Adaptive Behavior in Young Autistic Children: Associations with Irritability and ADHD Symptoms / Naomi O. DAVIS ; Marina SPANOS ; Maura SABATOS-DEVITO ; Rachel AIELLO ; Grace T. BARANEK ; Scott N. COMPTON ; Helen L. EGGER ; Lauren FRANZ ; Soo-Jeong KIM ; Bryan H. KING ; Alexander KOLEVZON ; Christopher J. MCDOUGLE ; Kevin SANDERS ; Jeremy VEENSTRA-VANDERWEELE ; Linmarie SIKICH ; Scott H. KOLLINS ; Geraldine DAWSON in Journal of Autism and Developmental Disorders, 54-9 (September 2024)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 54-9 (September 2024) . - p.3559-3566 Titre : Adaptive Behavior in Young Autistic Children: Associations with Irritability and ADHD Symptoms Type de document : Texte imprimé et/ou numérique Auteurs : Naomi O. DAVIS, Auteur ; Marina SPANOS, Auteur ; Maura SABATOS-DEVITO, Auteur ; Rachel AIELLO, Auteur ; Grace T. BARANEK, Auteur ; Scott N. COMPTON, Auteur ; Helen L. EGGER, Auteur ; Lauren FRANZ, Auteur ; Soo-Jeong KIM, Auteur ; Bryan H. KING, Auteur ; Alexander KOLEVZON, Auteur ; Christopher J. MCDOUGLE, Auteur ; Kevin SANDERS, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur ; Linmarie SIKICH, Auteur ; Scott H. KOLLINS, Auteur ; Geraldine DAWSON, Auteur Article en page(s) : p.3559-3566 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : Attention-deficit/hyperactivity disorder (ADHD) symptoms affect 40-60% of autistic children and have been linked to differences in adaptive behavior. It is unclear whether adaptive behavior in autistic youth is directly impacted by co-occurring ADHD symptoms or by another associated feature of both autism and ADHD, such as increased irritability. The current study examined relationships between irritability, ADHD symptoms, and adaptive behavior in 3- to 7-year-old autistic children. Results suggest that, after adjusting for co-occurring ADHD symptoms, higher levels of irritability are associated with differences in social adaptive behavior specifically. Understanding relationships between irritability, ADHD, and adaptive behavior in autistic children is critical because measures of adaptive behavior, such as the Vineland Scales of Adaptive Functioning, are often used as a proxy for global functioning, as well as for developing intervention plans and measuring outcomes as primary endpoints in clinical trials. En ligne : https://doi.org/10.1007/s10803-022-05753-2 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=534 [article] Adaptive Behavior in Young Autistic Children: Associations with Irritability and ADHD Symptoms [Texte imprimé et/ou numérique] / Naomi O. DAVIS, Auteur ; Marina SPANOS, Auteur ; Maura SABATOS-DEVITO, Auteur ; Rachel AIELLO, Auteur ; Grace T. BARANEK, Auteur ; Scott N. COMPTON, Auteur ; Helen L. EGGER, Auteur ; Lauren FRANZ, Auteur ; Soo-Jeong KIM, Auteur ; Bryan H. KING, Auteur ; Alexander KOLEVZON, Auteur ; Christopher J. MCDOUGLE, Auteur ; Kevin SANDERS, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur ; Linmarie SIKICH, Auteur ; Scott H. KOLLINS, Auteur ; Geraldine DAWSON, Auteur . - p.3559-3566. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 54-9 (September 2024) . - p.3559-3566 Index. décimale : PER Périodiques Résumé : Attention-deficit/hyperactivity disorder (ADHD) symptoms affect 40-60% of autistic children and have been linked to differences in adaptive behavior. It is unclear whether adaptive behavior in autistic youth is directly impacted by co-occurring ADHD symptoms or by another associated feature of both autism and ADHD, such as increased irritability. The current study examined relationships between irritability, ADHD symptoms, and adaptive behavior in 3- to 7-year-old autistic children. Results suggest that, after adjusting for co-occurring ADHD symptoms, higher levels of irritability are associated with differences in social adaptive behavior specifically. Understanding relationships between irritability, ADHD, and adaptive behavior in autistic children is critical because measures of adaptive behavior, such as the Vineland Scales of Adaptive Functioning, are often used as a proxy for global functioning, as well as for developing intervention plans and measuring outcomes as primary endpoints in clinical trials. En ligne : https://doi.org/10.1007/s10803-022-05753-2 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=534

Evaluation of clinical assessments of social abilities for use in autism clinical trials by the autism biomarkers consortium for clinical trials / Susan FAJA in Autism Research, 16-5 (May 2023)  

