Lennox-Gastaut SyndromePhase 4EpiGOALNCT07728097
A Study to Investigate the Effect of Gradual Titration to Optimize Cannabidiol Treatment in Adults With LGS
A Phase 4 study testing Cannabidiol Oral Solution in people with Lennox-Gastaut Syndrome, run by Jazz Pharmaceuticals at 3 sites in United States.
Trial summary
This is a Phase 3b/Phase 4 study. The purpose of this study is to learn more about the efficacy of using CBD-OS as an add-on therapy for the treatment of LGS in adults. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Lennox-Gastaut Syndrome
- What is being tested?
- Cannabidiol Oral Solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 3 sitesUnited States
- Study dates
- 18 Nov 2026to 28 Dec 2028
- Phase
- Phase 4
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Has a confirmed clinical diagnosis of LGS with a history of seizures and actively experiencing seizures within 6 months of screening.
- Is naïve to CBD-OS treatment or has been off CBD-OS treatment for at least 3 months prior to screening.
- Is willing to maintain any factors expected to affect seizures (eg, alcohol consumption, smoking, concomitant medication usage) stable for the duration of the study.
- Is currently treated with at least 1 ASM, but no more than 3 ASMs, and is on a stable regimen for at least 4 weeks prior to screening.
- Adequate contraceptive precautions
- Participant has a primary caregiver/legally authorized representative (LAR) who is willing and able, in the investigator's opinion, to participate throughout the duration of the study and to comply with all study requirements
- Experienced at least 4 or more countable major motor seizures (isolated seizures or seizures occurring in a cluster) during the 5-week Baseline Period preceding treatment.
- Caregiver completed at least 80% of seizure diary entries during the Baseline Period with no more than 3 consecutive days of missing entries.
Who cannot join
- Has a significant psychiatric illness
- Has an illness during the 4 weeks prior to screening other than epilepsy which, in the investigator's opinion, could affect study outcomes or seizure frequency.
- Has history of SE in the 3 months prior to screening.
- Has any other significant disease other than epilepsy, which, in the investigator's opinion, may either put the participant, other participants, or site staff at risk because of participation in the study, may influence the result of the study, or may affect the participant's ability to take part in the study.
- Has previously undergone significant surgery for epilepsy in the 6 months prior to screening or planned to undergo surgery for epilepsy during the study.
- Is currently using or has in the past used recreational or medicinal cannabis or synthetic cannabinoid-based medications, products, or supplements within the 3 months prior to screening and is not willing to undergo a 1-month washout period before being rescreened.
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- Has initiated felbamate within the 12 months prior to screening.
- Is not willing to adjust dosage of CBD-OS if needed when using strong inducers of CYP3A4 and/or strong inducers of CYP2C19 concomitantly with CBD-OS
- Previously discontinued cannabidiol due to severe adverse events (SAE), hypersensitivity or lack of efficacy
- Has clinically significant abnormal lab values
- Has clinically impaired hepatic or renal function
- History of, current risk or presence of actual suicidal ideations
- Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of CBD-OS, such as sesame oil.
- Has a known or suspected history of alcohol or substance abuse disorder
- Is currently consuming, and unwilling to stop consumption, or planning to consume tonic water on a regular basis throughout the study.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites
Neuro Pain Research Center
Fresno, California, 93722
Louisiana State University Health Sciences Center New Orleans
New Orleans, Louisiana, 70112
Velocity Clinical Research At Raleigh Neurology
Raleigh, North Carolina, 27607
About this study
Official title: A Phase 3b/4, Interventional, Prospective, Multicenter, Open-Label Study Evaluating the Effectiveness of a Gradual Titration Regimen to Optimize CBD-OS as Add-On Therapy in Adults With Lennox-Gastaut Syndrome
This is a Phase 3b/Phase 4 study. The purpose of this study is to learn more about the efficacy of using CBD-OS as an add-on therapy for the treatment of LGS in adults.
- Conditions
- Lennox-Gastaut Syndrome
- Being tested
- Cannabidiol Oral Solution
- Keywords
- LGS, Cannabidiol, CBD-OS, Epidiolex, Epidyolex
- Medical subject headings
- Lennox Gastaut Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cannabidiol Oral SolutionDrug
Also called JZP926, CBD-OS, Epidiolex, Epidyolex
100 mg/ml Cannabidiol Oral solution (CBD-OS)
| Study group | Role | What they receive |
|---|---|---|
| Cannabidiol Oral Solution Treatment group | Experimental | Participants who will receive the Cannabidiol Oral Solution (CBD-OS) titrated up to a dose of 20 mg/kg per day or the maximum tolerated dose for 24 consecutive weeks. Receives: Drug: Cannabidiol Oral Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of participants reporting 50% or more reduction in their 28-day average countable major motor seizure frequency
Measured: End of Treatment Period, Week 24
Secondary outcomes (25)
Retention Rate
Retention Rate is defined as percentage of participants completing or continuing CBD-OS treatment at different timepoints in the study
Measured: At Weeks 8, 12, 16 and 24
Percentage of participants experiencing worsening, change, no change or improvement in in their 28-day average countable major motor and total seizure frequency
As reported in participants' electronic diaries
Measured: Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Percentage of participants considered treatment responders
Treatment responders will be categorized as those experiencing a 50% or more reduction, a 75% or more reduction or 100% reduction in their 28-day average seizure frequency as reported in participants' electronic diaries
Measured: Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Change in 28-day average frequency of countable major motor and total seizures
Assessed per 28 days from electronic diary entries
Measured: Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Change in number of seizure-free days
Assessed per 28 days from electronic diary entries
Measured: Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of seizure scores
Caregiver Global Impression of Severity (CaGI-S) is a single-item, 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe"). Using this scale, caregivers will be asked to rate their impression of the participant's severity of seizures, and the severity of the impact caused by the seizure.
