Access and barriers to supports for children and caregivers attending public child developmental assessment services: Findings from the Sydney child neurodevelopment research registry / Kelsie A. BOULTON in Autism Research, 17-3 (March 2024)  

Public ISBD [article]  inAutism Research > 17-3 (March 2024) . - p.555-567 Titre : Access and barriers to supports for children and caregivers attending public child developmental assessment services: Findings from the Sydney child neurodevelopment research registry Type de document : texte imprimé Auteurs : Kelsie A. BOULTON, Auteur ; Marie-Antoinette HODGE, Auteur ; Kelera LEVU, Auteur ; Natalie ONG, Auteur ; Natalie SILOVE, Auteur ; Adam J. GUASTELLA, Auteur Article en page(s) : p.555-567 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : Abstract Families can spend years waiting for a developmental assessment. We sought to understand supports caregivers had accessed by the time of their child's first multi-disciplinary developmental assessment, the supports caregivers wanted, and the barriers caregivers reported to accessing these supports. We also sought to understand how government funding schemes (the National Disability Insurance Scheme) and sociodemographic factors influenced access to supports. Caregivers were emailed questionnaires on sociodemographic background and intervention history prior to their child's developmental assessment at a tertiary diagnostic and assessment service. Results from 202 caregivers showed most children were receiving less than 2 hours of support each week at assessment. The most common accessed supports were from general practitioners and pediatricians. Caregivers reported behavioral therapists (41%) and psychologists (29%) as the most desired but inaccessible child supports. Half of caregivers nominated a need for parenting interventions. The most frequent barriers to accessing child supports were wait lists, finance, and knowledge. Government funding improved access to the total number of services received (from 2.7 to 5.2 different services), but for disability specific services only (e.g., speech and occupational therapy). Results highlighted disparities for families without government funding, impacting certain groups (e.g., children over 7 years) and services (e.g., mental health). Socioeconomic disadvantage, parental separation, and, surprisingly, being from a non-culturally/linguistically diverse background were associated with fewer services and more barriers. Results highlight the need to facilitate access to supports for families to align with existing recommendations. En ligne : https://doi.org/10.1002/aur.3064 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=525 [article] Access and barriers to supports for children and caregivers attending public child developmental assessment services: Findings from the Sydney child neurodevelopment research registry [texte imprimé] / Kelsie A. BOULTON, Auteur ; Marie-Antoinette HODGE, Auteur ; Kelera LEVU, Auteur ; Natalie ONG, Auteur ; Natalie SILOVE, Auteur ; Adam J. GUASTELLA, Auteur . - p.555-567. Langues : Anglais (eng) in Autism Research > 17-3 (March 2024) . - p.555-567 Index. décimale : PER Périodiques Résumé : Abstract Families can spend years waiting for a developmental assessment. We sought to understand supports caregivers had accessed by the time of their child's first multi-disciplinary developmental assessment, the supports caregivers wanted, and the barriers caregivers reported to accessing these supports. We also sought to understand how government funding schemes (the National Disability Insurance Scheme) and sociodemographic factors influenced access to supports. Caregivers were emailed questionnaires on sociodemographic background and intervention history prior to their child's developmental assessment at a tertiary diagnostic and assessment service. Results from 202 caregivers showed most children were receiving less than 2 hours of support each week at assessment. The most common accessed supports were from general practitioners and pediatricians. Caregivers reported behavioral therapists (41%) and psychologists (29%) as the most desired but inaccessible child supports. Half of caregivers nominated a need for parenting interventions. The most frequent barriers to accessing child supports were wait lists, finance, and knowledge. Government funding improved access to the total number of services received (from 2.7 to 5.2 different services), but for disability specific services only (e.g., speech and occupational therapy). Results highlighted disparities for families without government funding, impacting certain groups (e.g., children over 7 years) and services (e.g., mental health). Socioeconomic disadvantage, parental separation, and, surprisingly, being from a non-culturally/linguistically diverse background were associated with fewer services and more barriers. Results highlight the need to facilitate access to supports for families to align with existing recommendations. En ligne : https://doi.org/10.1002/aur.3064 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=525

Caregiver-identified strengths in children attending their first neurodevelopmental assessment: Findings from the Sydney child development research registry and development of a child strengths checklist / Lorna HANKIN ; Marie-Antoinette HODGE ; Natalie ONG ; Natalie SILOVE ; Adam J. GUASTELLA in Autism, 29-7 (July 2025)  

