{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,12]],"date-time":"2026-02-12T12:35:11Z","timestamp":1770899711036,"version":"3.50.1"},"reference-count":24,"publisher":"Wiley","license":[{"start":{"date-parts":[[2025,6,26]],"date-time":"2025-06-26T00:00:00Z","timestamp":1750896000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J Paediatrics Child Health"],"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Aim<\/jats:title><jats:p>The aim of this Phase I\/II open\u2010label study was to assess the safety and efficacy of NTI164, a novel full\u2010spectrum medicinal cannabis plant extract 0.08% \u0394\u20109\u2010tetrahydrocannabinol (THC), in Rett syndrome (RTT).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Eleven female participants (5\u201316\u2009years) with a pathogenic variant in the <jats:italic>MECP2<\/jats:italic> gene were recruited to this study, receiving NTI164 twice daily for 12\u2009weeks. The primary outcome measure was the Clinical Global Impression\u2010Improvement (CGI\u2010I) Scale, with secondary outcomes measured using the CGI\u2010Severity (CGI\u2010S), RTT Behaviour Questionnaire (RSBQ), RTT\u2010Symptom Index Score (RTT\u2010SIS), RTT\u2010Domain\u2010Specific Concerns\u2010Visual\u2010Analog Scale (RTT\u2010DSC\u2010VAS), Impact of Childhood Neurological Disability\/Quality of Life (ICND+QoL), and RTT\u2010Caregiver Burden Inventory (RTT\u2010CBI). Paired\u2010samples <jats:italic>t<\/jats:italic>\u2010test was used to assess significance between baseline and Week 12.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Improvements were seen in the total CGI\u2010I score (<jats:italic>p<\/jats:italic>\u2009=\u20090.028), with improvements in communication skills (<jats:italic>p<\/jats:italic>\u2009=\u20090.003), mental alertness (<jats:italic>p<\/jats:italic>\u2009=\u20090.033), socialisation\/eye contact (<jats:italic>p<\/jats:italic>\u2009=\u20090.0004), attentiveness (<jats:italic>p<\/jats:italic>\u2009=\u20090.001), and anxiety (<jats:italic>p<\/jats:italic>\u2009=\u20090.004). CGI\u2010S also demonstrated better outcomes after NTI164 administration (<jats:italic>p<\/jats:italic>\u2009=\u20090.008). RSBQ showed improvements in total score (<jats:italic>p<\/jats:italic>\u2009=\u20090.0005), general mood (<jats:italic>p<\/jats:italic>\u2009=\u20090.0003), breathing problems (<jats:italic>p<\/jats:italic>\u2009=\u20090.041), repetitive face movements (<jats:italic>p<\/jats:italic>\u2009=\u20090.004), and fear\/anxiety (<jats:italic>p<\/jats:italic>\u2009=\u20090.006). RTT\u2010DSC\u2010VAS showed positive developments in abilities to communicate choices (<jats:italic>p<\/jats:italic>\u2009=\u20090.041). ICND total score was improved (<jats:italic>p<\/jats:italic>\u2009=\u20090.003), as well as cognition (<jats:italic>p<\/jats:italic>\u2009=\u20090.027) and Quality of Life (<jats:italic>p<\/jats:italic>\u2009=\u20090.0002). Total score on the RTT\u2010CBI was improved (<jats:italic>p<\/jats:italic>\u2009=\u20090.006).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>NTI164 demonstrated safety and improved some clinical and functional outcomes in RTT. These improvements justify ongoing research into NTI164, which may be a potential adjunct therapy in RTT.<\/jats:p><\/jats:sec>","DOI":"10.1111\/jpc.70122","type":"journal-article","created":{"date-parts":[[2025,6,26]],"date-time":"2025-06-26T10:28:22Z","timestamp":1750933702000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Full\u2010Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An\u00a0Open\u2010Label Study"],"prefix":"10.1111","author":[{"ORCID":"https:\/\/orcid.org\/0009-0002-6444-2628","authenticated-orcid":false,"given":"B. A.","family":"Keating","sequence":"first","affiliation":[{"name":"Fenix Innovation Group Pty Ltd.  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Y.","family":"Ogru","sequence":"additional","affiliation":[{"name":"Fenix Innovation Group Pty Ltd.  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"T. G.","family":"Duthy","sequence":"additional","affiliation":[{"name":"Neurotech International Ltd.  Nedlands Western Australia Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"L.","family":"Douglas","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics The Children\u2019s Hospital at Westmead  Sydney New South Wales Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"K.","family":"Lichkus","sequence":"additional","affiliation":[{"name":"Genetic Metabolic Disorders Service The Children\u2019s Hospital at Westmead  Sydney New South Wales Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"E.","family":"Isikgel","sequence":"additional","affiliation":[{"name":"Fenix Innovation Group Pty Ltd.  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M. 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