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Lennox-Gastaut Syndrome (LGS)Phase 1/2NCT06738732

CBD Delivery with the A-Synaptic GT4 Transdermal Delivery System in with Dravet Syndrome And/or Lennox-Gastaut Syndrome

A Phase 1/2 study testing CBD: GT4 Transdermal Delivery System in people with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS), run by Alexander Rotenberg at 1 site in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 2 to 55 years
What is being tested?
CBD: GT4 Transdermal Delivery System
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
1 siteUnited States
Study dates
Jan 2025to Jan 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 55 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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)
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  • 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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Clinical Research Manager contact

617-919-4617

melissa.dibacco@childrens.harvard.edu

United States1 site

  • Boston Childrens' Hospital

    Boston, Massachusetts, 02115

    Melissa DiBacco, MD, 617-919-4617, melissa.dibacco@childrens.harvard.edu

Other trials in United States

About this study

Official title: 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

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.

Read more

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.

Being tested
CBD: GT4 Transdermal Delivery System
Keywords
CBD, transdermal
Medical subject headings
Lennox Gastaut Syndrome, Epilepsies, Myoclonic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CBD: GT4 Transdermal Delivery SystemDrug

    CBD: GT4 Transdermal Delivery System

Study groups in NCT06738732
Study groupRoleWhat they receive
Active TherapyExperimentalCBD: GT4 Transdermal Delivery System
Receives: Drug: CBD: GT4 Transdermal Delivery System

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Compliance

    Proportion of participants compliant with study dosing regimen for visits at baseline, 7, 14, 21, 28, 56, 84, and 112 days post treatment;

    Measured: 112 Days

  2. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

    Pre-emergent and post-emergent adverse events will be described in separate frequency tables. The description, frequency, type, severity, causality, and outcome of each adverse event will also be listed.

    Measured: 112 Days

Secondary outcomes (2)

  1. Seizure Frequency

    Proportion, relative to baseline will be computed for three seizure types: (1) drop, (2) generalized tonic, and (3) generalized tonic-clonic. Seizure freqeuncy values will be collected 28, 56, 84 and 112 days post treatment to compute percent change in frequency at 28, 56, 84 and 112 days post treatment.

    Measured: 112 Days

  2. CBD, 7-COOH-CBD, and 7-OH-CBD blood concentrations, and blood concentrations of concomitant AEDs

    Blood will be collected at morning trough, before morning medicaiton adminitration. Summary statistics including means, standard deviations, medians, minimums, maximums, on secondary outcome variables: CBD, 7-COOH-CBD, and 7-OH-CBD blood concentrations, and blood concentrations of concomitant AEDs will be obtained for baseline, 1, 7, 14, 21, 28, 56, 84 and 112 days post treatment for the overall ITT and PP populations. Similar summary statistics will also be obtained for changes from baseline to 1, 7, 14, 21, 28, 56, 84 and 112 days post treatment.

    Measured: 112 Days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Dec 2024
  2. Study startJan 2025 (estimated)
  3. Primary completionJan 2027 (estimated)
  4. Study completionJan 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
25 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06738732
Sponsor's protocol ID
IRB-P00047233
Status last verified
Nov 2024
First submitted
8 Nov 2024
Last updated
18 Dec 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CBD: GT4 Transdermal Delivery System in people with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS), run by Alexander Rotenberg at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 18 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Clinical Research Manager (617-919-4617, melissa.dibacco@childrens.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Compliance (time frame: 112 Days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06738732.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2024.

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