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.
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,
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
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.
Single group
CBD: GT4 Transdermal Delivery System
DrugCBD: GT4 Transdermal Delivery System
Compliance
Proportion of participants compliant with study dosing regimen for visits at baseline, 7, 14, 21, 28, 56, 84, and 112 days post treatment;
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.
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.
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.
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