Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

NCT05044819 | Active Not Recruiting Phase 4 FDA Drug

• 1 other identifier: GWEP19022
• Study Type: interventional
• Target: 154
• Locations: 1 country
• Sites: 26

Brief Summary

This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.

Conditions

Lennox Gastaut Syndrome; Dravet Syndrome; Tuberous Sclerosis Complex

Keywords

Liver injury; Liver fibrosis; Epidiolex; Cannabidiol

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Liver Fibrosis and Evaluable Fibrotic Changes as Determined and Assessed by an Independent Adjudication Committee

  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

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

• Change from Baseline in the Enhanced Liver Fibrosis Score

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

• Change From Baseline in Fibrosis-4

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

• Change From Baseline in FibroScan Scoring (FibroScan subset only)

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

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

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

Study Arms (1)

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).

Drug: Cannabidiol

Interventions

Cannabidiol DRUG

Oral Cannabidiol solution 100 mg/mL

Also known as: Epidiolex

Cannabidiol

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead
• Jazz Pharmaceuticals Research UK Limited: collaborator

Study Sites (26)

Clinical Trial Site

Little Rock, Arkansas, 72758, United States

Location

Clinical Trial Site

Downey, California, 90242, United States

Location

Clinical Trial Site

Long Beach, California, 90806, United States

Location

Clinical Trial Site

Sacramento, California, 95817, United States

Location

Clinical Trial Site

Miami, Florida, 33136, United States

Location

Clinical Trial Site

Miami, Florida, 33176, United States

Location

Clinical Trial Site

Orlando, Florida, 32806, United States

Location

Clinical Trial Site

Tampa, Florida, 33606, United States

Location

Clinical Trial Site

Augusta, Georgia, 30912, United States

Location

Clinical Trial Site

Wichita, Kansas, 67214, United States

Location

Clinical Trial Site

Lexington, Kentucky, 40504, United States

Location

Clinical Trial Site

Baltimore, Maryland, 21205, United States

Location

Clinical Trial Site

Boston, Massachusetts, 02114, United States

Location

Clinical Trial Site

New Brunswick, New Jersey, 08901, United States

Location

Clinical Trial Site

New York, New York, 10016, United States

Location

Clinical Trial Site

Charlotte, North Carolina, 28203, United States

Location

Clinical Trial Site

Durham, North Carolina, 27708, United States

Location

Clinical Trial Site

Cincinnati, Ohio, 45219, United States

Location

Clinical Trial Site

Oklahoma City, Oklahoma, 73104, United States

Location

Clinical Trial Site

Philadelphia, Pennsylvania, 19107, United States

Location

Clinical Trial Site

Charleston, South Carolina, 29425, United States

Location

Clinical Trial Site

Austin, Texas, 78758, United States

Location

Clinical Trial Site

Dallas, Texas, 75251, United States

Location

Clinical Trial Site

Houston, Texas, 77030, United States

Location

Clinical Trial Site

Round Rock, Texas, 78681, United States

Location

Clinical Trial Site

Winchester, Virginia, 22601, United States

Location

MeSH Terms

Conditions

Lennox Gastaut Syndrome; Epilepsies, Myoclonic; Tuberous Sclerosis; Liver Cirrhosis

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Epileptic Syndromes; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Epilepsy, Generalized; Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Liver Diseases; Digestive System Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2021
• First Posted: September 16, 2021
• Study Start: July 7, 2021
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): March 31, 2028
• Last Updated: April 22, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (26)