Public ISBD [article]  inAutism > 29-7 (July 2025) . - p.1769-1781 Titre : Caregiver-identified strengths in children attending their first neurodevelopmental assessment: Findings from the Sydney child development research registry and development of a child strengths checklist Type de document : texte imprimé Auteurs : Lorna HANKIN, Auteur ; Marie-Antoinette HODGE, Auteur ; Natalie ONG, Auteur ; Natalie SILOVE, Auteur ; Adam J. GUASTELLA, Auteur Article en page(s) : p.1769-1781 Langues : Anglais (eng) Mots-clés : assessment  autism spectrum disorders  neurodevelopment  ADHD  communication disorders  strengths Index. décimale : PER Périodiques Résumé : There has been a growing focus on the importance of understanding strengths in children with neurodevelopmental conditions and how such knowledge can support clinical practices. However, limited research has explored systematic reports of strengths from caregivers of children with neurodevelopmental conditions, most commonly autism. In this study, we explored caregiver-identified strengths in children attending their first neurodevelopmental assessment. Caregivers of 686 children attending a tertiary assessment service answered a survey question about their children s strengths. Content analysis identified 61 unique categories of strengths, which were grouped into six themes: cognitive and intellectual, social and interpersonal, hobbies and passions, character and personality, physical, and behavioural. The most frequently reported specific strengths were kind caring, and compassionate, social and friendly, loving and affectionate, music singing and dancing, and good memory recall. The breadth of positive strengths identified here may reflect the larger population sampled or the diversity of presentations in this cohort. We then present a caregiver checklist, the Child Autism and Neurodevelopment Strengths (CANS) Checklist, that was developed with community representatives, to inform assessment and feedback of child strengths. We discuss what is required to use this knowledge to inform strengths-based practices that can support clinical practice and inform on child development and family well-being.Lay Abstract There has been a growing focus on the importance of understanding strengths in children with neurodevelopmental conditions, but there is little research exploring caregiver-reported strengths at the time of diagnostic assessment. In this study, we explored caregiver-identified strengths in 686 children who were attending a neurodevelopmental assessment. Content analysis identified 61 unique categories of strengths, which we grouped into six main themes. These six themes were cognitive and intellectual, social and interpersonal, hobbies and passions, character and personality, physical, and behavioural. The most common strengths identified by caregivers were 'kind, caring, and compassionate", 'social and friendly", 'loving and affectionate", 'music, singing, and dancing", and 'good memory recall". Based on these strengths, we present a checklist that was developed with community representatives, to make sure a strengths-based framework can be used during the diagnostic process. We discuss how we can use this knowledge to develop strengths-based practices that can support clinical practice and inform on child development and family well-being. En ligne : https://dx.doi.org/10.1177/13623613251325287 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=558 [article] Caregiver-identified strengths in children attending their first neurodevelopmental assessment: Findings from the Sydney child development research registry and development of a child strengths checklist [texte imprimé] / Lorna HANKIN, Auteur ; Marie-Antoinette HODGE, Auteur ; Natalie ONG, Auteur ; Natalie SILOVE, Auteur ; Adam J. GUASTELLA, Auteur . - p.1769-1781. Langues : Anglais (eng) in Autism > 29-7 (July 2025) . - p.1769-1781 Mots-clés : assessment  autism spectrum disorders  neurodevelopment  ADHD  communication disorders  strengths Index. décimale : PER Périodiques Résumé : There has been a growing focus on the importance of understanding strengths in children with neurodevelopmental conditions and how such knowledge can support clinical practices. However, limited research has explored systematic reports of strengths from caregivers of children with neurodevelopmental conditions, most commonly autism. In this study, we explored caregiver-identified strengths in children attending their first neurodevelopmental assessment. Caregivers of 686 children attending a tertiary assessment service answered a survey question about their children s strengths. Content analysis identified 61 unique categories of strengths, which were grouped into six themes: cognitive and intellectual, social and interpersonal, hobbies and passions, character and personality, physical, and behavioural. The most frequently reported specific strengths were kind caring, and compassionate, social and friendly, loving and affectionate, music singing and dancing, and good memory recall. The breadth of positive strengths identified here may reflect the larger population sampled or the diversity of presentations in this cohort. We then present a caregiver checklist, the Child Autism and Neurodevelopment Strengths (CANS) Checklist, that was developed with community representatives, to inform assessment and feedback of child strengths. We discuss what is required to use this knowledge to inform strengths-based practices that can support clinical practice and inform on child development and family well-being.Lay Abstract There has been a growing focus on the importance of understanding strengths in children with neurodevelopmental conditions, but there is little research exploring caregiver-reported strengths at the time of diagnostic assessment. In this study, we explored caregiver-identified strengths in 686 children who were attending a neurodevelopmental assessment. Content analysis identified 61 unique categories of strengths, which we grouped into six main themes. These six themes were cognitive and intellectual, social and interpersonal, hobbies and passions, character and personality, physical, and behavioural. The most common strengths identified by caregivers were 'kind, caring, and compassionate", 'social and friendly", 'loving and affectionate", 'music, singing, and dancing", and 'good memory recall". Based on these strengths, we present a checklist that was developed with community representatives, to make sure a strengths-based framework can be used during the diagnostic process. We discuss how we can use this knowledge to develop strengths-based practices that can support clinical practice and inform on child development and family well-being. En ligne : https://dx.doi.org/10.1177/13623613251325287 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=558

