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Auteur C. O'NEILL |
Documents disponibles écrits par cet auteur (2)
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A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome / H. HEUSSLER in Journal of Neurodevelopmental Disorders, 11-1 (December 2019)
[article]
Titre : A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome Type de document : Texte imprimé et/ou numérique Auteurs : H. HEUSSLER, Auteur ; J. COHEN, Auteur ; N. SILOVE, Auteur ; N. TICH, Auteur ; M. O. BONN-MILLER, Auteur ; W. DU, Auteur ; C. O'NEILL, Auteur ; T. SEBREE, Auteur Article en page(s) : 16 p. Langues : Anglais (eng) Mots-clés : Cannabidiol Fragile X Pediatric Transdermal Zyn002 Zynerba Index. décimale : PER Périodiques Résumé : BACKGROUND: Fragile X syndrome (FXS) is characterized by a range of developmental, neuropsychiatric, and behavioral symptoms that cause significant impairment in those with the disorder. Cannabidiol (CBD) holds promise as a potential treatment for FXS symptoms due to its safety profile and positive effects on a number of emotional and behavioral symptoms associated with FXS. The aim of the current study was to evaluate the safety, tolerability, and initial efficacy of ZYN002, a transdermal CBD gel, in a pediatric population with FXS. METHODS: Twenty children and adolescents (aged 6-17 years) with a diagnosis of FXS (confirmed through molecular documentation of FMR1 full mutation) were enrolled in an open-label, multi-site, trial of ZYN002. Transdermal CBD gel was administered twice daily for 12 weeks, titrated from 50 mg to a maximum daily dose of 250 mg. The primary efficacy endpoint was change from screening to week 12 on the Anxiety, Depression, and Mood Scale (ADAMS). Secondary endpoint measures included the Aberrant Behavior Checklist-Community for FXS (ABC-CFXS), Pediatric Anxiety Rating Scale (PARS-R), Pediatric Quality of Life Inventory (PedsQL), three Visual Analogue Scales (VAS), and the Clinical Global Impression Scale-Severity (CGI-S) and Improvement (CGI-I). RESULTS: The majority of treatment-emergent AEs (reported by 85% of participants) were mild in severity (70%), and no serious adverse events were reported. There was a statistically significant reduction in ADAMS total score from screening to week 12 and significant reductions on nearly all other secondary endpoints, including all ADAMS subscales (except depressed mood), all ABC-CFXS subscale scores (e.g., social avoidance, irritability), PARS-R total severity score, and PedsQL total score. CONCLUSIONS: ZYN002 was well tolerated and produced clinically meaningful reductions in anxiety and behavioral symptoms in children and adolescents with FXS. These findings support further study of ZYN002 in a randomized, well-controlled trial for the treatment of behavioral symptoms of FXS. TRIAL REGISTRATION: ANZCTR, ACTRN12617000150347 Registered 27 January 2017. En ligne : https://dx.doi.org/10.1186/s11689-019-9277-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=409
in Journal of Neurodevelopmental Disorders > 11-1 (December 2019) . - 16 p.[article] A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome [Texte imprimé et/ou numérique] / H. HEUSSLER, Auteur ; J. COHEN, Auteur ; N. SILOVE, Auteur ; N. TICH, Auteur ; M. O. BONN-MILLER, Auteur ; W. DU, Auteur ; C. O'NEILL, Auteur ; T. SEBREE, Auteur . - 16 p.
Langues : Anglais (eng)
in Journal of Neurodevelopmental Disorders > 11-1 (December 2019) . - 16 p.
