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

Brief Summary
This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.
Brief Title
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
Completion Date
Completion Date Type
Estimated
Conditions
Lennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Eligibility Criteria
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.
Inclusion Criteria
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.

Gender
All
Gender Based
false
Keywords
Liver injury
Liver fibrosis
Epidiolex
Cannabidiol
Healthy Volunteers
No
Last Update Submit Date
Minimum Age
1 Year
NCT Id
NCT05044819
Org Class
Industry
Org Full Name
Jazz Pharmaceuticals
Org Study Id
GWEP19022
Overall Status
Active, not recruiting
Phases
Phase 4
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
Primary Outcomes
Outcome Measure
Number of Participants With Liver Fibrosis and Evaluable Fibrotic Changes as Determined and Assessed by an Independent Adjudication Committee
Outcome Time Frame
Screening, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Secondary Outcomes
Outcome Time Frame
Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Change From Baseline in Aspartate Aminotransferase (AST) to Platelet Ratio Index
Outcome Time Frame
Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Change from Baseline in the Enhanced Liver Fibrosis Score
Outcome Time Frame
Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Change From Baseline in Fibrosis-4
Outcome Time Frame
Baseline, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Change From Baseline in FibroScan Scoring (FibroScan subset only)
Outcome Time Frame
Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Number of Participants With Potential Drug-Induced Liver Injury (DILI)
Outcome Time Frame
Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Number of Participants with Severe Treatment-emergent Adverse Events (TEAEs)
Outcome Time Frame
Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Number of Participants With Clinically Significant Clinical Laboratory Findings
Outcome Time Frame
Day 1, Day 365, Day 730, Day 1095, Day 1460, and Day 1825
Outcome Measure
Number of Participants With Clinically Significant Physical Examinations
Start Date
Start Date Type
Actual
Status Verified Date
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
1
Investigators
Investigator Type
Principal Investigator
Investigator Name
Puja Patel
Investigator Email
pupatel@montefiore.org
Investigator Phone

Categories Mesh Debug
Liver --- LIVER CIRRHOSIS
Alzheimer's --- BRAIN DISEASES
Brain, Spinal Cord & Nervous System --- BRAIN DISEASES
Brain, Spine & Nerve Cancers --- BRAIN DISEASES
Alzheimer's --- CENTRAL NERVOUS SYSTEM DISEASES
Brain, Spinal Cord & Nervous System --- CENTRAL NERVOUS SYSTEM DISEASES
Brain, Spine & Nerve Cancers --- CENTRAL NERVOUS SYSTEM DISEASES
Brain, Spinal Cord & Nervous System --- NERVOUS SYSTEM DISEASES
Brain, Spine & Nerve Cancers --- NERVOUS SYSTEM DISEASES
Cancer --- NEOPLASMS
Blood & Bone Marrow Cancers --- CONGENITAL ABNORMALITIES
Digestive System --- LIVER DISEASES
Liver --- LIVER DISEASES
Gastrointestinal (GI) Cancers --- DIGESTIVE SYSTEM DISEASES
Digestive System --- DIGESTIVE SYSTEM DISEASES
Liver --- DIGESTIVE SYSTEM DISEASES
MeSH Terms
LENNOX GASTAUT SYNDROME
EPILEPSIES, MYOCLONIC
TUBEROUS SCLEROSIS
LIVER CIRRHOSIS
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
CANNABIDIOL
CANNABINOIDS
TERPENES
HYDROCARBONS
ORGANIC CHEMICALS