Focusing on autism symptoms masks sex-specific needs of autistic children: An example from the Sydney Child Neurodevelopment Research Registry / Marie-Antoinette HODGE in Autism, 29-5 (May 2025)  

Public ISBD [article]  inAutism > 29-5 (May 2025) . - p.1318-1332 Titre : Focusing on autism symptoms masks sex-specific needs of autistic children: An example from the Sydney Child Neurodevelopment Research Registry Type de document : texte imprimé Auteurs : Marie-Antoinette HODGE, Auteur ; Rebecca SUTHERLAND, Auteur ; Kelsie A. BOULTON, Auteur ; Sarah Jane BARACZ, Auteur ; Natalie ONG, Auteur ; Beverley BENNETT, Auteur ; Adam J. GUASTELLA, Auteur ; Natalie SILOVE, Auteur Article en page(s) : p.1318-1332 Langues : Anglais (eng) Mots-clés : adaptive skills  age at diagnosis  assigned sex at birth  autism symptoms  developmental skills  intellectual ability Index. décimale : PER Périodiques Résumé : Studies have shown that there are differences between the presentations of males and females diagnosed with autism. There remains a developing understanding about how the presentation of autism differs between boys (hereafter referred to as 'assigned males at birth') and girls (assigned females at birth). This study sought to investigate the presence of sex differences in autistic children. Participants (1.11-17.97 years) attended an assessment clinic and participated in measures of intelligence/development, social/communication skills and behaviour. Adaptive skills were evaluated using a range of standardised measures, and other clinical and demographic variables were collected (e.g. age, intelligence quotient, ratio of male to female). Assigned males at birth displayed more autism characteristics and greater symptom autism severity than assigned females at birth. No significant differences were found between assigned males at birth and assigned females at birth on any measure of intelligence. Children assigned males at birth received assessments 6 months earlier than children assigned females at birth on average. Externalising behaviour problems were more evident in assigned males at birth, but statistically significant differences in adaptive skills were not apparent between assigned males at birth and assigned females at birth. This study showed assigned females at birth and assigned males at birth differ in autism symptoms and severity and age at diagnosis based on a real-world sample. It highlights the importance of balancing assessments of symptoms with assessment of adaptive function.Lay abstract Studies have shown that there is a difference between biological sex at birth in autism spectrum disorder. There remains a lack of understanding about how the symptoms of autism differ between assigned males at birth and assigned females at birth. We looked at the presence of sex differences in a large group of autistic toddlers, children and adolescents, who were seen in a large diagnosis and assessment clinic. They participated in measures of intelligence/development, social/communication skills and behaviour. Their adaptive skills were evaluated and other clinical and information were collected. Assigned males at birth displayed more autism characteristics and greater symptom autism severity than assigned females at birth. There were no statistically significant differences between assigned males at birth and assigned females at birth on any measure of intellectual assessment. Assigned females at birth showed better nonverbal performance than assigned males at birth on formal developmental assessments. Children assigned males at birth received assessments 6 months earlier than children assigned females at birth. Externalising behaviour problems were more evident in assigned males at birth. This study provides evidence to show that autistic children assigned females at birth and assigned males at birth differ in terms of autism symptoms and severity and age at diagnosis based on a sample recruited in a real-world clinic. It highlights the importance of the growing debate between balancing assessments of symptoms with assessment of adaptive function. En ligne : https://dx.doi.org/10.1177/13623613241303550 