ClinicalTrials.gov record NCT06738732
CBD Delivery with the A-Synaptic GT4 Transdermal Delivery System in with Dravet Syndrome And/or Lennox-Gastaut Syndrome
Preliminary Evaluation of the Safety and Efficacy of CBD Delivery with the A-Synaptic GT4 Transdermal Delivery System in Individuals Diagnosed with Dravet Syndrome And/or Lennox-Gastaut Syndrome
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This early-phase (Phase 1 and 2) open-label, single-arm study tests transdermal cannabidiol (CBD) delivered with the A‑Synaptic GT4 system in people diagnosed with Dravet syndrome and/or Lennox‑Gastaut syndrome. The study plans to enroll about 25 participants aged 2 to 55, with dosing starting at Visit #2 and a schedule of 11 visits over roughly 160 days. Investigators will monitor safety, treatment-emergent adverse events, compliance, seizure counts, and blood CBD levels.
What it is studying. The study is testing the safety and efficacy of CBD when given through the A‑Synaptic GT4 transdermal delivery system in people with Dravet syndrome and/or Lennox‑Gastaut syndrome. It will also record seizure frequency, measure blood concentrations of CBD and its metabolites (and concurrent antiseizure medicines), and track adherence and adverse events.
What the researchers hope to learn. To evaluate safety and tolerability of CBD delivered with the GT4 transdermal system; to measure changes in seizure frequency; to characterize blood concentrations of CBD and its metabolites and any interactions with concurrent antiseizure drugs; and to assess participant compliance with the delivery regimen.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- CBD: GT4 Transdermal Delivery System
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Males and females between the age of 2-55 years, inclusive
- Females not of child-bearing potential, defined as those who have undergone a sterilization procedure (e.g. hysterectomy, bilateral oophorectomy, bilateral tubal ligation, complete endometrial ablation), have been post-menopausal for at least 1 year prior to screening, or have not reached menarche Or,
- 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. All hormonal birth control must have been in use for a minimum of three months. Acceptable methods of birth control include:
- Hormonal contraceptives including oral contraceptives, hormone birth control patch (Ortho Evra), vaginal contraceptive ring (NuvaRing), injectable contraceptives (Depo-Provera, Lunelle), or hormone implant (Norplant System)
- Double-barrier method
- Intrauterine devices
- Non-heterosexual lifestyle or agrees to use contraception if planning on changing to heterosexual partner(s)
- Vasectomy of partner at least 6 months prior to screening
- Abstinence and agrees to use contraception if planning on becoming sexually active
- Clinically confirmed and documented diagnosis of refractory DS and/or LGS. Documentation of diagnosis must be provided by a neurologist, pediatrician, or primary care practitioner
- Reported ≥4 countable seizures during the 28-day run in period
- Participants taking ≥1 AED at a stable dose for ≥4 weeks prior to screening, and participants and/or caregivers willing to maintain dose for duration of study period
- Non-pharmacological therapies (e.g., vagus nerve stimulation, ketogenic diet, modified Atkins diet) stable for ≥4 weeks prior to screening, and participants and/or caregivers willing to maintain a stable regimen for the duration of the study period
- Adults to provide voluntary, written, informed consent to participate in the study. If under the age of consent or unable to consent due to cognitive impairment, the participant and the participant's parent(s), legal guardian(s), or caregiver(s) to provide voluntary, written, informed assent and consent, respectively, for participation in the study
- Otherwise healthy as determined by medical history, laboratory results, electroencephalogram (EEG), vital signs, and physical examination, as assessed by the QI/MD
Exclusion criteria
- Individuals who are pregnant, breast feeding, or planning to become pregnant during the study
- Allergy, sensitivity, or intolerance to the investigational product's active and/or inactive ingredients
- Acute or chronic skin disease (e.g., atopic dermatitis, eczema, rosacea, psoriasis) or dermatological conditions (scars, moles, etc.) in the proposed area of application that may interfere with the application and absorption of the investigational product, as assessed by the QI/MD
- Etiology of participant seizures is related to progressive neurologic disease, as assessed by the QI/MD.
