Trial Title:
A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex
NCT ID:
NCT05864846
Condition:
Tuberous Sclerosis Complex Associated Neuropsychiatric Disease
Conditions: Official terms:
Tuberous Sclerosis
Sclerosis
Cannabidiol
Conditions: Keywords:
Seizures
Tuberous Sclerosis Complex (TSC)
Tuberous Sclerosis Complex Associated Neuropsychiatric Disorders (TAND)
Epidiolex
Epidyolex
GWP42003-P
JZP926
TSC Associated Neuropsychiatric Disorders
Cannabidiol
Behavior
Study type:
Interventional
Study phase:
Phase 4
Overall status:
Recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
Cannabidiol Oral Solution [Epidiolex]
Description:
100 mg/ml Cannabidiol Oral solution
Arm group label:
Cannabidiol Oral Solution
Other name:
Epidiolex
Summary:
The purpose of this study is to investigate behavioral and other co-occurring outcomes
with EPID(I/Y)OLEX as an add-on therapy in participants aged 1 to 65 years with tuberous
sclerosis complex (TSC) who experience seizures.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
1. Is within the required age range at the time of signing (or at the time of the
participant's parent(s)/Legally Authorized Representative (LAR) signing) the
informed consent or providing assent (as applicable):
- Participants based in the US: 1 to 65 years of age, inclusive.
- Participants based outside the US: 2 to 65 years of age, inclusive.
2. Has a confirmed clinical diagnosis of TSC with a history of seizures in accordance
with the 2012 International Tuberous Sclerosis Complex Consensus Conference
criteria.
3. Has behaviors (eg, aggression, impulsivity, temper tantrum, self-injury,
hyperactivity, extreme shyness, mood swings, poor eye contact, repetitive behaviors,
restlessness, difficulty getting along with peers, rigid/inflexible to procedure
and/or change) that are considered moderate or severe per the CareGI-S at Screening.
4. Is taking 1 or more anti-seizure medicine (ASM) at a dose that has been stable for
at least 4 weeks prior to Screening.
• All medications or interventions for epilepsy (including ketogenic diet and any
neurostimulation devices for epilepsy) must have been stable for 4 weeks prior to
screening and any major changes to treatment regimens should be discussed with the
medical monitor.
5. Is naïve to CBD-OS treatment or has been off CBD-OS treatment for at least 3 months
prior to Screening.
6. Is willing to maintain any factors expected to affect seizures stable (eg, alcohol
consumption, smoking, concomitant medication usage).
7. Is male or female
1. Male participants:
• Male participants are eligible to participate if they agree to the following
during the intervention period and for at least 2 weeks, corresponding to the
time needed to eliminate the study intervention after the last dose of study
intervention:
- Refrain from donating fresh unwashed semen. PLUS
- Use a male condom in addition to a second method of acceptable
contraception used by their female partners when having sexual intercourse
with a women of childbearing potential (WOCBP) who is not currently
pregnant.
2. Female participants:
- A female participant is eligible to participate if she is not pregnant or
breastfeeding and one of the following conditions applies:
- Is a woman of nonchildbearing potential. OR
- Is a WOCBP and using a contraceptive method that is highly effective,
preferably with low user dependency during the study intervention
period and for at least 3 months after the last dose of study
intervention. The investigator should evaluate the potential for
contraceptive method failure (eg, noncompliance, recently initiated)
in relationship to the first dose of study intervention.
- A WOCBP must have a negative highly sensitive pregnancy test within 28
days before the first dose of study intervention.
Exclusion Criteria:
1. Has a clinically significant unstable medical condition other than epilepsy.
2. Has an illness during the 4 weeks prior to screening other than epilepsy which, in
the investigator's opinion, could affect study outcomes.
3. Has TSC-specific tumor growth which, in the investigator's opinion, could affect the
effectiveness endpoints.
4. Has previously undergone significant surgery for epilepsy that, in the
investigator's opinion, may impact the assessment of outcomes.