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=555 [article] Focusing on autism symptoms masks sex-specific needs of autistic children: An example from the Sydney Child Neurodevelopment Research Registry [texte imprimé] / Marie-Antoinette HODGE, Auteur ; Rebecca SUTHERLAND, Auteur ; Kelsie A. BOULTON, Auteur ; Sarah Jane BARACZ, Auteur ; Natalie ONG, Auteur ; Beverley BENNETT, Auteur ; Adam J. GUASTELLA, Auteur ; Natalie SILOVE, Auteur . - p.1318-1332. Langues : Anglais (eng) in Autism > 29-5 (May 2025) . - p.1318-1332 Mots-clés : adaptive skills  age at diagnosis  assigned sex at birth  autism symptoms  developmental skills  intellectual ability Index. décimale : PER Périodiques Résumé : Studies have shown that there are differences between the presentations of males and females diagnosed with autism. There remains a developing understanding about how the presentation of autism differs between boys (hereafter referred to as 'assigned males at birth') and girls (assigned females at birth). This study sought to investigate the presence of sex differences in autistic children. Participants (1.11-17.97 years) attended an assessment clinic and participated in measures of intelligence/development, social/communication skills and behaviour. Adaptive skills were evaluated using a range of standardised measures, and other clinical and demographic variables were collected (e.g. age, intelligence quotient, ratio of male to female). Assigned males at birth displayed more autism characteristics and greater symptom autism severity than assigned females at birth. No significant differences were found between assigned males at birth and assigned females at birth on any measure of intelligence. Children assigned males at birth received assessments 6 months earlier than children assigned females at birth on average. Externalising behaviour problems were more evident in assigned males at birth, but statistically significant differences in adaptive skills were not apparent between assigned males at birth and assigned females at birth. This study showed assigned females at birth and assigned males at birth differ in autism symptoms and severity and age at diagnosis based on a real-world sample. It highlights the importance of balancing assessments of symptoms with assessment of adaptive function.Lay abstract Studies have shown that there is a difference between biological sex at birth in autism spectrum disorder. There remains a lack of understanding about how the symptoms of autism differ between assigned males at birth and assigned females at birth. We looked at the presence of sex differences in a large group of autistic toddlers, children and adolescents, who were seen in a large diagnosis and assessment clinic. They participated in measures of intelligence/development, social/communication skills and behaviour. Their adaptive skills were evaluated and other clinical and information were collected. Assigned males at birth displayed more autism characteristics and greater symptom autism severity than assigned females at birth. There were no statistically significant differences between assigned males at birth and assigned females at birth on any measure of intellectual assessment. Assigned females at birth showed better nonverbal performance than assigned males at birth on formal developmental assessments. Children assigned males at birth received assessments 6 months earlier than children assigned females at birth. Externalising behaviour problems were more evident in assigned males at birth. This study provides evidence to show that autistic children assigned females at birth and assigned males at birth differ in terms of autism symptoms and severity and age at diagnosis based on a sample recruited in a real-world clinic. It highlights the importance of the growing debate between balancing assessments of symptoms with assessment of adaptive function. En ligne : https://dx.doi.org/10.1177/13623613241303550 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=555

Interventions used with an Australian sample of preschool children with autism spectrum disorders / Mark CARTER in Research in Autism Spectrum Disorders, 5-3 (July-September 2011)  