- Currently prescribed >4 concurrent AEDs
- Current unstable significant psychiatric or psychological condition (e.g., schizophrenia, bipolar disorder, clinical depression, eating disorders) and/or history of suicidal behavior or any suicidal ideation as assessed by the C-SSRS at screening, as appropriate, as assessed by the QI/MD (See Section 9.13.2)
- History of psychosis in immediate family including schizophrenia and affective psychosis
- Anoxic episode requiring resuscitation in the past 6 months
- Unstable hypertension. Treatment on a stable dose of medication for at least 3 months will be considered by the QI/MD
- Type I or Type II diabetes
- Significant cardiovascular event in the past 6 months. Participants with no significant cardiovascular event on stable medication may be included after assessment by the QI/MD on a case-by-case basis
- History of or current diagnosis with kidney and/or liver diseases as assessed by the QI/MD on a case-by-case basis, with the exception of history of kidney stones in participants who are symptom free for 6 months
- Self-reported confirmation of current or pre-existing thyroid condition. Treatment on a stable dose of medication for at least 3 months will be considered by the QI/MD
- Major surgery in the past 3 months or individuals who have planned surgery during the course of the study. Participants with minor surgery will be considered on a case-by-case basis by the QI/MD
- Cancer, except skin basal cell carcinoma completely excised with no chemotherapy or radiation with a follow up that is negative. Volunteers with cancer in full remission for more than five years after diagnosis are acceptable
- Individuals with an autoimmune disease or are immune compromised
- Self-reported confirmation of a HIV-, Hepatitis B- and/or C-positive diagnosis
- Self-reported confirmation of blood/bleeding disorders as assessed by QI/MD
- Use of medical or recreational cannabinoid products prior to screening (see Section 7.3). Participants must agree to abstain for the duration of the run-in and study period
- Regular use of tobacco or nicotine products in the past 6 months and during the run-in and study period, as assessed by the QI/MD
- Alcohol intake average of >2 standard drinks per day, as assessed by the QI/MD. Occasional consumers required to abstain for the duration of the run-in and study period
- Alcohol or drug abuse within the last 24 months
- Current use of any prescribed or over-the counter medications and/or supplements that may affect the safety and/or efficacy of the investigational product, as assessed by the QI/MD (See Sections 7.3.1 and 7.3.2)
- Plans to travel outside country of residence during the study period
- Clinically significant abnormal laboratory results, adverse events, or abnormalities in the EEG at screening, as assessed by the QI/MD
- Participation in other clinical research studies 30 days prior to baseline, as assessed by the QI/MD
- Any other condition, chronic disease, or lifestyle factor, that, in the opinion of the QI/MD, may adversely affect the participant's ability to complete the study or its measures or pose significant risk to the participant
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will have the GT4 transdermal CBD applied starting at Visit 2 and attend about 11 clinic visits over approximately 160 days. Visits include dosing, safety checks (vitals, physical exam, EEG as required), and regular blood draws to measure CBD, CBD metabolites, and levels of concomitant AEDs at multiple time points. Caregivers or participants will track and report countable seizures to the study team and report adverse events. Participants must abstain from other cannabinoid products, avoid regular tobacco/nicotine use, keep AEDs and stable non-drug therapies unchanged, and follow contraception and other screening-related requirements.
How the study is structured. An open-label, single-group interventional study: all participants receive the active GT4 transdermal CBD product (no placebo, no blinding). Participants attend 11 visits over about 160 days; dosing begins at Visit #2. Key measurements (safety, compliance, seizure counts, and blood drug levels) are collected at multiple visits up to 112 days after treatment.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Dravet syndrome
This trial is also listed on this site under CDKL5 deficiency disorder.
Important dates
- Study start
- January 2025 (estimated)
- Primary completion
- January 2027 (estimated)
- Study completion
- January 2027 (estimated)
- Target enrollment
- 25 participants (estimated)
- Last updated on registry
- Dec 18, 2024
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Alexander Rotenberg
- Collaborators
- A-Synaptic
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Clinical Research Manager 📞 617-919-4617 ✉ melissa.dibacco@childrens.harvard.edu
Participating sites (1)
- Boston Childrens' Hospital
Boston, Massachusetts, 02115 · United States
- Melissa DiBacco, MD · 617-919-4617 · melissa.dibacco@childrens.harvard.edu
Terms used on this page · explanations by MedicalUpdates.ai
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- Phase II
- A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- expanded access
- A way for some people with serious conditions to receive an investigational treatment outside a clinical trial, under specific rules. It is not itself a clinical trial and is not designed to test whether the treatment works.
- sponsor
- The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- stable dose
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- assent
- A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
- investigational product
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- follow up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- run-in
- A period at the start of a study, before the study treatment begins, used for example to confirm that participants meet the criteria or to record their usual symptoms.
- adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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