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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new method to deliver CBD, a compound from cannabis, through the skin to help manage seizures in people with Dravet and Lennox-Gastaut syndromes. The researchers aim to determine if this method, known as the GT4 Transdermal Delivery System, is safe and effective, potentially offering more stable CBD levels in the bloodstream. Participants must have a confirmed diagnosis of one of these conditions and experience frequent seizures despite current treatments. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative treatment.

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.

What prior data suggests that this transdermal CBD delivery system is safe for individuals with epilepsy?

Earlier studies found the GT4 patch, which delivers CBD through the skin, to be safe and well tolerated. Research shows that this method does not cause the "high" feelings usually linked to cannabis. The patch allows CBD to enter the bloodstream through the skin, avoiding some side effects that can occur with oral CBD.

CBD is generally considered safe and is often used to help manage seizures in conditions like Dravet and Lennox-Gastaut syndromes, which are the focus of this trial. Some people have reported mild side effects, such as sleepiness, but these effects are usually temporary.

Since this trial is in later stages after initial safety testing, the treatment has shown a promising safety record so far. This indicates it has been safe enough in earlier research to be tested in larger groups. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about the CBD: GT4 Transdermal Delivery System for epilepsy because it offers a unique way to manage seizures. Unlike traditional treatments like oral antiepileptic drugs, this system delivers CBD through the skin, potentially offering a more consistent release and avoiding the digestive system. This method could mean fewer side effects and more stable blood levels of the medication. Additionally, using CBD, a compound derived from cannabis, might offer a new mechanism of action, particularly for patients who do not respond well to existing medications.

What evidence suggests that this transdermal CBD treatment might be effective for epilepsy?

Research has shown that CBD can greatly reduce seizures in people with epilepsy, including those with Dravet and Lennox-Gastaut syndromes. Some patients have even stopped having seizures after using CBD. In this trial, participants will receive the CBD: GT4 Transdermal Delivery System, which applies CBD to the skin. This transdermal method might maintain more stable CBD levels in the blood compared to oral or inhaled administration. It can also help avoid some drug interactions and deliver CBD more effectively into the bloodstream. Studies suggest that side effects, such as drowsiness, can occur but are usually manageable. ^{1 2 4 6 7}

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

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]

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]

Citations

1. withpower.com withpower.com/trial/phase-2-epilepsies-myoclonic-2025-42a05

Transdermal CBD for Epilepsy · Info for Participants Studies have demonstrated that CBD can lead to significant improvements in seizure frequency, with some patients becoming seizure-free or experiencing more than ...

2. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/561439

CBD Delivery with the A-Synaptic GT4 Transdermal Delivery ... This study is a preliminary open-label, single-arm Phase II investigation into the safety and efficacy of transdermal cannabidiol (CBD) delivered using GT4 ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9859876/

Examining the Systemic Bioavailability of Cannabidiol and ... Furthermore, transdermal delivery of CBD and THC appeared to result in a smaller Cmax than has been found for inhaled and orally consumed cannabis.

4. ichgcp.net ichgcp.net/clinical-trials-registry/NCT06738732

CBD: GT4 Transdermal Delivery System in Dravet Syndrome ... This study is a preliminary open-label, single-arm Phase II investigation into the safety and efficacy of transdermal cannabidiol (CBD) ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S0928098725000351

Cannabidiol—A friend or a foe? In epilepsy studies, including severe cases like Dravet syndrome, CBD reduced seizures effectively, though side effects like somnolence, ...

6. ctv.veeva.com ctv.veeva.com/study/cbd-delivery-with-the-a-synaptic-gt4-transdermal-delivery-system-in-with-dravet-syndrome-and-or-lenn

CBD Delivery with the A-Synaptic GT4 Transdermal Delivery ... This study is a preliminary open-label, single-arm Phase II investigation into the safety and efficacy of transdermal cannabidiol (CBD) ...

7. go.drugbank.com go.drugbank.com/conditions/DBCOND0140482

Dravet Syndrome (DS) (DBCOND0140482) An active cannabinoid used as an adjunctive treatment for the management of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome and ...