Measured: Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of impact of seizures scores
Caregiver Global Impression of Severity (CaGI-S) is a single-item, 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe"). Using this scale, caregivers will be asked to rate their impression of the participant's severity of the impact caused by the seizure.
Measured: Baseline to Week 16, Baseline to Week 24
Change in VAS scores
Visual Analog Scale (VAS) is a caregiver-reported tool used to quantify the severity of impact of subjective non-seizure symptoms and assesses sleep, communication, and disruptive behavior. The VAS enables sensitive detection of changes in non-seizure domains (disruptive behavior, Sleep, and Communication) important to patients with LGS and their caregivers. The VAS has a total score ranging from 0 to 100 with the higher score indicating worse conditions.
Measured: Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of level of alertness scores
CaGI-S Alertness is a caregiver single item assessment on a 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe") assessing their impression of participant's severity of level of awareness
Measured: Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of overall condition scores
CaGI-S Overall Condition is a caregiver single item assessment on a 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe") assessing their impression of participant's severity of overall condition
Measured: Baseline to Week 16, Baseline to Week 24
CaGI-C level of alertness scores
Caregiver Global Impression of Change (CaGI-C) Alertness is a caregiver single item assessment on a 7-point Likert-type rating scale (ranging from 1 "very much improved" to 7 "very severe") assessing their impression of participant's severity of level of awareness
Measured: At Week 16, at week 24
CaGI-C overall condition scores
Caregiver Global Impression of Change (CaGI-C) overall condition is a caregiver single item assessment on a 7-point Likert-type rating scale (ranging from 1 "very much improved" to 7 "very severe") assessing their impression of participant's severity of overall condition
Measured: At Week 16, at week 24
Change in CGI-S overall condition scores
Clinician Global Impression of severity (CGI-S) Overall Condition is a clinician single item assessment on a 5-point Likert-type rating scale (ranging from 1 "not severe at all" to 5 "very severe") assessing their impression of participant's severity of level of overall condition
Measured: Baseline to Week 16, Baseline to Week 24
CGI-C overall condition scores
Clinician Global Impression of Change (CGI-C) Overall Condition is a clinician single item assessment on a 7-point Likert-type rating scale (ranging from 1 "very much improved" to 5 "very much worse") assessing their impression of participant's change of overall condition
Measured: At Week 16, At Week 24
Change in ELDQOL Behavior subscale scores
Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL behavior subscale includes 9 of the 28 total main questions with a total score ranging from 9 to 36 with the higher score indicating greater behavioral problems
Measured: Baseline to Week 16, Baseline to Week 24
Change in ELDQOL seizure severity subscale scores
Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL seizure severity subscale includes 14 of the 28 total main questions with a total score ranging from 10 to 56 with the higher score indicating greater seizure severity
Measured: Baseline to Week 16, Baseline to Week 24
Change in ELDQOL mood scale subscale scores
Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL mood scale subscale includes 1 of the 28 total questions with a total score ranging from 16 to 64 with the higher score indicating greater mood problems
Measured: Baseline to Week 16, Baseline to Week 24
Change in ELDQOL side-effects profile subscale scores
Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL side-effects profile subscale includes 2 of the 28 total main questions with a score ranging from 19 to 76 with the higher score indicating greater side effects
Measured: Baseline to Week 16, Baseline to Week 24
Number of participants reporting adverse events leading to discontinuation
Measured: Up to week 24
Time the adverse events started from baseline
Measured: Up to week 24
Number of Participants with Abnormal Laboratory Values
Measured: Up to week 24
Change in suicidal ideation scores
As evaluated by the Columbia-Suicide Severity Rating Scale (C-SSRS). The questionnaire has a total score ranging from 0 to 5 with a score of 0 indicating no suicidal ideation is present.
Measured: Baseline, up to week 24
Change in number of suicide attempts
As per the response in the C-SSRS
Measured: Baseline, up to week 24
Percentage of participants requiring inpatient hospitalization due to epilepsy
Measured: Up to week 24
Change in number of usage of rescue medication
Measured: Baseline, Up to week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2026
- Study start18 Nov 2026 (estimated)
- Primary completion28 Dec 2028 (estimated)
- Study completion28 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lennox-Gastaut SyndromeStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Jazz Pharmaceuticals Research UK Limited
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07728097
- Sponsor's protocol ID
- JZP926-403
- Acronym
- EpiGOAL
- Status last verified
- Oct 2026
- First submitted
- 22 Jul 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with ICMJE requirements, Jazz Pharmaceuticals may provide qualified external researchers access to individual participant data (IPD) and clinical trial data that underlie the results of this trial upon request. Qualified researchers can submit a request on https://www.jazzpharma.com/science/clinical-trial-data-sharing/ as outlined. Jazz Pharmaceuticals reserves the right not to consider a request. For inquiries about Jazz's data sharing policy contact clinicaldatasharing@jazzpharma.com.