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)  

Public ISBD [article]  inJournal of Neurodevelopmental Disorders > 11-1 (December 2019) . - 16 p. 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 [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

Predictors of adaptive functioning in preschool aged children with autism spectrum disorder / M. A. HODGE in Autism Research, 14-7 (July 2021)  

Public ISBD [article]  inAutism Research > 14-7 (July 2021) . - p.1444-1455 Titre : Predictors of adaptive functioning in preschool aged children with autism spectrum disorder Type de document : Texte imprimé et/ou numérique Auteurs : M. A. HODGE, Auteur ; Kelsie A. BOULTON, Auteur ; R. SUTHERLAND, Auteur ; D. BARNETT, Auteur ; B. BENNETT, Auteur ; Esther W. CHAN, Auteur ; J. CRAMSIE, Auteur ; S. DREVENSEK, Auteur ; V. EAPEN, Auteur ; K. GANESALINGAM, Auteur ; A. MASI, Auteur ; N. ONG, Auteur ; M. WILLIAMSZ, Auteur ; A. J. GUASTELLA, Auteur ; N. SILOVE, Auteur Article en page(s) : p.1444-1455 Langues : Anglais (eng) Mots-clés : Autism Spectrum Disorder/complications  Autistic Disorder  Child  Child, Preschool  Cognition  Humans  Motivation  Social Skills  adaptive functioning  autism spectrum disorder  children  cognitive function  preschool  socioeconomic status  symptom severity Index. décimale : PER Périodiques Résumé : Difficulties in adaptive functioning are common in autism spectrum disorder (ASD) and contribute to negative outcomes across the lifespan. Research indicates that cognitive ability is related to degree of adaptive functioning impairments, particularly in young children with ASD. However, the extent to which other factors, such as socioeconomic status (SES) and ASD symptom severity, predict impairments in adaptive functioning remains unclear. The goal of this study was to determine the extent to which SES, ASD symptom severity, and cognitive ability contribute to variability in domain-specific and global components of adaptive functioning in preschool-aged children with ASD. Participants were 99 preschool-aged children (2-6?years) with ASD who attended a tertiary diagnostic service. Results demonstrate that cognitive ability accounted for a significant proportion of variance in domain-specific and global components of adaptive functioning, with higher cognitive ability predicting better adaptive functioning. Results also demonstrate that SES accounted for some variability in domain-specific communication skills and global adaptive functioning when compared to basic demographic factors alone (age and gender). By contrast, ASD symptom severity did not predict variability in domain-specific or global components of adaptive functioning. These findings provide support for a relationship between cognitive ability and adaptive functioning in preschool-aged children with ASD and help to explain specific contributions of verbal and nonverbal ability to adaptive functioning; from this, we can better understand which children are likely to show the greatest degree of impairments across components of adaptive functioning early in development. LAY SUMMARY: People with autism often have difficulties with everyday communication, daily living, and social skills, which are also called adaptive functioning skills. This study investigated factors that might be related to these difficulties in preschoolers with autism. We found that better cognitive ability, but not autism symptoms, were associated with better adaptive functioning. This suggests that interventions for young children with autism should take into account cognitive ability to better understand which children are likely to have difficulties with adaptive functioning. En ligne : http://dx.doi.org/10.1002/aur.2501 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=449 [article] Predictors of adaptive functioning in preschool aged children with autism spectrum disorder [Texte imprimé et/ou numérique] / M. A. HODGE, Auteur ; Kelsie A. BOULTON, Auteur ; R. SUTHERLAND, Auteur ; D. BARNETT, Auteur ; B. BENNETT, Auteur ; Esther W. CHAN, Auteur ; J. CRAMSIE, Auteur ; S. DREVENSEK, Auteur ; V. EAPEN, Auteur ; K. GANESALINGAM, Auteur ; A. MASI, Auteur ; N. ONG, Auteur ; M. WILLIAMSZ, Auteur ; A. J. GUASTELLA, Auteur ; N. SILOVE, Auteur . - p.1444-1455. Langues : Anglais (eng) in Autism Research > 14-7 (July 2021) . - p.1444-1455 Mots-clés : Autism Spectrum Disorder/complications  Autistic Disorder  Child  Child, Preschool  Cognition  Humans  Motivation  Social Skills  adaptive functioning  autism spectrum disorder  children  cognitive function  preschool  socioeconomic status  symptom severity Index. décimale : PER Périodiques Résumé : Difficulties in adaptive functioning are common in autism spectrum disorder (ASD) and contribute to negative outcomes across the lifespan. Research indicates that cognitive ability is related to degree of adaptive functioning impairments, particularly in young children with ASD. However, the extent to which other factors, such as socioeconomic status (SES) and ASD symptom severity, predict impairments in adaptive functioning remains unclear. The goal of this study was to determine the extent to which SES, ASD symptom severity, and cognitive ability contribute to variability in domain-specific and global components of adaptive functioning in preschool-aged children with ASD. Participants were 99 preschool-aged children (2-6?years) with ASD who attended a tertiary diagnostic service. Results demonstrate that cognitive ability accounted for a significant proportion of variance in domain-specific and global components of adaptive functioning, with higher cognitive ability predicting better adaptive functioning. Results also demonstrate that SES accounted for some variability in domain-specific communication skills and global adaptive functioning when compared to basic demographic factors alone (age and gender). By contrast, ASD symptom severity did not predict variability in domain-specific or global components of adaptive functioning. These findings provide support for a relationship between cognitive ability and adaptive functioning in preschool-aged children with ASD and help to explain specific contributions of verbal and nonverbal ability to adaptive functioning; from this, we can better understand which children are likely to show the greatest degree of impairments across components of adaptive functioning early in development. LAY SUMMARY: People with autism often have difficulties with everyday communication, daily living, and social skills, which are also called adaptive functioning skills. This study investigated factors that might be related to these difficulties in preschoolers with autism. We found that better cognitive ability, but not autism symptoms, were associated with better adaptive functioning. This suggests that interventions for young children with autism should take into account cognitive ability to better understand which children are likely to have difficulties with adaptive functioning. En ligne : http://dx.doi.org/10.1002/aur.2501 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=449

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