Lennox Gastaut SyndromePhase 4NCT05044819
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
A Phase 4 study testing Cannabidiol in people with Lennox Gastaut Syndrome and Dravet Syndrome, run by Jazz Pharmaceuticals at 26 sites in United States.
Trial summary
This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- What is being tested?
- Cannabidiol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 154 participants
- Where
- 26 sitesUnited States
- Study dates
- 7 Jul 2021to 31 Mar 2028
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participant is within the approved age range as per labeling recommendations. Participants aged 1 to 3 years who are in the FibroScan subset will not undergo FibroScan assessments until they reach 4 years of age.
- Participant is either on existing Epidiolex therapy for treatment of a seizure disorder or is to be started on Epidiolex for treatment of an Food and Drug Administration (FDA)-approved indication.
- Participant is willing to refrain from strenuous exercise 48 to 72 hours prior to all study visits with the exception of unscheduled visits.
- Any non-pharmacological therapies (e.g., ketogenic diet) must also be stable up to 4 weeks prior to Screening Visit and expected to be stable throughout the duration of the study.
Who cannot join
- Participant is currently using or within 3 months of screening has used recreational or medicinal cannabis, or synthetic cannabinoid-based medications, not including Epidiolex if currently prescribed.
- Participant is not planning to abstain from using recreational cannabinoids or medicinal cannabis, or synthetic cannabinoid-based medications during the study.
- Female participant is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the study and for 3 months thereafter.
- Participant has any other significant disease or disorder which, in the opinion of the investigator, 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
- Participant has diseases or disorders which are associated with liver fibrosis with a FibroScan score of ≥ 6.5 Kilopascals (where FibroScan results are available).
- Positive serology panel (including hepatitis B surface antigen and hepatitis C virus antibody) and/or positive human immunodeficiency virus antibody/p24 antigen screens at screening.
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- Following a physical examination, if the participant has any abnormalities that, in the opinion of the investigator, would prevent the participant from safe participation in the study.
- Participant has significantly impaired hepatic function at Screening Visit alanine aminotransferase or aminotransferase > 3 x upper limit of normal (ULN), and total bilirubin > 2 x ULN or international normalized ratio > 1.5.
- Participant is planning to have epilepsy surgery or other major surgery within five years.
- Participant has or plans to have any medical device implanted that is contraindicated for use with FibroScan (only applicable for FibroScan subset), with the investigator consulting with the Sponsor as needed.
- Participation in any clinical trial involving an investigational medicinal product within 3 months prior to the Screening Visit or at any point during this study.
Trial locations and contacts
26 sites in 1 country.
26 sites in 1 country
United States26 sites
Clinical Trial Site
Augusta, Georgia, 30912
Clinical Trial Site
Austin, Texas, 78758
Clinical Trial Site
Baltimore, Maryland, 21205
Clinical Trial Site
Boston, Massachusetts, 02114
Clinical Trial Site
Charleston, South Carolina, 29425
Clinical Trial Site
Charlotte, North Carolina, 28203
Clinical Trial Site
Cincinnati, Ohio, 45219
Clinical Trial Site
Dallas, Texas, 75251
Clinical Trial Site
Downey, California, 90242
Clinical Trial Site
Durham, North Carolina, 27708
Clinical Trial Site
Houston, Texas, 77030
Clinical Trial Site
Lexington, Kentucky, 40504
Clinical Trial Site
Little Rock, Arkansas, 72758
Clinical Trial Site
Long Beach, California, 90806
Clinical Trial Site
Miami, Florida, 33136
Clinical Trial Site
Miami, Florida, 33176
Clinical Trial Site
New Brunswick, New Jersey, 08901
Clinical Trial Site
New York, New York, 10016
Clinical Trial Site
Oklahoma City, Oklahoma, 73104
Clinical Trial Site
Orlando, Florida, 32806
Clinical Trial Site
Philadelphia, Pennsylvania, 19107
Clinical Trial Site
Round Rock, Texas, 78681
Clinical Trial Site
Sacramento, California, 95817
Clinical Trial Site
Tampa, Florida, 33606
Clinical Trial Site
Wichita, Kansas, 67214
Clinical Trial Site
Winchester, Virginia, 22601
About this study
Official title: A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.
