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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05149898




Registration number
NCT05149898
Ethics application status
Date submitted
5/10/2021
Date registered
8/12/2021
Date last updated
3/11/2023

Titles & IDs
Public title
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome
Scientific title
Open-Label, Tolerability and Efficacy Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome
Secondary ID [1] 0 0
ZYN2-CL-031
Universal Trial Number (UTN)
Trial acronym
INSPIRE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
1. Male or female children and adolescents aged 4 to less than 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.
3. Patients must have a diagnosis of 22q Deletion Syndrome (22qDS) confirmed by genetic testing.
4. Patients have a Clinical Global Impression-Severity (CGI-S) score of 4 or higher at Screening and Visit 2.
5. Patients must have a Pediatric Anxiety Rating Scale-Revised (PARS-R) severity score of 10 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 12-35 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.
Minimum age
4 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 for the duration of therapy and for three months after the last dose of study medication.
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 = 30 days prior to Screening or at any time during the study.
4. Patient has ALT, AST, or total bilirubin levels = 2 times the ULN)or has alkaline phosphatase levels = 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.
7. Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin 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, St. John's Wort, and grapefruit juice/products.
9. Patient with diagnosis of known genetic disorder, other than 22qDS.
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, PTSD or major depressive disorder.
12. Patient 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 requirements.
16. Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies.
17. Patients at risk of needing cardiovascular surgical repair within the upcoming 12 months.
18. Patient has unstable cardiovascular disease, such as advanced arteriosclerosis, 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), other serious or other clinically unstable cardiac problems as indicated by history, physical examination, or ECG.
19. 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.
20. Any skin disease or condition that may affect treatment application, application site assessments, or absorption of the study drug.
21. History of treatment for, or evidence of, drug abuse within the past year.
22. Patient responds "yes" to Question '4' or '5' on the Columbia Suicide Severity Rating Scale Children (C-SSRS) during Screening or at any time on study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/03/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
21/11/2022
Sample size
Target
Accrual to date
Final
20
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Zynerba Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in patients ages 4 to \< 18 years, in the treatment of 22q.11.2 Deletion Syndrome (22qDS).
Trial website
https://clinicaltrials.gov/study/NCT05149898
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Carol O'Neill
Address 0 0
VP, Development
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05149898