Mots-clés : Cannabidiol Fragile X Pediatric Transdermal Zyn002 Zynerba Index. décimale : PER Périodiques Résumé : BACKGROUND: Fragile X syndrome (FXS) is characterized by a range of developmental, neuropsychiatric, and behavioral symptoms that cause significant impairment in those with the disorder. Cannabidiol (CBD) holds promise as a potential treatment for FXS symptoms due to its safety profile and positive effects on a number of emotional and behavioral symptoms associated with FXS. The aim of the current study was to evaluate the safety, tolerability, and initial efficacy of ZYN002, a transdermal CBD gel, in a pediatric population with FXS. METHODS: Twenty children and adolescents (aged 6-17 years) with a diagnosis of FXS (confirmed through molecular documentation of FMR1 full mutation) were enrolled in an open-label, multi-site, trial of ZYN002. Transdermal CBD gel was administered twice daily for 12 weeks, titrated from 50 mg to a maximum daily dose of 250 mg. The primary efficacy endpoint was change from screening to week 12 on the Anxiety, Depression, and Mood Scale (ADAMS). Secondary endpoint measures included the Aberrant Behavior Checklist-Community for FXS (ABC-CFXS), Pediatric Anxiety Rating Scale (PARS-R), Pediatric Quality of Life Inventory (PedsQL), three Visual Analogue Scales (VAS), and the Clinical Global Impression Scale-Severity (CGI-S) and Improvement (CGI-I). RESULTS: The majority of treatment-emergent AEs (reported by 85% of participants) were mild in severity (70%), and no serious adverse events were reported. There was a statistically significant reduction in ADAMS total score from screening to week 12 and significant reductions on nearly all other secondary endpoints, including all ADAMS subscales (except depressed mood), all ABC-CFXS subscale scores (e.g., social avoidance, irritability), PARS-R total severity score, and PedsQL total score. CONCLUSIONS: ZYN002 was well tolerated and produced clinically meaningful reductions in anxiety and behavioral symptoms in children and adolescents with FXS. These findings support further study of ZYN002 in a randomized, well-controlled trial for the treatment of behavioral symptoms of FXS. TRIAL REGISTRATION: ANZCTR, ACTRN12617000150347 Registered 27 January 2017. En ligne : https://dx.doi.org/10.1186/s11689-019-9277-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=409 The economic costs and its predictors for childhood autism spectrum disorders in Ireland: How is the burden distributed? / A. RODDY in Autism, 23-5 (July 2019)
[article]
Titre : The economic costs and its predictors for childhood autism spectrum disorders in Ireland: How is the burden distributed? Type de document : Texte imprimé et/ou numérique Auteurs : A. RODDY, Auteur ; C. O'NEILL, Auteur Article en page(s) : p.1106-1118 Langues : Anglais (eng) Mots-clés : autism spectrum disorders cost shifting economic burden service utilization Index. décimale : PER Périodiques Résumé : Autism spectrum disorders are associated with a substantial economic burden; although little is known about the relationship between state and family out-of-pocket expenditure. The objective of this study is to estimate the societal cost of childhood autism spectrum disorders and explain the variation in costs between state and family out-of-pocket expenditure. A bottom-up prevalence based cost-of-illness methodology was implemented using data from a combination of multiple convenience samples in Ireland of 195 parents of 222 children aged between 2 and 18 years of age with a clinically diagnosed autism spectrum disorder collected in 2014/2015. The findings show the average annual cost per child for families amounted to euro28,464.89 related to private autism spectrum disorder services, lost income and informal care. By comparison, annual state expenditure per child on autism spectrum disorder-related health, social and educational resources was euro14,192. Regression analyses indicate that autism spectrum disorder severity is significantly associated with higher out of pocket expenditures but not state health expenditures. The results suggest that parents are central to meeting the needs of young people with autism spectrum disorders in Ireland. Policy implications of these findings suggest that significant investment and commitment is needed to address the needs of individuals living with autism spectrum disorders and their families. En ligne : http://dx.doi.org/10.1177/1362361318801586 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=401
in Autism > 23-5 (July 2019) . - p.1106-1118[article] The economic costs and its predictors for childhood autism spectrum disorders in Ireland: How is the burden distributed? [Texte imprimé et/ou numérique] / A. RODDY, Auteur ; C. O'NEILL, Auteur . - p.1106-1118.
Langues : Anglais (eng)
in Autism > 23-5 (July 2019) . - p.1106-1118
Mots-clés : autism spectrum disorders cost shifting economic burden service utilization Index. décimale : PER Périodiques Résumé : Autism spectrum disorders are associated with a substantial economic burden; although little is known about the relationship between state and family out-of-pocket expenditure. The objective of this study is to estimate the societal cost of childhood autism spectrum disorders and explain the variation in costs between state and family out-of-pocket expenditure. A bottom-up prevalence based cost-of-illness methodology was implemented using data from a combination of multiple convenience samples in Ireland of 195 parents of 222 children aged between 2 and 18 years of age with a clinically diagnosed autism spectrum disorder collected in 2014/2015. The findings show the average annual cost per child for families amounted to euro28,464.89 related to private autism spectrum disorder services, lost income and informal care. By comparison, annual state expenditure per child on autism spectrum disorder-related health, social and educational resources was euro14,192. Regression analyses indicate that autism spectrum disorder severity is significantly associated with higher out of pocket expenditures but not state health expenditures. The results suggest that parents are central to meeting the needs of young people with autism spectrum disorders in Ireland. Policy implications of these findings suggest that significant investment and commitment is needed to address the needs of individuals living with autism spectrum disorders and their families. En ligne : http://dx.doi.org/10.1177/1362361318801586 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=401