- Being tested
- Cannabidiol
- Keywords
- Liver injury, Liver fibrosis, Epidiolex, Cannabidiol
- Medical subject headings
- Lennox Gastaut Syndrome, Epilepsies, Myoclonic, Tuberous Sclerosis, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CannabidiolDrug
Also called Epidiolex
Oral Cannabidiol solution 100 mg/mL
| Study group | Role | What they receive |
|---|---|---|
| Cannabidiol | Experimental | Cannabidiol solution 100 milligrams per milliliter (mg/mL) will be administered orally at a dose level of 5 mg/kg/day for 1 week, then increase to 10 mg/kg/day by the participant or their caregiver twice each day (morning and evening). Receives: Drug: Cannabidiol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Liver Fibrosis and Evaluable Fibrotic Changes as Determined and Assessed by an Independent Adjudication Committee
Measured: Screening, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary outcomes (8)
Change From Baseline in Aspartate Aminotransferase (AST) to Platelet Ratio Index
Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Change from Baseline in the Enhanced Liver Fibrosis Score
Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Change From Baseline in Fibrosis-4
Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Change From Baseline in FibroScan Scoring (FibroScan subset only)
Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants With Potential Drug-Induced Liver Injury (DILI)
Measured: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants with Severe Treatment-emergent Adverse Events (TEAEs)
Measured: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants With Clinically Significant Clinical Laboratory Findings
Measured: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants With Clinically Significant Physical Examinations
Measured: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2021
- Study start7 Jul 2021
- Primary completion1 Aug 2027 (estimated)
- Study completion31 Mar 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
- 154 participants (actual)
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
- NCT05044819
- Sponsor's protocol ID
- GWEP19022
- Status last verified
- Aug 2026
- First submitted
- 30 Aug 2021
- Last updated
- 26 Aug 2026
- Results posted
- No
- 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 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
- ClinicalTrials.gov ID
- NCT05044819
- Acronym / sponsor ID
- GWEP19022
- Keywords
- Liver injury, Liver fibrosis, Epidiolex, Cannabidiol
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 154 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Jazz Pharmaceuticals Research UK Limited
- Study start
- 7 Jul 2021 (actual)
- Primary completion
- 1 Aug 2027 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 16 Sep 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 study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cannabidiol | Experimental | Drug: CannabidiolCannabidiol solution 100 milligrams per milliliter (mg/mL) will be administered orally at a dose level of 5 mg/kg/day for 1 week, then increase to 10 mg/kg/day by the participant or their caregiver twice each day (morning and evening). |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Participant is within the approved age range as per labeling recommendations. Participants aged 1 to 3 years who are in the FibroScan subset will not undergo FibroScan assessments until they reach 4 years of age.
- Participant is either on existing Epidiolex therapy for treatment of a seizure disorder or is to be started on Epidiolex for treatment of an Food and Drug Administration (FDA)-approved indication.
- Participant is willing to refrain from strenuous exercise 48 to 72 hours prior to all study visits with the exception of unscheduled visits.
- Any non-pharmacological therapies (e.g., ketogenic diet) must also be stable up to 4 weeks prior to Screening Visit and expected to be stable throughout the duration of the study.
Key exclusion criteria (11 listed)
- Participant is currently using or within 3 months of screening has used recreational or medicinal cannabis, or synthetic cannabinoid-based medications, not including Epidiolex if currently prescribed.
- Participant is not planning to abstain from using recreational cannabinoids or medicinal cannabis, or synthetic cannabinoid-based medications during the study.
- Female participant is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the study and for 3 months thereafter.
- Participant has any other significant disease or disorder which, in the opinion of the investigator, 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
- Participant has diseases or disorders which are associated with liver fibrosis with a FibroScan score of ≥ 6.5 Kilopascals (where FibroScan results are available).
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: EpidiolexOral Cannabidiol solution 100 mg/mL | Drug | Cannabidiol |
Primary outcome
Number of Participants With Liver Fibrosis and Evaluable Fibrotic Changes as Determined and Assessed by an Independent Adjudication Committee
Time frame: Screening, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
8 secondary outcomes
Change From Baseline in Aspartate Aminotransferase (AST) to Platelet Ratio Index
Time frame: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Change from Baseline in the Enhanced Liver Fibrosis Score
Time frame: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Change From Baseline in Fibrosis-4
Time frame: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Change From Baseline in FibroScan Scoring (FibroScan subset only)
Time frame: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants With Potential Drug-Induced Liver Injury (DILI)
Time frame: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants with Severe Treatment-emergent Adverse Events (TEAEs)
Time frame: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants With Clinically Significant Clinical Laboratory Findings
Time frame: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Number of Participants With Clinically Significant Physical Examinations
Time frame: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- Individual participant dataSharing planned: No
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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.
26 sites 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 in people with Lennox Gastaut Syndrome and Dravet Syndrome, run by Jazz Pharmaceuticals at 26 sites 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 4 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: under way, not looking for participants, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites 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?
This study is still under way. Results are not available in the source record yet.
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?
Number of Participants With Liver Fibrosis and Evaluable Fibrotic Changes as Determined and Assessed by an Independent Adjudication Committee (time frame: Screening, Day 365, Day 730, Day 1095, Day 1460, and Day 1825). 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 26 Aug 2026.