None listed
Conditions
Brief summary
This is an open-label single-center study, to assess the safety, tolerability and efficacy of cannabidiol administered as ZYN002, a transdermal gel, for the treatment of child and adolescent patients with Autism Spectrum Disorder (ASD). Male and female patients with ASD will be treated for 14 weeks. Patients taking Anti Epileptic Drug medications will have an additional one or two week Taper Period after the completion of doing with ZYN002. Approximately 36 male and female patients, ages 4 to < 18 years, will receive ZYN002
Interventions
This is an open-label single-center study, to assess the safety, tolerability and efficacy of ZYN002 (a synthetic cannabidiol) administered as a transdermal gel, for the treatment of child and adolescent patients with Autism Spectrum Disorder. Approximately 36 male and female patients ages 4 to < 18 years will be treated for 14 weeks.
The study gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms.
All participants will undergo a screening process to assess eligibili
This is an open-label single-center study, to assess the safety, tolerability and efficacy of ZYN002 (a synthetic cannabidiol) administered as a transdermal gel, for the treatment of child and adolescent patients with Autism Spectrum Disorder. Approximately 36 male and female patients ages 4 to < 18 years will be treated for 14 weeks. The study gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms. All participants will undergo a screening process to assess eligibility. Eligible participants will then participate in up to a 14 week treatment period, where all participants will received ZYN002. Parents/caregivers will apply the study gel twice daily for the treatment period. Participants who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002 (125 mg of cannabidiol), applied every 12 hours (± 2 hours). Participants who weigh more than 35 kg will receive 2 sachets of ZYN002 (250 mg of cannabidiol), applied every 12 hours (± 2 hours). Participants who are taking anti-epileptic drugs may have an additional one or two weeks of treatment to taper off study treatment. After the final dose, patients will be followed weekly for 4 weeks by telephone, prior to discharge from the study. Patient compliance with the intervention will be monitored by the study coordinator through drug accountability at each study visit.
Sponsors
Zynerba Pharmaceuticals Pty Ltd
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
4 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Male or female children and adolescents aged 4 to <18 years, at the time of Screening. 2. Judged by the Investigator to be in generally good health at Screening 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 ASD confirmed by DSM-5 criteria. 4. Patients have a CGI-S score of 4 or higher at Screening and Visit 2. 5. Patients must have a score on the ABC-C Irritability Subscale of 18 or higher at Screening and Visit 2. 6. Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of one or two AEDs, or must be seizure-free for one year if not currently receiving AEDs. 7. If patients are receiving non-pharmacological behavioral and/or dietary interventions, they must be stable and have been doing so for three months prior to Screening. 8. Patients have a body mass index between 12–30 kg / m2 (inclusive). 9. Females of childbearing potential must have a negative pregnancy test at the Screening Visit and a negative pregnancy test at all designated study visits. 10. Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures. 11. Patients and parents/caregivers must be adequately informed of the nature and risks of the study and give written informed consent (and assent if applicable) prior to Screening. 12. Parents/caregiver(s) must provide written consent to assist in study drug administration. 13. In the Investigator’s opinion, patients and parents/caregivers are reliable and willing and able to comply with all protocol requirements and procedures.
Exclusion criteria
1. Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception as outlined below for the duration of therapy and for three months after the last dose of study medication. Standard acceptable methods of contraception include abstinence or the use of a highly effective method of contraception, including hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, vasectomy, or intrauterine device. 2. History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients. 3. Exposure to any investigational drug or device less than or equal to 30 days prior to Screening or at any time during the study. 4. Patient has alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels greater than or equal to 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels greater than or equal to 3 times the ULN as determined from Screening safety laboratories. 5. Use of cannabis or any THC or CBD-containing product within three months of Screening Visit or during the study. 6. Patient has a positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines, and opiates. 7. Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, or vigabatrin. 8. Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for CYP3A4 including but not limited to the following medications: midazolam, oral ketoconazole, fluconazole, nefazadone, rifampin, alfentanil, alfuzosin, amiodarone, cyclosporine, dasatinib, docetaxol, eplerenone, ergotamine, everolimus, fentanyl, halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide, quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinioin, vincristine, vinorelbine, and St. John’s Wort. 9. Patient with diagnosis of known genetic disorder (ie. Prader-Willi Syndrome, Angelman Syndrome, Fragile X Syndrome, Rett Syndrome etc). 10. Patient has a primary psychiatric diagnosis other than ASD or anxiety, including bipolar disorder, psychosis, schizophrenia, post-traumatic stress disorder (PTSD) or major depressive disorder. 11. Patients may not be taking more than two psychoactive medications at screening or throughout the study. 12. Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations. 13. Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study. 14. Patient has an acute or progressive neurological disease, or any psychiatric disorder or severe mental abnormalities that are likely to require changes in drug therapy or interfere with the objectives of the study or ability to adhere to protocol requirements. 15. Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies. 16. Patient has known history of cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, risk factors for Torsades de pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome), or other serious cardiac problems. 17. Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study medication. 18. Any skin disease or condition, including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration, that may affect treatment application, application site assessments, or absorption of the study drug. 19. History of treatment for, or evidence of, drug abuse within the past year. 20. Patient responds “yes” to Question ‘4’ or ‘5’ on the C-SSRS (Children) during Screening or at any time on study.
Outcome results
None listed