Public ISBD [article]  inResearch in Autism Spectrum Disorders > 5-3 (July-September 2011) . - p.1033-1041 Titre : Interventions used with an Australian sample of preschool children with autism spectrum disorders Type de document : texte imprimé Auteurs : Mark CARTER, Auteur ; Jacqueline ROBERTS, Auteur ; Katrina WILLIAMS, Auteur ; David EVANS, Auteur ; Trevor R. PARMENTER, Auteur ; Natalie SILOVE, Auteur ; Trevor CLARK, Auteur ; Anthony WARREN, Auteur Année de publication : 2011 Article en page(s) : p.1033-1041 Langues : Anglais (eng) Mots-clés : Autism  Parents  Survey  Treatment  Education  Complementary and alternative medicine Index. décimale : PER Périodiques Résumé : This study examined the previous and current range of educational, therapy, medical and CAM interventions used by a clearly described Australian sample of 84 families of preschool-aged children with autism spectrum disorders who were enrolled in a controlled trial of early intervention services. With regard to educational and therapy interventions, the most frequently used services were speech–language pathology, preschool and childcare, generic early intervention, and occupational therapy. With the exception of preschool and childcare, the access frequency for most of these services indicated they were used at relatively low intensity. Exclusion diets, oils/fatty acids and vitamin and mineral supplements were the primary CAM interventions used by families. There was no clear evidence of a relationship between the number of interventions used by families and developmental status although this may have been due to the relatively recent diagnoses. Implications of these findings and directions for future research are discussed. En ligne : http://dx.doi.org/10.1016/j.rasd.2010.11.009 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=119 [article] Interventions used with an Australian sample of preschool children with autism spectrum disorders [texte imprimé] / Mark CARTER, Auteur ; Jacqueline ROBERTS, Auteur ; Katrina WILLIAMS, Auteur ; David EVANS, Auteur ; Trevor R. PARMENTER, Auteur ; Natalie SILOVE, Auteur ; Trevor CLARK, Auteur ; Anthony WARREN, Auteur . - 2011 . - p.1033-1041. Langues : Anglais (eng) in Research in Autism Spectrum Disorders > 5-3 (July-September 2011) . - p.1033-1041 Mots-clés : Autism  Parents  Survey  Treatment  Education  Complementary and alternative medicine Index. décimale : PER Périodiques Résumé : This study examined the previous and current range of educational, therapy, medical and CAM interventions used by a clearly described Australian sample of 84 families of preschool-aged children with autism spectrum disorders who were enrolled in a controlled trial of early intervention services. With regard to educational and therapy interventions, the most frequently used services were speech–language pathology, preschool and childcare, generic early intervention, and occupational therapy. With the exception of preschool and childcare, the access frequency for most of these services indicated they were used at relatively low intensity. Exclusion diets, oils/fatty acids and vitamin and mineral supplements were the primary CAM interventions used by families. There was no clear evidence of a relationship between the number of interventions used by families and developmental status although this may have been due to the relatively recent diagnoses. Implications of these findings and directions for future research are discussed. En ligne : http://dx.doi.org/10.1016/j.rasd.2010.11.009 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=119

Long-term safety and tolerability of transdermal cannabidiol gel in children and adolescents with Fragile X syndrome (ZYN2-CL-017): an interim analysis of an ongoing open-label extension study / Elizabeth BERRY-KRAVIS in Journal of Neurodevelopmental Disorders, 17 (2025)  

