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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.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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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
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

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

Other trials in United States

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 groups in NCT05044819
Study groupRoleWhat they receive
CannabidiolExperimentalCannabidiol 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.

  1. 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)

  1. Change From Baseline in Aspartate Aminotransferase (AST) to Platelet Ratio Index

    Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825

  2. Change from Baseline in the Enhanced Liver Fibrosis Score

    Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825

  3. Change From Baseline in Fibrosis-4

    Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825

  4. Change From Baseline in FibroScan Scoring (FibroScan subset only)

    Measured: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825

  5. Number of Participants With Potential Drug-Induced Liver Injury (DILI)

    Measured: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825

  6. Number of Participants with Severe Treatment-emergent Adverse Events (TEAEs)

    Measured: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825

  7. Number of Participants With Clinically Significant Clinical Laboratory Findings

    Measured: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825

  8. 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.

  1. First posted16 Sep 2021
  2. Study start7 Jul 2021
  3. Primary completion1 Aug 2027 (estimated)
  4. Study completion31 Mar 2028 (estimated)

Resources

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)
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

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