Epidiolex for Tuberous Sclerosis Complex

(EpiCom Trial)

Recruiting in Palo Alto (17 mi)

+19 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Recruiting

Sponsor: Jazz Pharmaceuticals

Must be taking: Anti-seizure medicines

Must not be taking: Cannabis, Synthetic cannabinoids

Disqualifiers: Unstable medical condition, Tumor growth, others

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial investigates how adding EPIDIOLEX to existing treatments affects behavior and other outcomes in people aged 1 to 65 with TSC who have seizures. The medication aims to reduce seizure frequency and severity by influencing brain chemicals. Epidiolex, a cannabidiol (CBD) product, has been studied for its safety and effectiveness in treating seizures related to tuberous sclerosis complex (TSC).

Will I have to stop taking my current medications?

The trial requires that you continue taking your current anti-seizure medications at a stable dose for at least 4 weeks before joining. Any changes to your treatment should be discussed with the medical team.

Is Epidiolex (CBD) safe for humans?

Epidiolex (CBD) has been studied for safety in people with epilepsy and other conditions. Some studies suggest that certain side effects might limit its use, but it is generally considered safe for human use when prescribed by a doctor. ^{1 2 3 4 5}

Research Team

Eligibility Criteria

This trial is for individuals aged 1 to 65 with tuberous sclerosis complex (TSC) who have seizures. Participants must be on stable anti-seizure medication, not have used CBD-OS recently, and agree to consistent contraceptive use if applicable. They cannot join if they've had certain medical conditions or treatments that could affect the study, or a history of substance abuse.

Inclusion Criteria

Has behaviors considered moderate or severe per the CareGI-S at Screening

I am within the age range required for this study.

I have been diagnosed with TSC and have a history of seizures.

Exclusion Criteria

Has abnormal laboratory values at the Baseline Visit that are of clinical significance

Has received an investigational medicinal product within the 3 months prior to the Screening Visit

I have a serious health condition that is not epilepsy.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Cannabidiol Oral Solution titrated up to 12.5 mg/kg twice daily for 26 weeks

26 weeks

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment for an additional 26 weeks

26 weeks

Treatment Details

Interventions

• Epidiolex (Cannabinoid)

Trial Overview The study tests Epidiolex (Cannabidiol Oral Solution) as an additional treatment for TSC-related seizures and behavioral issues. It aims to see how adding this solution affects patients' behavior and other related outcomes alongside their current seizure medications.

Participant Groups

Group I: Cannabidiol Oral Solution Experimental Treatment 1 Intervention

Participants who will receive the Cannabidiol Oral Solution (CBD-OS) titrated up to a dose of 12.5 mg/kg administered twice daily for a total dose of up to 25 mg/kg/day for 26 consecutive weeks. Participants will have the option to continue receiving the CBD-OS for an additional 26 weeks, for a total of 52 weeks.

Who Is Running the Clinical Trial?

Jazz Pharmaceuticals

Lead Sponsor

Trials

252

Recruited

35,100+

Bruce C. Cozadd

Jazz Pharmaceuticals

Chief Executive Officer since 2009

BA in Economics from Yale University, MBA from Stanford University

Dr. Austin

Jazz Pharmaceuticals

Chief Medical Officer since 2023

MD from the Royal College of Surgeons in Ireland

Findings from Research

A single oral dose of 750 mg of cannabidiol (CBD) showed significantly increased exposure when taken with a high-fat/calorie meal, with a 3.8-fold increase in overall exposure (AUC0-∞) and a 5.2-fold increase in maximum concentration (Cmax) compared to fasting.

CBD was well-tolerated with no severe adverse events reported, indicating its safety in healthy adults, while its absorption was also enhanced by low-fat meals, whole milk, and alcohol, albeit to a lesser extent.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase 1, randomized, pharmacokinetic trial of the effect of different meal compositions, whole milk, and alcohol on cannabidiol exposure and safety in healthy subjects. Crockett, J., Critchley, D., Tayo, B., et al.[2021]

Epidiolex, a standardized CBD solution, was found to be safe and well-tolerated in a phase I study involving 21 patients with biochemically recurrent prostate cancer, with no dose-limiting toxicities observed at doses of 600 mg and 800 mg.

At the 12-week mark, 88% of patients showed stable biochemical disease, indicating preliminary anti-tumor activity, with one patient experiencing a significant 41% decline in PSA levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Phase I Dose Escalation and Expansion Study of Epidiolex (Cannabidiol) in Patients with Biochemically Recurrent Prostate Cancer. Myint, ZW., St Clair, WH., Strup, SE., et al.[2023]

In a study of 108 patients with refractory epilepsy taking Epidiolex®, 75% remained on the treatment at the final evaluation, indicating that it is generally well-tolerated and effective for long-term use.

However, 46.3% of patients experienced treatment-emergent adverse effects, with 14.5% discontinuing due to these effects, highlighting the need for further research on managing side effects and drug interactions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world, long-term evaluation of the tolerability and therapy retention of Epidiolex® (cannabidiol) in patients with refractory epilepsy. Georgieva, D., Langley, J., Hartkopf, K., et al.[2023]

In a study involving 45 children with treatment-resistant epilepsy, cannabidiol (CBD) significantly reduced major seizure frequency by 54-72% and increased seizure-free days by an average of 7.52 days over 36 months, demonstrating its efficacy as an adjunctive treatment.

CBD was well tolerated at doses up to 50 mg/kg/day, although children who increased their dose beyond 25 mg/kg/day initially reported more adverse events; however, the overall rate of adverse events decreased after transitioning to higher doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term efficacy and safety of cannabidiol (CBD) in children with treatment-resistant epilepsy: Results from a state-based expanded access program. Park, YD., Linder, DF., Pope, J., et al.[2021]

References

1. United States pubmed.ncbi.nlm.nih.gov

A phase 1, randomized, pharmacokinetic trial of the effect of different meal compositions, whole milk, and alcohol on cannabidiol exposure and safety in healthy subjects. [2021]

2. Switzerland pubmed.ncbi.nlm.nih.gov

A Phase I Dose Escalation and Expansion Study of Epidiolex (Cannabidiol) in Patients with Biochemically Recurrent Prostate Cancer. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Cannabidiol: A New Hope for Patients With Dravet or Lennox-Gastaut Syndromes. [2020]

4. United States pubmed.ncbi.nlm.nih.gov

Real-world, long-term evaluation of the tolerability and therapy retention of Epidiolex® (cannabidiol) in patients with refractory epilepsy. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Long-term efficacy and safety of cannabidiol (CBD) in children with treatment-resistant epilepsy: Results from a state-based expanded access program. [2021]