Please use this identifier to cite or link to this item: https://dora.health.qld.gov.au/qldresearchjspui/handle/1/4119
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dc.contributor.authorTartaglia, N.en
dc.contributor.authorBudimirovic, D.en
dc.contributor.authorErickson, C.en
dc.contributor.authorHeussler, Helenen
dc.contributor.authorPalumbo, J.en
dc.contributor.authorTich, N.en
dc.contributor.authorSebree, T.en
dc.contributor.authorCohen, J.en
dc.contributor.authorHagerman, R.en
dc.contributor.authorBerry-Kravis, E.en
dc.date.accessioned2022-11-07T23:49:33Z-
dc.date.available2022-11-07T23:49:33Z-
dc.date.issued2021en
dc.identifier.citation65, (9), 2021, p. 805en
dc.identifier.otherRISen
dc.identifier.urihttp://dora.health.qld.gov.au/qldresearchjspui/handle/1/4119-
dc.description.abstractBackground: 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.isoenen
dc.relation.ispartofJournal of Intellectual Disability Researchen
dc.titleA pivotal study of zyn002 cannabidiol transdermal gel in children and adolescents with fragile x syndrome [connect-fx (ZYN2-cl-016)]en
dc.typeArticleen
dc.identifier.doi10.1111/jir.12875en
dc.subject.keywordsadolescenten
dc.subject.keywordsapplication site painen
dc.subject.keywordsavoidance behavioren
dc.subject.keywordschilden
dc.subject.keywordsClinical Global Impression scaleen
dc.subject.keywordscomparative effectivenessen
dc.subject.keywordsconference abstracten
dc.subject.keywordsdouble blind procedureen
dc.subject.keywordsdrug safetyen
dc.subject.keywordsdrug tolerabilityen
dc.subject.keywordselectrocardiogramen
dc.subject.keywordsfemaleen
dc.subject.keywordsfragile X syndromeen
dc.subject.keywordsgene silencingen
dc.subject.keywordshumanen
dc.subject.keywordsmessenger RNAen
dc.subject.keywordsmajor clinical studyen
dc.subject.keywordsmaleen
dc.subject.keywordsmethylationen
dc.subject.keywordsmosaicismen
dc.subject.keywordspharmacokineticsen
dc.subject.keywordsprotein expressionen
dc.subject.keywordsrandomized controlled trialen
dc.subject.keywordsrisk assessmenten
dc.subject.keywordsschool childen
dc.subject.keywordstransdermal drug administrationen
dc.subject.keywordsfragile X mental retardation proteinen
dc.subject.keywordscannabidiolendogenous compounden
dc.subject.keywordsirritabilityen
dc.subject.keywordsplaceboen
dc.subject.keywordsaberrant behavior checklisten
dc.relation.urlhttps://www.embase.com/search/results?subaction=viewrecord&id=L635848979&from=exporthttp://dx.doi.org/10.1111/jir.12875 |en
dc.identifier.risid2331en
dc.description.pages805en
item.grantfulltextnone-
item.openairetypeArticle-
item.fulltextNo Fulltext-
item.languageiso639-1en-
item.cerifentitytypePublications-
item.openairecristypehttp://purl.org/coar/resource_type/c_18cf-
Appears in Sites:Children's Health Queensland Publications
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