Transdermal CBD for Epilepsy

Overseen By Clinical Research Manager

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Alexander Rotenberg

Must be taking: AEDs

Must not be taking: Cannabinoids, Tobacco, Alcohol, others

Disqualifiers: Pregnancy, Skin disease, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 12

Duration: 16 weeks

25 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This study is a preliminary open-label, single-arm Phase II investigation into the safety and efficacy of transdermal cannabidiol (CBD) delivered using GT4 skin bream technology in individuals diagnosed with Dravet and/or Lennox-Gastatu syndrome (DS and/or LGS). We aim to enroll 25 participants between the ages of 2 and 55 diagnosed with DS and/or LGS. Transdermal delivery of cannabinoids may provide advantages over other traditional routes of administration. Noted advantages include avoidance of first pass metabolism which mitigates potentially dangerous drug-drug interactions due to delayed cannabinoid accumulation, and more stable and constant plasma cannabinoid concentrations. GT4 technology, uses emulsion technology containing penetrating agents, basement membrane disruptors, and vasodilators to overcome hydrophilic and lipophilic structures to open channels and transport cannabinoids deep into the dermis layer of the skin. Once in the dermis, vasodilators dilate the capillary bed to increase fluid dynamic flow into and out of the application site, delivering cannabinoids into the blood stream. The primary objective is to investigate the safety and efficacy of CBD delivery with the A-Synaptic GT4 Transdermal Delivery System in individuals diagnosed with DS and/orLGS. Dr. Rotenberg will apply for and hold the expanded access IND for this study, as the sponsor is running this study as an investigator-initiated study. The study consists of 11 visits over \~160 days, dosing begins at Visit #2.

Will I have to stop taking my current medications?

Participants must be on a stable dose of at least one anti-epileptic drug (AED) for at least 4 weeks before the study and must maintain this dose during the study. However, if you are taking more than four AEDs or any other medications that might affect the study, you may need to stop or adjust them as assessed by the study doctor.

Is transdermal CBD safe for humans?

CBD has been shown to be generally safe in humans, with common mild side effects like drowsiness and reduced appetite. However, it can interact with other medications, and there is limited information on its long-term safety. ^{1 2 3 4 5}

How is the GT4 Transdermal Delivery System for CBD different from other epilepsy treatments?

The GT4 Transdermal Delivery System for CBD is unique because it delivers CBD through the skin, which may offer a more consistent absorption compared to oral formulations that have variable bioavailability. This method could potentially reduce the side effects associated with oral CBD, such as gastrointestinal issues, by bypassing the digestive system. ^{2 4 6 7 8}

What data supports the effectiveness of the drug GT4 Transdermal Delivery System for epilepsy?

Research shows that CBD, the active ingredient in the GT4 Transdermal Delivery System, has been effective in reducing seizures in people with treatment-resistant epilepsy. Studies have demonstrated that CBD can lead to significant improvements in seizure frequency, with some patients becoming seizure-free or experiencing more than a 50% reduction in seizures. ^{2 4 9 10 11}

Who Is on the Research Team?

Alexander Rotenberg, MD, PhD

Principal Investigator

Boston Children's Hospital

Are You a Good Fit for This Trial?

This trial is for individuals aged 2 to 55 with Dravet Syndrome (DS) and/or Lennox-Gastaut Syndrome (LGS), which are forms of epilepsy. Participants must be diagnosed with one or both conditions to qualify.

Inclusion Criteria

I had 4 or more seizures in the last 28 days.

My non-drug treatments have been the same for the last 4 weeks and I can keep them stable.

Individuals of child-bearing potential must have a negative baseline urine pregnancy test and agree to use a medically approved method of birth control for the duration of the study

Exclusion Criteria

I have or had a thyroid condition.

I have not had major surgery in the last 3 months and have no plans for surgery during the study.

I needed revival from a severe lack of oxygen in the last 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive transdermal CBD using the GT4 system, with dosing beginning at Visit #2

16 weeks

11 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CBD: GT4 Transdermal Delivery System

Trial Overview The study tests a new way to deliver CBD through the skin using GT4 technology in patients with DS and/or LGS. It aims to see if this method is safe and works well by providing stable CBD levels in the blood without traditional drug interactions.

How Is the Trial Designed?

Group I: Active Therapy Experimental Treatment 1 Intervention

CBD: GT4 Transdermal Delivery System

Who Is Running the Clinical Trial?

Alexander Rotenberg

Lead Sponsor

Trials

1

Recruited

30+

A-Synaptic

Collaborator

Trials

1

Recruited

30+

Published Research Related to This Trial

In a study involving 169 participants with treatment-resistant epilepsy, highly purified cannabidiol (CBD) demonstrated significant reductions in seizure frequency and severity over a two-year period, with 61% of children and 71% of adults achieving at least a 50% reduction in seizures by the end of the study.

CBD was generally well tolerated, with the most common side effects being diarrhea, sedation, and decreased appetite, and only a minority of participants discontinued treatment due to adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of highly purified cannabidiol for treatment refractory epilepsy. Gaston, TE., Ampah, SB., Martina Bebin, E., et al.[2021]

Cannabidiol (CBD), particularly in the form of Epidiolex®, has shown efficacy and an adequate safety profile in treating treatment-resistant epilepsy (TRE) in children and young adults, especially those with Dravet Syndrome and Lennox-Gastaut Syndrome, based on Phase 3 randomized controlled trials.

Despite the historical use of cannabis for epilepsy, valid data on the safety and efficacy of artisanal cannabis preparations remains lacking, highlighting the importance of relying on well-conducted clinical trials for treatment decisions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cannabinoids in treatment-resistant epilepsy: A review. O'Connell, BK., Gloss, D., Devinsky, O.[2022]

Citations

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

An Open Retrospective Study of a Standardized Cannabidiol Based-Oil in Treatment-Resistant Epilepsy. [2022]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Cannabidiol-enriched oil in children and adults with treatment-resistant epilepsy-does tolerance exist? [2022]

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

The role of cannabinoids in epilepsy treatment: a critical review of efficacy results from clinical trials. [2020]

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

Long-term safety and efficacy of highly purified cannabidiol for treatment refractory epilepsy. [2021]

5. Italy pubmed.ncbi.nlm.nih.gov

Cannabis-based magistral formulation is highly effective as an adjuvant treatment in drug-resistant focal epilepsy in adult patients: an open-label prospective cohort study. [2023]

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

Cannabinoids in treatment-resistant epilepsy: A review. [2022]

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

Therapeutic and clinical foundations of cannabidiol therapy for difficult-to-treat seizures in children and adults with refractory epilepsies. [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Cannabidiol in the Treatment of Epilepsy: A Focused Review of Evidence and Gaps. [2020]

9. New Zealand pubmed.ncbi.nlm.nih.gov

Pharmacological and Therapeutic Properties of Cannabidiol for Epilepsy. [2020]

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

The safety, tolerability, and effectiveness of PTL-101, an oral cannabidiol formulation, in pediatric intractable epilepsy: A phase II, open-label, single-center study. [2020]

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

A prospective open-label trial of a CBD/THC cannabis oil in dravet syndrome. [2022]