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

Dravet Syndrome Clinical Trial

Official title:

A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05044819
• Other study ID #: GWEP19022
• Status: Active, not recruiting
• Phase: Phase 4
• Start date: July 7, 2021
• Est. completion date: March 31, 2028

Study information

• Verified date: June 2024
• Source: Jazz Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 154
• Est. completion date: March 31, 2028
• Est. primary completion date: August 1, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Dravet Syndrome
• Epilepsies, Myoclonic
• Lennox Gastaut Syndrome
• Syndrome
• Tuberous Sclerosis
• Tuberous Sclerosis Complex

Intervention

Drug:
• Cannabidiol
Oral Cannabidiol solution 100 mg/mL

Locations

Country	Name	City	State
United States	Clinical Trial Site	Augusta	Georgia
United States	Clinical Trial Site	Baltimore	Maryland
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Charleston	South Carolina
United States	Clinical Trial Site	Charlotte	North Carolina
United States	Clinical Trial Site	Cincinnati	Ohio
United States	Clinical Trial Site	Downey	California
United States	Clinical Trial Site	Durham	North Carolina
United States	Clinical Trial Site	Henrico	Virginia
United States	Clinical Trial Site	Houston	Texas
United States	Clinical Trial Site	Lexington	Kentucky
United States	Clinical Trial Site	Little Rock	Arkansas
United States	Clinical Trial Site	Long Beach	California
United States	Clinical Trial Site	Miami	Florida
United States	Clinical Trial Site	Miami	Florida
United States	Clinical Trial Site	Miami	Florida
United States	Clinical Trial Site	New Brunswick	New Jersey
United States	Clinical Trial Site	New York	New York
United States	Clinical Trial Site	Oklahoma City	Oklahoma
United States	Clinical Trial Site	Orlando	Florida
United States	Clinical Trial Site	Philadelphia	Pennsylvania
United States	Clinical Trial Site	Round Rock	Texas
United States	Clinical Trial Site	Sacramento	California
United States	Clinical Trial Site	Tampa	Florida
United States	Clinical Trial Site	Wichita	Kansas
United States	Clinical Trial Site	Winchester	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Jazz Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change From Baseline in Aspartate Aminotransferase (AST) to Platelet Ratio Index		Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary	Change from Baseline in the Enhanced Liver Fibrosis Score		Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary	Change From Baseline in Fibrosis-4		Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary	Change From Baseline in FibroScan Scoring (FibroScan subset only)		Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary	Number of Participants With Potential Drug-Induced Liver Injury (DILI)		Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary	Number of Participants with Severe Treatment-emergent Adverse Events (TEAEs)		Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary	Number of Participants With Clinically Significant Clinical Laboratory Findings		Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary	Number of Participants With Clinically Significant Physical Examinations		Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825