ACTRN12618000516280
Recruiting
Phase 2
Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a Transdermal Gel to Children and Adolescents (3 to <18 years) with Developmental and Epileptic Encephalopathy (BELIEVE 1)
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Epilepsy
- Sponsor
- Zynerba Pharmaceuticals Pty Ltd
- Enrollment
- 55
- Status
- Recruiting
- Last Updated
- 7 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Male or female, 3 to less than 18 years of age, inclusive, at the time of screening.
- •2\. Judged by the Investigator to be in generally good health at the Screening Visit based upon the results of a medical history, physical examination, 12\-lead ECG, and clinical laboratory test results.
- •3\. Patients must have a diagnosis of developmental and epileptic encephalopathy (DEE) as defined by the International League Against Epilepsy Classification (Scheffer 2017\) with generalized motor, focal motor and focal impaired awareness or focal bilateral to tonic\-clonic seizures. Examples of DEE that may be included, but not limited to: Lennox\-Gastaut Syndrome, Dravet Syndrome, West Syndrome/ Infantile Spasms and Doose Syndrome. The diagnosis must be established for 1 years and documented by review of electroencephalogram (EEG), , and or genetic testing if available, and narrative from the physician who manages the patient’s epilepsy. The patient/legally authorized representation may elect to have the patient participate in genetic seizure panel testing to characterize a genetic diagnosis.
- •4\. Patient must experience a threshold number of generalized motor seizures (i.e. tonic, clonic, atonic, tonic\-clonic seizures, and generalized tonic\-clonic), focal motor, focal impaired awareness or focal to bilateral tonic\-clonic seizures during the Baseline period.
- •5\. Patient is currently being treated and maintained with a stable regimen of between one (1\) and four (4\) AEDs for at least 4 weeks before screening, and willing to maintain a stable regimen during the treatment period. If a benzodiazepine is used as a rescue medication greater than 2 times per week, it will be counted as an AED. Patient taking ethosuximide, felbamate and vigabatrin must be on stable therapy for at least 6 months. Patient taking felbamate must have had no clinically relevant changes in hematology or liver function.
- •6\. Patient has history of developmental delay with regression, slowing or plateau in at least one developmental domain.
- •7\. All interventions for epilepsy must be stable for at least one month prior to screening.
- •8\. Patient/caregiver is able and willing to maintain daily diaries for seizures, daily skin assessments and good day/bad day assessment.
- •9\. Patient has a body mass index between 13 and 35 kg/m2 and weighs no less than 12 kg.
- •10\. Sexually active females of childbearing potential must use an acceptable method of contraception. Acceptable methods of contraception include: hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, vasectomy, and intrauterine device.
Exclusion Criteria
- •1\. Patient has a history of significant allergic condition, significant drug\-related hypersensitivity, or allergic reaction to any adhesives, compound, or chemical class related to ZYN002 or its excipients.
- •2\. Patient has been exposed to any investigational drug or device less than 30 days prior to screening or plans to take another investigational drug at any time during the study.
- •3\. Patient has used cannabis or any CBD\- or THC\-containing product within 12 weeks of the Screening Visit or during the study.
- •4\. Patient on the following AEDs for less than 6 months: ethosuximide, felbamate and vigabatrin.
- •5\. Patient has had a change in AED regimen in the 4 weeks prior to screening.
- •6\. Patient has alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels equal to 3x the upper limit of normal (ULN) as determined from Screening safety laboratories.
- •7\. Patient/caregiver demonstrates behavior indicating unreliability or inability to comply with the requirements of the protocol.
- •8\. Patient has had a change in epilepsy dietary therapy in the 4 weeks prior to screening.
- •9\. Patient has seizures secondary to illicit drug or alcohol use.
- •10\. Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for CYP3A4 including the following medications: midazolam, oral ketoconazole, fluconazole, nefazodone, rifampin, alfentanil, alfuzosin, amiodarone, cyclosporine, dasatinib, docetaxel, eplerenone, ergotamine, everolimus, fentanyl, halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide, quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinoin, vincristine, vinorelbine and St. John’s Wort.
Outcomes
Primary Outcomes
Not specified
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