Clinical Study of Synthetic Cannabidiol in Children and Adolescents with 22q11.2 Deletion Syndrome
- Conditions
- Pediatric 22q11.2 deletion syndromeNeurological - Other neurological disordersMental Health - Psychosis and personality disordersHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
- Registration Number
- ACTRN12619000673145
- Lead Sponsor
- Zynerba Pharmaceuticals Pty Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 20
1.Male or female children and adolescents aged 6 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, and clinical laboratory test results. Laboratory results outside of the reference range must be documented as not clinically significant by the Investigator.
3.Patients must have a diagnosis of 22qDS confirmed by genetic testing, with or without autistic features.
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 for three months prior to Screening.
8.Patient has demonstrated stable calcium levels for one year prior to Screening.
9.Patients have a body mass index between 12–30 kg / m2 (inclusive).
10.Females of childbearing potential must have a negative pregnancy test at the Screening Visit and a negative pregnancy test at all designated study visits.
11.Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.
12.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.
13.Parents/caregiver(s) must provide written consent to assist in study drug administration.
14.In the Investigator’s opinion, patients and parents/caregivers are reliable and willing and able to comply with all protocol requirements and procedures.
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.
a.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 less than or equal to 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels less 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 for sympathomimetic amines (amphetamines (unless prescribed); benzodiazepines; buprenorphine; cannabinoids; methadone; cocaine (metabolites); and opiates; (excludes midazolam used at blood draws, and barbiturates used as AED medication), including ethanol.
7.Patient is using the following AEDs: 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 (except single doses administered for the purposes of obtaining blood samples and ECG’s), 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, other than 22qDS (i.e. Prader-Willi Syndrome, Angelman Syndrome, Fragile X Syndrome, Rett Syndrome etc.).
10.Patient has diagnosis of DiGeorge or Velocardiofacial syndrome without the presence of 22qDS.
11.Patient has a primary psychiatric diagnosis other than 22qDS or anxiety, including bipolar disorder, psychosis, schizophrenia, post-traumatic stress disorder (PTSD) or major depressive disorder.
12.Patients is on stable treatment of >6 months of not more than two psychoactive medications at screening or throughout the study (with the exception of one psychoactive medication prescribed for sleep).
13.Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations.
14.Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
15.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 req
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method