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

Sponsor

Jazz Pharmaceuticals (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05044819

Collaborator

(none)

150

Enrollment

Locations

Arm

80.8

Anticipated Duration (Months)

4.8

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Lennox Gastaut Syndrome Dravet Syndrome Tuberous Sclerosis Complex	Drug: Cannabidiol	Phase 4

Study Design

Study Type:

Interventional

Anticipated Enrollment :

150 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Jul 7, 2021

Anticipated Primary Completion Date :

Aug 1, 2027

Anticipated Study Completion Date :

Mar 31, 2028

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cannabidiol 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 Oral Cannabidiol solution 100 mg/mL Other Names: Epidiolex

Arm

Intervention/Treatment

Experimental: Cannabidiol

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

Oral Cannabidiol solution 100 mg/mL

Other Names:

Epidiolex

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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 [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]
Number of Participants with Severe Treatment-emergent Adverse Events [Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825]
Number of Participants With Clinically Significant Clinical Laboratory Findings [Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825]
Number of Participants With Clinically Significant Physical Examinations [Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825]

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Participant is within the approved age range as per labeling recommendations.
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 known severe liver fibrosis with a FibroScan score of ≥ 6.5 Kilopascals.
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, 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Trial Site	Downey	California	United States	90242
2	Clinical Trial Site	Long Beach	California	United States	90806
3	Clinical Trial Site	Los Angeles	California	United States	90095-1752
4	Clinical Trial Site	Sacramento	California	United States	95817
5	Clinical Trial Site	Loxahatchee Groves	Florida	United States	33470
6	Clinical Trial Site	Miami	Florida	United States	33136
7	Clinical Trial Site	Miami	Florida	United States	33155
8	Clinical Trial Site	Miami	Florida	United States	33176
9	Clinical Trial Site	Orlando	Florida	United States	32806
10	Clinical Trial Site	Tampa	Florida	United States	33606
11	Clinical Trial Site	Atlanta	Georgia	United States	30329
12	Clinical Trial Site	Augusta	Georgia	United States	30912
13	Clinical Trial Site	Lexington	Kentucky	United States	40504
14	Clinical Trial Site	Baltimore	Maryland	United States	21205
15	Clinical Trial Site	Boston	Massachusetts	United States	02114-2743
16	Clinical Trial Site	New Brunswick	New Jersey	United States	08901
17	Clinical Trial Site	New York	New York	United States	10016
18	Clinical Trial Site	Asheville	North Carolina	United States	28806
19	Clinical Trial Site	Charlotte	North Carolina	United States	28203
20	Clinical Trial Site	Durham	North Carolina	United States	27708
21	Clinical Trial Site	Cincinnati	Ohio	United States	45219
22	Clinical Trial Site	Oklahoma City	Oklahoma	United States	73104
23	Clinical Trial Site	Philadelphia	Pennsylvania	United States	19107
24	Clinical Trial Site	Charleston	South Carolina	United States	29425
25	Clinical Trial Site	Austin	Texas	United States	78758
26	Clinical Trial Site	Dallas	Texas	United States	75251
27	Clinical Trial Site	Houston	Texas	United States	77030-3000
28	Clinical Trial Site	Round Rock	Texas	United States	78681
29	Clinical Trial Site	Henrico	Virginia	United States	23226
30	Clinical Trial Site	Winchester	Virginia	United States	22601
31	Clinical Trial Site	Crab Orchard	West Virginia	United States	25827