Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q… (NCT05149898) | Clinical Trial Compass
CompletedPhase 2
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
United States, Australia20 participantsStarted 2020-02-19
Plain-language summary
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to \<18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).
Who can participate
Age range4 Years – 17 Years
SexALL
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Inclusion criteria
✓. Male or female children and adolescents aged 4 to less than 18 years, at the time of Screening.
✓. 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.
✓. Participants must have a diagnosis of 22qDS confirmed by genetic testing.
✓. Participants have a Clinical Global Impression-Severity (CGI-S) score of 4 or higher at Screening and Visit 2.
✓. Participants must have a Pediatric Anxiety Rating Scale-Revised (PARS-R) severity score of 10 or higher at Screening and Visit 2.
✓. Participants 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.
✓. If participants are receiving non-pharmacological behavioral and/or dietary interventions, they must be stable for three months prior to Screening.
✓. The participant has demonstrated stable calcium levels for one year prior to Screening.
Exclusion criteria
✕. Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male participants 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.
✕
What they're measuring
1
Overall Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Timeframe: From first dose of study drug administration (Day 1) up to end of the study, approximately 306 days
. History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
✕. Exposure to any investigational drug or device ≤ 30 days prior to Screening or at any time during the study.
✕. Participant has Alanine transaminase (ALT), Aspartate transaminase (AST), or total bilirubin levels ≥ 2 times the Upper limit of normal (ULN) or has alkaline phosphatase levels ≥ 3 times the ULN as determined from Screening safety laboratories.
✕. Use of cannabis or any Δ9-tetrahydrocannabinol (THC) or CBD-containing product within three months of Screening Visit or during the study.
✕. The participant has a positive drug screen.
✕. The participant is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin or vigabatrin.
✕. Participant 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.