A study to investigate behavioral and other co-occurring outcomes with Epid(i/y)olex as add-on therapy in participants aged 1 to 65 years of age with tuberous sclerosis complex.

Phase 1

• Conditions: Tuberous Scerlosis Complex; MedDRA version: 21.0Level: PTClassification code: 10080584Term: Tuberous sclerosis complex Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: CTIS2023-507426-17-00
• Lead Sponsor: Jazz Pharmaceuticals Research UK Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-01-11
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

Is within the required age range at the time of signing (or at the time of the participant’s parent(s)/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., Participant’s parent(s)/ or LAR is willing to allow the responsible authorities to be notified of the participant’s involvement in the study, if mandated by local law., Participant’s parent(s)/LAR is willing to allow his or her primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the study, if the primary care practitioner/consultant is different to the investigator., Participant (including adults lacking capacity) has a dedicated caregiver to participate in the study., Has a confirmed clinical diagnosis of TSC with a history of seizures in accordance with the 2012 International Tuberous Sclerosis Complex Consensus Conference criteria., 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. • At Baseline, participants must have a most problematic behavior score of = 6 to remain eligible., 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. • At Baseline, participants must have a most problematic behavior score of = 6 to remain eligible., Is naïve to CBD-OS treatment or has been off CBD-OS treatment for at least 3 months prior to Screening., Is willing to maintain any factors expected to affect seizures stable (eg, alcohol consumption, smoking, concomitant medication usage)., Is male or female a. 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 (eg, 5 terminal half-lives) 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 WOCBP who is not currently pregnant. b. 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 (with a failure rate of < 1% per year), preferably with low user dependency, as described in (Appendix 4 Contraceptive and Barrier Guidance), 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., Participant or the participant’s parent(s)/LAR is capable of giving signed informed consent, which includes compl

Exclusion Criteria

Has a clinically significant unstable medical condition other than epilepsy., Has laboratory values at the Baseline Visit that are abnormal and of clinical significance in the investigator’s opinion., Participant has significantly impaired hepatic function at the Baseline Visit, defined as any of the following: a. Serum ALT or AST > 5 × ULN b. Serum ALT or AST > 3 × ULN and TBL > 2 × ULN or INR > 1.5 c. Serum ALT or AST > 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, jaundice, fever, rash, and/or eosinophilia (> 5%) Note: This criterion can only be confirmed once the laboratory results are available; participants enrolled into the study who are later found to meet this criterion must be withdrawn from the study., 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)., Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of CBD-OS., Has a known or suspected history of alcohol or substance abuse., Has an illness during the 4 weeks prior to screening other than epilepsy which, in the investigator’s opinion, could affect study outcomes., Has TSC-specific tumor growth which, in the investigator’s opinion, could affect the effectiveness endpoints., Has any other significant disease or disorder which, in the investigator’s opinion, 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., Has previously undergone significant surgery for epilepsy that, in the investigator’s opinion, may impact the assessment of outcomes., Has initiated felbamate within the last 12 months prior to Screening., 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., Has received an investigational medicinal product within the 3 months prior to the Screening Visit., Has previously been assigned study intervention for this study or is currently enrolled in any other interventional study (including behavioral intervention studies).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified