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DC Field | Value | Language |
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dc.contributor.author | Tartaglia, N. | en |
dc.contributor.author | Budimirovic, D. | en |
dc.contributor.author | Erickson, C. | en |
dc.contributor.author | Heussler, Helen | en |
dc.contributor.author | Palumbo, J. | en |
dc.contributor.author | Tich, N. | en |
dc.contributor.author | Sebree, T. | en |
dc.contributor.author | Cohen, J. | en |
dc.contributor.author | Hagerman, R. | en |
dc.contributor.author | Berry-Kravis, E. | en |
dc.date.accessioned | 2022-11-07T23:49:33Z | - |
dc.date.available | 2022-11-07T23:49:33Z | - |
dc.date.issued | 2021 | en |
dc.identifier.citation | 65, (9), 2021, p. 805 | en |
dc.identifier.other | RIS | en |
dc.identifier.uri | http://dora.health.qld.gov.au/qldresearchjspui/handle/1/4119 | - |
dc.description.abstract | Background: ZYN002 is a pharmaceutically manufactured transdermal cannabidiol gel. CONNECT-FX was a randomised, doubleblind, placebo-controlled study to evaluate efficacy and safety of ZYN002 in patients 3-17 years with full mutation fragile X syndrome (FXS). Silencing of the FMR1 gene is now believed to require complete or near-complete methylation of the gene. Patients without silencing of the gene or significant mosaicism may represent a different population with higher levels of FMRP and perhaps mRNA. Methods: Patients were randomised to ZYN002 or placebo for 12 weeks. The primary endpoint was change from baseline in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale. Key secondary endpoints included change from baseline in the ABC-CFXS Irritability and Socially Unresponsive/Lethargic subscales and Clinical Global Impression, Improvement (CGI-I). A pre-planned ad hoc analysis was conducted in patients with ≥90% methylation of the FMR1 gene. Safety assessments included adverse events, laboratories and electrocardiograms. Results: Two hundred twelve patients were randomised. Mean age was 9.7 years; 75% were male. Improvements in ABC-CFXS Social Avoidance, Irritability and Socially Unresponsive/Lethargic scores and the CGI-I, while greater for ZYN002, were not significant in the overall group. Patients with ≥90% methylation (n = 169) showed a significant improvement in ABC-CFXS Social Avoidance (P = 0.020), and significantly more patients had a clinically meaningful change in Social Avoidance (OR 2.04, P = 0.031) and Irritability (OR 2.17, P = 0.036). ZYN002 was well tolerated. Adverse events were mild to moderate severity; application site pain was the most common treatment-related event (placebo 1%; ZYN002 6.4%). No serious or severe events nor other relevant abnormalities occurred. Conclusion: ZYN002 was well tolerated and demonstrated significant improvement in behavioural symptoms of FXS in patients with ≥90% methylation of the FMR1 gene, representing a biological population within FXS most likely to have silencing of the FMR1 gene.L6358489792021-09-03 <br /> | en |
dc.language.iso | en | en |
dc.relation.ispartof | Journal of Intellectual Disability Research | en |
dc.title | A pivotal study of zyn002 cannabidiol transdermal gel in children and adolescents with fragile x syndrome [connect-fx (ZYN2-cl-016)] | en |
dc.type | Article | en |
dc.identifier.doi | 10.1111/jir.12875 | en |
dc.subject.keywords | adolescent | en |
dc.subject.keywords | application site pain | en |
dc.subject.keywords | avoidance behavior | en |
dc.subject.keywords | child | en |
dc.subject.keywords | Clinical Global Impression scale | en |
dc.subject.keywords | comparative effectiveness | en |
dc.subject.keywords | conference abstract | en |
dc.subject.keywords | double blind procedure | en |
dc.subject.keywords | drug safety | en |
dc.subject.keywords | drug tolerability | en |
dc.subject.keywords | electrocardiogram | en |
dc.subject.keywords | female | en |
dc.subject.keywords | fragile X syndrome | en |
dc.subject.keywords | gene silencing | en |
dc.subject.keywords | human | en |
dc.subject.keywords | messenger RNA | en |
dc.subject.keywords | major clinical study | en |
dc.subject.keywords | male | en |
dc.subject.keywords | methylation | en |
dc.subject.keywords | mosaicism | en |
dc.subject.keywords | pharmacokinetics | en |
dc.subject.keywords | protein expression | en |
dc.subject.keywords | randomized controlled trial | en |
dc.subject.keywords | risk assessment | en |
dc.subject.keywords | school child | en |
dc.subject.keywords | transdermal drug administration | en |
dc.subject.keywords | fragile X mental retardation protein | en |
dc.subject.keywords | cannabidiolendogenous compound | en |
dc.subject.keywords | irritability | en |
dc.subject.keywords | placebo | en |
dc.subject.keywords | aberrant behavior checklist | en |
dc.relation.url | https://www.embase.com/search/results?subaction=viewrecord&id=L635848979&from=exporthttp://dx.doi.org/10.1111/jir.12875 | | en |
dc.identifier.risid | 2331 | en |
dc.description.pages | 805 | en |
item.grantfulltext | none | - |
item.openairetype | Article | - |
item.fulltext | No Fulltext | - |
item.languageiso639-1 | en | - |
item.cerifentitytype | Publications | - |
item.openairecristype | http://purl.org/coar/resource_type/c_18cf | - |
Appears in Sites: | Children's Health Queensland Publications |
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