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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05044819
Recruitment Status : Recruiting
First Posted : September 16, 2021
Last Update Posted : May 10, 2023
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : July 7, 2021
Estimated Primary Completion Date : August 1, 2027
Estimated Study Completion Date : March 31, 2028

Resource links provided by the National Library of Medicine

Drug Information available for: Cannabidiol

Arms and Interventions
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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 Name: Epidiolex


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Change from Baseline in the Enhanced Liver Fibrosis Score [ Time Frame: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825 ]
  3. Change From Baseline in Fibrosis-4 [ Time Frame: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825 ]
  4. Change From Baseline in FibroScan Scoring [ Time Frame: Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825 ]
  5. 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 ]
  6. Number of Participants with Severe Treatment-emergent Adverse Events [ Time Frame: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825 ]
  7. Number of Participants With Clinically Significant Clinical Laboratory Findings [ Time Frame: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825 ]
  8. Number of Participants With Clinically Significant Physical Examinations [ Time Frame: Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825 ]

Eligibility Criteria
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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05044819


Contacts
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Contact: Medical Enquiries 1-833-424-6724 medinfo.USA@gwpharm.com

Locations
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United States, California
Clinical Trial Site Recruiting
Downey, California, United States, 90242
Clinical Trial Site Recruiting
Long Beach, California, United States, 90806
Clinical Trial Site Withdrawn
Los Angeles, California, United States, 90095-1752
Clinical Trial Site Recruiting
Sacramento, California, United States, 95817
United States, Florida
Clinical Trial Site Recruiting
Loxahatchee Groves, Florida, United States, 33470
Clinical Trial Site Recruiting
Miami, Florida, United States, 33136
Clinical Trial Site Recruiting
Miami, Florida, United States, 33155
Clinical Trial Site Recruiting
Miami, Florida, United States, 33176
Clinical Trial Site Recruiting
Orlando, Florida, United States, 32806
Clinical Trial Site Recruiting
Tampa, Florida, United States, 33606
United States, Georgia
Clinical Trial Site Withdrawn
Atlanta, Georgia, United States, 30329
Clinical Trial Site Recruiting
Augusta, Georgia, United States, 30912
United States, Kentucky
Clinical Trial Site Recruiting
Lexington, Kentucky, United States, 40504
United States, Maryland
Clinical Trial Site Recruiting
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Clinical Trial Site Not yet recruiting
Boston, Massachusetts, United States, 02114-2743
United States, New Jersey
Clinical Trial Site Recruiting
New Brunswick, New Jersey, United States, 08901
United States, New York
Clinical Trial Site Not yet recruiting
New York, New York, United States, 10016
United States, North Carolina
Clinical Trial Site Withdrawn
Asheville, North Carolina, United States, 28806
Clinical Trial Site Recruiting
Charlotte, North Carolina, United States, 28203
Clinical Trial Site Recruiting
Durham, North Carolina, United States, 27708
United States, Ohio
Clinical Trial Site Recruiting
Cincinnati, Ohio, United States, 45219
United States, Oklahoma
Clinical Trial Site Recruiting
Oklahoma City, Oklahoma, United States, 73104
United States, Pennsylvania
Clinical Trial Site Recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, South Carolina
Clinical Trial Site Recruiting
Charleston, South Carolina, United States, 29425
United States, Texas
Clinical Trial Site Recruiting
Austin, Texas, United States, 78758
Clinical Trial Site Recruiting
Dallas, Texas, United States, 75251
Clinical Trial Site Recruiting
Houston, Texas, United States, 77030-3000
Clinical Trial Site Recruiting
Round Rock, Texas, United States, 78681
United States, Virginia
Clinical Trial Site Recruiting
Henrico, Virginia, United States, 23226
Clinical Trial Site Recruiting
Winchester, Virginia, United States, 22601
United States, West Virginia
Clinical Trial Site Withdrawn
Crab Orchard, West Virginia, United States, 25827
Sponsors and Collaborators
Jazz Pharmaceuticals
More Information
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Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05044819    
Other Study ID Numbers: GWEP19022
First Posted: September 16, 2021    Key Record Dates
Last Update Posted: May 10, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Jazz Pharmaceuticals:
Liver injury
Liver fibrosis
Epidiolex
Cannabidiol
Additional relevant MeSH terms:
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Tuberous Sclerosis
Epilepsies, Myoclonic
Lennox Gastaut Syndrome
Syndrome
Disease
Pathologic Processes
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
 Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Epileptic Syndromes
Cannabidiol
Anticonvulsants