5. Has initiated felbamate within the last 12 months prior to Screening.
6. Is currently using or has in the past used recreational or medicinal cannabis or
synthetic cannabinoid-based medications within the 3 months prior to Screening and
is not willing to undergo a 1-month washout period before being rescreened.
7. Has received an investigational medicinal product within the 3 months prior to the
Screening Visit.
8. Has previously been assigned study intervention for this study or is currently
enrolled in any other interventional study.
9. Has laboratory values at the Baseline Visit that are abnormal and of clinical
significance in the investigator's opinion.
10. Participant has significantly impaired hepatic function at the Baseline Visit.
11. Has any history of suicidal behavior or any suicidal ideation of type 4 or 5 as
evaluated with C-SSRS or Children's C-SSRS at the Screening Visit (for participants
≥ 4 years of age).
12. Has any known or suspected hypersensitivity to cannabinoids or any of the excipients
of CBD-OS.
13. Has a known or suspected history of alcohol or substance abuse.
Gender:
All
Minimum age:
1 Year
Maximum age:
65 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
University of California, Los Angeles (UCLA)
Address:
City:
Los Angeles
Zip:
90095
Country:
United States
Status:
Withdrawn
Facility:
Name:
Children's Hospital Colorado
Address:
City:
Aurora
Zip:
80045
Country:
United States
Status:
Recruiting
Facility:
Name:
University of Florida Health - Department of Neurology
Address:
City:
Gainesville
Zip:
32608
Country:
United States
Status:
Recruiting
Facility:
Name:
Nicklaus Children's Health, Miami
Address:
City:
Miami
Zip:
33155
Country:
United States
Status:
Recruiting
Facility:
Name:
Massachusetts General Hospital
Address:
City:
Boston
Zip:
02114
Country:
United States
Status:
Recruiting
Facility:
Name:
Minnesota Epilepsy Group
Address:
City:
Roseville
Zip:
55113
Country:
United States
Status:
Recruiting
Facility:
Name:
Duke University Hospital
Address:
City:
Durham
Zip:
27705
Country:
United States
Status:
Withdrawn
Facility:
Name:
Atrium Health Wake Forest Baptist Medical Center
Address:
City:
Winston-Salem
Zip:
27157
Country:
United States
Status:
Recruiting
Facility:
Name:
Cincinnati Children's Hospital Medical Center
Address:
City:
Cincinnati
Zip:
45229
Country:
United States
Status:
Recruiting
Facility:
Name:
Le Bonheur Children's Hospital
Address:
City:
Memphis
Zip:
38103
Country:
United States
Status:
Recruiting
Facility:
Name:
University of Texas Health Science Center at Houston - Clinical Research Unit
Address:
City:
Houston
Zip:
77030
Country:
United States
Status:
Recruiting
Facility:
Name:
University of Texas Health Science Center - San Antonio
Address:
City:
San Antonio
Zip:
78229
Country:
United States
Status:
Recruiting
Facility:
Name:
University of Virginia, Charlottesville
Address:
City:
Charlottesville
Zip:
22903
Country:
United States
Status:
Recruiting
Facility:
Name:
The Hospital for Sick Children
Address:
City:
Toronto
Zip:
M5G 1X8
Country:
Canada
Status:
Recruiting
Facility:
Name:
The Children's Memorial Health Institute
Address:
City:
Warszawa
Zip:
04-736
Country:
Poland
Status:
Not yet recruiting
Facility:
Name:
University Hospitals Bristol NHS Foundation Trust
Address:
City:
Bristol
Zip:
BS1 3NU
Country:
United Kingdom
Status:
Recruiting
Facility:
Name:
Sheffield Children's NHS Foundation Trust
Address:
City:
Sheffield
Zip:
2TH
Country:
United Kingdom
Status:
Recruiting
Start date:
June 29, 2023
Completion date:
January 1, 2026
Lead sponsor:
Agency:
Jazz Pharmaceuticals
Agency class:
Industry
Source:
Jazz Pharmaceuticals
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05864846