Public ISBD [article]  inJournal of Neurodevelopmental Disorders > 17 (2025) Titre : Long-term safety and tolerability of transdermal cannabidiol gel in children and adolescents with Fragile X syndrome (ZYN2-CL-017): an interim analysis of an ongoing open-label extension study Type de document : texte imprimé Auteurs : Elizabeth BERRY-KRAVIS, Auteur ; Randi HAGERMAN, Auteur ; Jonathan COHEN, Auteur ; Dejan BUDIMIROVIC, Auteur ; Caroline B. BUCHANAN, Auteur ; Natalie SILOVE, Auteur ; Nancy TICH, Auteur ; Anthony THIBODEAU, Auteur ; Thomas DOBBINS, Auteur ; Terri SEBREE, Auteur ; Stephen O'QUINN, Auteur ; David S. ALBERS, Auteur ; Kristen G. BZDEK, Auteur ; George NOMIKOS, Auteur ; Kumar BUDUR, Auteur Langues : Anglais (eng) Mots-clés : Humans  Fragile X Syndrome/drug therapy  Adolescent  Child  Male  Administration, Cutaneous  Cannabidiol/administration & dosage/adverse effects  Female  Gels  Child, Preschool  Endocannabinoid system  Fragile x syndrome  Irritability  Social avoidance  Transdermal cannabidiol  conducted in accordance with the ethical principles outlined in the Declaration  of Helsinki and was approved by Advarra, Inc. Institutional Review Board (IRB  Approval Number: Pro00060799. Consent for publication: Not applicable. Competing  interests: EB-K, RH, JC, DB, CBB, and NS have received funding from Harmony  Biosciences for the conduct of this trial as investigators. TD, TS, and SOQ are  paid consultants of Harmony Biosciences. CBB is a paid consultant of Acadia  Pharmaceuticals. NT, AT, DSA, KGB, GN, and KB are employees of Harmony  Biosciences. Index. décimale : PER Périodiques Résumé : BACKGROUND: Dysregulated endocannabinoid signaling is involved in Fragile X syndrome (FXS), suggesting a potential role for the endocannabinoid signaling modulator, cannabidiol, in treatment. ZYN002 is a synthetic cannabidiol that has been uniquely formulated as a gel for transdermal delivery and is currently under investigation for the treatment of behavioral symptoms associated with FXS. DESIGN: ZYN2-CL-017 is an ongoing, long-term, open-label extension (OLE) safety trial of ZYN002 in patients with FXS. We are enrolling patients from past and current ZYN002 clinical trials to evaluate the safety and tolerability of ZYN002 in patients with FXS. METHODS: Primary safety assessments were conducted in patients who enrolled into the OLE from 2 completed ZYN002 trials. Secondary analyses, conducted in a subgroup enrolled from a completed placebo-controlled trial of ZYN002, included the FXS-specific Aberrant Behavior Checklist-Community Social Avoidance and Irritability subscales (ABC-C(FXS) SA and ABC-C(FXS) Irr, examined change from baseline of the randomized study) and the Caregiver Global Impression of Change (CaGI-C, examined change from baseline of the OLE), in which caregivers were asked to rate the change in their child's overall behavior. RESULTS: At the time of this interim analysis data cut (January 31, 2024), 240 patients had been enrolled from 2 completed ZYN002 trials. Mean age at entry to the OLE was 9.7 years (range 3-17 years), and the majority were male (76.3%) and White (80.4%). Mean exposure to ZYN002 during the initial trials and OLE was 28 months. Treatment-related adverse events (AEs) were reported in 12.9% of patients; the most common (6.7% of patients) was short-term application site pain. The highest degree of skin irritation reported by investigators was moderate erythema in 7 patients (2.9%). In the secondary analysis cohort (n=196 evaluable patients), patients demonstrated clinically meaningful changes in ABC-C(FXS) SA, ABC-C(FXS) Irr, and CaGI-C scores. CONCLUSIONS: Interim analysis results of the ongoing OLE in children, adolescents, and young adults with FXS demonstrated that ZYN002 has a favorable long-term safety profile and is generally well tolerated. Clinically meaningful changes in behaviors from baseline continued to be observed during the OLE. These findings support further study of ZYN002 in patients with FXS. TRIAL REGISTRATION: ZYN2-CL-017 is registered on Clinicaltrials.gov (NCT03802799) on December 26, 2018. En ligne : https://dx.doi.org/10.1186/s11689-025-09657-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=576 [article] Long-term safety and tolerability of transdermal cannabidiol gel in children and adolescents with Fragile X syndrome (ZYN2-CL-017): an interim analysis of an ongoing open-label extension study [texte imprimé] / Elizabeth BERRY-KRAVIS, Auteur ; Randi HAGERMAN, Auteur ; Jonathan COHEN, Auteur ; Dejan BUDIMIROVIC, Auteur ; Caroline B. BUCHANAN, Auteur ; Natalie SILOVE, Auteur ; Nancy TICH, Auteur ; Anthony THIBODEAU, Auteur ; Thomas DOBBINS, Auteur ; Terri SEBREE, Auteur ; Stephen O'QUINN, Auteur ; David S. ALBERS, Auteur ; Kristen G. BZDEK, Auteur ; George NOMIKOS, Auteur ; Kumar BUDUR, Auteur. Langues : Anglais (eng) in Journal of Neurodevelopmental Disorders > 17 (2025) Mots-clés : Humans  Fragile X Syndrome/drug therapy  Adolescent  Child  Male  Administration, Cutaneous  Cannabidiol/administration & dosage/adverse effects  Female  Gels  Child, Preschool  Endocannabinoid system  Fragile x syndrome  Irritability  Social avoidance  Transdermal cannabidiol  conducted in accordance with the ethical principles outlined in the Declaration  of Helsinki and was approved by Advarra, Inc. Institutional Review Board (IRB  Approval Number: Pro00060799. Consent for publication: Not applicable. Competing  interests: EB-K, RH, JC, DB, CBB, and NS have received funding from Harmony  Biosciences for the conduct of this trial as investigators. TD, TS, and SOQ are  paid consultants of Harmony Biosciences. CBB is a paid consultant of Acadia  Pharmaceuticals. NT, AT, DSA, KGB, GN, and KB are employees of Harmony  Biosciences. Index. décimale : PER Périodiques Résumé : BACKGROUND: Dysregulated endocannabinoid signaling is involved in Fragile X syndrome (FXS), suggesting a potential role for the endocannabinoid signaling modulator, cannabidiol, in treatment. ZYN002 is a synthetic cannabidiol that has been uniquely formulated as a gel for transdermal delivery and is currently under investigation for the treatment of behavioral symptoms associated with FXS. DESIGN: ZYN2-CL-017 is an ongoing, long-term, open-label extension (OLE) safety trial of ZYN002 in patients with FXS. We are enrolling patients from past and current ZYN002 clinical trials to evaluate the safety and tolerability of ZYN002 in patients with FXS. METHODS: Primary safety assessments were conducted in patients who enrolled into the OLE from 2 completed ZYN002 trials. Secondary analyses, conducted in a subgroup enrolled from a completed placebo-controlled trial of ZYN002, included the FXS-specific Aberrant Behavior Checklist-Community Social Avoidance and Irritability subscales (ABC-C(FXS) SA and ABC-C(FXS) Irr, examined change from baseline of the randomized study) and the Caregiver Global Impression of Change (CaGI-C, examined change from baseline of the OLE), in which caregivers were asked to rate the change in their child's overall behavior. RESULTS: At the time of this interim analysis data cut (January 31, 2024), 240 patients had been enrolled from 2 completed ZYN002 trials. Mean age at entry to the OLE was 9.7 years (range 3-17 years), and the majority were male (76.3%) and White (80.4%). Mean exposure to ZYN002 during the initial trials and OLE was 28 months. Treatment-related adverse events (AEs) were reported in 12.9% of patients; the most common (6.7% of patients) was short-term application site pain. The highest degree of skin irritation reported by investigators was moderate erythema in 7 patients (2.9%). In the secondary analysis cohort (n=196 evaluable patients), patients demonstrated clinically meaningful changes in ABC-C(FXS) SA, ABC-C(FXS) Irr, and CaGI-C scores. CONCLUSIONS: Interim analysis results of the ongoing OLE in children, adolescents, and young adults with FXS demonstrated that ZYN002 has a favorable long-term safety profile and is generally well tolerated. Clinically meaningful changes in behaviors from baseline continued to be observed during the OLE. These findings support further study of ZYN002 in patients with FXS. TRIAL REGISTRATION: ZYN2-CL-017 is registered on Clinicaltrials.gov (NCT03802799) on December 26, 2018. En ligne : https://dx.doi.org/10.1186/s11689-025-09657-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=576

Non-pharmacological interventions for autistic children: An umbrella review / David TREMBATH in Autism, 27-2 (February 2023)  

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Patterns of sensory modulation by age and sex in young people on the autism spectrum / Alison E. LANE in Autism Research, 15-10 (October 2022)  

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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 / Helen S. HEUSSLER in Journal of Neurodevelopmental Disorders, 11-1 (December 2019)  

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

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Quality and accessibility of written development assessment reports provided to caregivers in a publicly funded child developmental assessment service / Martha MUNRO in Autism, 27-6 (August 2023)  

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A randomised controlled trial of two early intervention programs for young children with autism: Centre-based with parent program and home-based / Jacqueline ROBERTS in Research in Autism Spectrum Disorders, 5-4 (October-December 2011)  

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Screen to Screen Versus Face to Face: Evaluating Telehealth Autism Diagnostic Assessments for Young Children in a Diverse Clinical Setting / Rebecca SUTHERLAND in Autism Research, 18-10 (October 2025)  

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Validity of the Social Communication Questionnaire in Assessing Risk of Autism in Preschool Children with Developmental Problems / C. Wendy ALLEN in Journal of Autism and Developmental Disorders, 37-7 (August 2007)  

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