including, but not limited to, epileptic spasms, generalized tonic seizures, atonic seizures, or focal seizures. Subjects with GTCS or myoclonic seizures are eligible for the study.
- Participants with inadequately treated psychotic or mood disorder (e.g., schizophrenia, major depression, bipolar disorder).
- Presence of severe intellectual disability, severe autism spectrum disorder, or severe developmental disorder such that the subject cannot sign the ICF or cannot cooperate with the study procedures.
- Presence of positive urine drug screen for drugs of abuse, except if this is explained by use of an allowed prescription medicine.
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- Significant hepatic (AST/ALT or bilirubin ≥2 × ULN) or renal disease (creatinine clearance ≤39 mL/min).
- A current C-SSRS score of 4 or 5 at screening or history of suicide attempt within the past year.
- Participant has any medical condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study.
- Treatment with an investigational agent within 30 days prior to the first dose of EPIDIOLEX or planning to receive an investigational agent during the study.
- Concurrent legal use of CBD obtained from a state dispensary.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety and Efficacy of CBD (Epidiolex) in Idiopathic Generalized Epilepsy With Typical Absence Seizures
This is a Pilot study, open-label study consisting of a screening period of up to 4 weeks, a 4-week dose-titration treatment period to dose of up to 20 mg/kg/day BID of CBD (Epidiolex), and a 30 day safety follow-up period following the last dose of study medication.
- Being tested
- Cannabidiol (Epidiolex)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cannabidiol (Epidiolex)Drug
Use of Epidiolex to control absence seizures.
| Study group | Role | What they receive |
|---|---|---|
| Absence Seizures Treated with Epidiolex | Experimental | This is a pilot, open-label study consisting of a screening period of up to 28 Days, a 4-week dose-titration treatment period to dose of up to 20 mg/kg/day BID of CBD (EPIDIOLEX ), and a 30-day safety follow-up period following the last dose of study medication. Receives: Drug: Cannabidiol (Epidiolex) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Amount of epileptiform activity.
The EEG epileptiform activity will be measured in seconds per 24 hours period.
Measured: From baseline EEG to the EEG after 90 days of treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2021
- Study start30 Jul 2021
- Primary completion27 May 2023
- Study completion27 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Study the Efficacy of Epidiolex for Typical Absence SeizuresStudies 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Principal investigator, Selim Benbadis, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04899050
- Sponsor's protocol ID
- STUDY001072
- Status last verified
- Jul 2023
- First submitted
- 19 May 2021
- Last updated
- 11 Jul 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of CBD (Epidiolex) in Idiopathic Generalized Epilepsy With Typical Absence Seizures
- ClinicalTrials.gov ID
- NCT04899050
- Acronym / sponsor ID
- STUDY001072
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 30 Jul 2021 (actual)
- Primary completion
- 27 May 2023 (actual)
- Study completion
- 27 May 2023 (actual)
- First posted
- 24 May 2021 (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 Pilot study, open-label study consisting of a screening period of up to 4 weeks, a 4-week dose-titration treatment period to dose of up to 20 mg/kg/day BID of CBD (Epidiolex), and a 30 day safety follow-up period following the last dose of study medication.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Absence Seizures Treated with Epidiolex | Experimental | Drug: Cannabidiol (Epidiolex)This is a pilot, open-label study consisting of a screening period of up to 28 Days, a 4-week dose-titration treatment period to dose of up to 20 mg/kg/day BID of CBD (EPIDIOLEX ), and a 30-day safety follow-up period following the last dose of study medication. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Participants are eligible to be included in the study only if all of the following criteria apply:
- Participant is willing to sign the informed consent form (ICF) indicating that the subject has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side effects, risks, and discomforts.
- Participant is male or female 6 years of age and older, with diagnosis of uncontrolled Genetic (Idiopathic) Generalized Epilepsy with Typical Absence Seizures.
- Clinical diagnosis of GGE (including, but not limited to, CAE, JAE, juvenile myoclonic epilepsy, or Jeavons syndrome) with absence seizures consistent with the International League against Epilepsy Revised Classification of Seizures (2017).
- Absence seizures, by history on average once per hour, persisting despite standard of care (SOC) treatment, defined as treatment with at least 2 AEDs appropriate for the patient's epilepsy syndrome. SOC failure, per investigator discretion, will be defined as insufficient clinical response or intolerable side effects, which precludes use of the appropriate AED.
Key exclusion criteria (11 listed)
- Participant who has history of surgical intervention for treatment of epilepsy.
- Additional seizure (clinical and electrographic) types, including, but not limited to, epileptic spasms, generalized tonic seizures, atonic seizures, or focal seizures. Subjects with GTCS or myoclonic seizures are eligible for the study.
- Participants with inadequately treated psychotic or mood disorder (e.g., schizophrenia, major depression, bipolar disorder).
- Presence of severe intellectual disability, severe autism spectrum disorder, or severe developmental disorder such that the subject cannot sign the ICF or cannot cooperate with the study procedures.
- Presence of positive urine drug screen for drugs of abuse, except if this is explained by use of an allowed prescription medicine.
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 (Epidiolex)Use of Epidiolex to control absence seizures. | Drug | Absence Seizures Treated with Epidiolex |
Primary outcome
Amount of epileptiform activity.
Time frame: From baseline EEG to the EEG after 90 days of treatment.
The EEG epileptiform activity will be measured in seconds per 24 hours period.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2023.
- Individual participant dataSharing planned: No
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 Phase 4 study testing Cannabidiol (Epidiolex) in people with Study the Efficacy of Epidiolex for Typical Absence Seizures, run by University of South Florida 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 9 inclusion and 11 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: finished, as of the sponsor's last update on 11 Jul 2023. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Amount of epileptiform activity. (time frame: From baseline EEG to the EEG after 90 days of treatment.). 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 11 Jul 2023; AzTrials last refreshed its copy on 27 Oct 2026.