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
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Lennox-Gastaut Syndrome (LGS)
- Sponsor
- Alexander Rotenberg
- Enrollment
- 25
- Locations
- 1
- Primary Endpoint
- Compliance
- Status
- Not yet recruiting
- Last Updated
- last year
Overview
Brief 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). 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.
Investigators
Alexander Rotenberg
Professor of Neurology, Harvard Medical School
Boston Children's Hospital
Eligibility Criteria
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
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
Outcomes
Primary Outcomes
Compliance
Time Frame: 112 Days
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]
Time Frame: 112 Days
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.
Secondary Outcomes
- Seizure Frequency(112 Days)
- CBD, 7-COOH-CBD, and 7-OH-CBD blood concentrations, and blood concentrations of concomitant AEDs(112 Days)