Public ISBD [article]  inAutism Research > 16-5 (May 2023) . - p.981-996 Titre : Evaluation of clinical assessments of social abilities for use in autism clinical trials by the autism biomarkers consortium for clinical trials Type de document : Texte imprimé et/ou numérique Auteurs : Susan FAJA, Auteur ; Maura SABATOS-DEVITO, Auteur ; Aksheya SRIDHAR, Auteur ; Jocelyn L. KUHN, Auteur ; Julia I. NIKOLAEVA, Auteur ; Catherine A. SUGAR, Auteur ; Sara Jane WEBB, Auteur ; Raphael A. BERNIER, Auteur ; Linmarie SIKICH, Auteur ; Gerhard HELLEMANN, Auteur ; Damla SENTURK, Auteur ; Adam J. NAPLES, Auteur ; Frederick SHIC, Auteur ; April R. LEVIN, Auteur ; Helen A. SEOW, Auteur ; James D. DZIURA, Auteur ; Shafali S. JESTE, Auteur ; Katarzyna CHAWARSKA, Auteur ; Charles A. NELSON III, Auteur ; Geraldine DAWSON, Auteur ; James C. MCPARTLAND, Auteur ; Autism Biomarkers Consortium for Clinical TRIALS, Auteur Article en page(s) : p.981-996 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : Abstract Clinical trials in autism spectrum disorder (ASD) often rely on clinician rating scales and parent surveys to measure autism-related features and social behaviors. To aid in the selection of these assessments for future clinical trials, the Autism Biomarkers Consortium for Clinical Trials (ABC-CT) directly compared eight common instruments with respect to acquisition rates, sensitivity to group differences, equivalence across demographic sub-groups, convergent validity, and stability over a 6-week period. The sample included 280 children diagnosed with ASD (65 girls) and 119 neurotypical children (36 girls) aged from 6 to 11?years. Full scale IQ for ASD ranged from 60 to 150 and for neurotypical ranged from 86 to 150. Instruments measured clinician global assessment and autism-related behaviors, social communication abilities, adaptive function, and social withdrawal behavior. For each instrument, we examined only the scales that measured social or communication functioning. Data acquisition rates were at least 97.5% at T1 and 95.7% at T2. All scales distinguished diagnostic groups. Some scales significantly differed by participant and/or family demographic characteristics. Within the ASD group, most clinical instruments exhibited weak (? |0.1|) to moderate (? |0.4|) intercorrelations. Short-term stability was moderate (ICC: 0.5-0.75) to excellent (ICC: >0.9) within the ASD group. Variations in the degree of stability may inform viability for different contexts of use, such as identifying clinical subgroups for trials versus serving as a modifiable clinical outcome. All instruments were evaluated in terms of their advantages and potential concerns for use in clinical trials. En ligne : http://dx.doi.org/https://doi.org/10.1002/aur.2905 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=503 [article] Evaluation of clinical assessments of social abilities for use in autism clinical trials by the autism biomarkers consortium for clinical trials [Texte imprimé et/ou numérique] / Susan FAJA, Auteur ; Maura SABATOS-DEVITO, Auteur ; Aksheya SRIDHAR, Auteur ; Jocelyn L. KUHN, Auteur ; Julia I. NIKOLAEVA, Auteur ; Catherine A. SUGAR, Auteur ; Sara Jane WEBB, Auteur ; Raphael A. BERNIER, Auteur ; Linmarie SIKICH, Auteur ; Gerhard HELLEMANN, Auteur ; Damla SENTURK, Auteur ; Adam J. NAPLES, Auteur ; Frederick SHIC, Auteur ; April R. LEVIN, Auteur ; Helen A. SEOW, Auteur ; James D. DZIURA, Auteur ; Shafali S. JESTE, Auteur ; Katarzyna CHAWARSKA, Auteur ; Charles A. NELSON III, Auteur ; Geraldine DAWSON, Auteur ; James C. MCPARTLAND, Auteur ; Autism Biomarkers Consortium for Clinical TRIALS, Auteur . - p.981-996. Langues : Anglais (eng) in Autism Research > 16-5 (May 2023) . - p.981-996 Index. décimale : PER Périodiques Résumé : Abstract Clinical trials in autism spectrum disorder (ASD) often rely on clinician rating scales and parent surveys to measure autism-related features and social behaviors. To aid in the selection of these assessments for future clinical trials, the Autism Biomarkers Consortium for Clinical Trials (ABC-CT) directly compared eight common instruments with respect to acquisition rates, sensitivity to group differences, equivalence across demographic sub-groups, convergent validity, and stability over a 6-week period. The sample included 280 children diagnosed with ASD (65 girls) and 119 neurotypical children (36 girls) aged from 6 to 11?years. Full scale IQ for ASD ranged from 60 to 150 and for neurotypical ranged from 86 to 150. Instruments measured clinician global assessment and autism-related behaviors, social communication abilities, adaptive function, and social withdrawal behavior. For each instrument, we examined only the scales that measured social or communication functioning. Data acquisition rates were at least 97.5% at T1 and 95.7% at T2. All scales distinguished diagnostic groups. Some scales significantly differed by participant and/or family demographic characteristics. Within the ASD group, most clinical instruments exhibited weak (? |0.1|) to moderate (? |0.4|) intercorrelations. Short-term stability was moderate (ICC: 0.5-0.75) to excellent (ICC: >0.9) within the ASD group. Variations in the degree of stability may inform viability for different contexts of use, such as identifying clinical subgroups for trials versus serving as a modifiable clinical outcome. All instruments were evaluated in terms of their advantages and potential concerns for use in clinical trials. En ligne : http://dx.doi.org/https://doi.org/10.1002/aur.2905 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=503

Exploring the Manifestations of Anxiety in Children with Autism Spectrum Disorders / Victoria HALLETT in Journal of Autism and Developmental Disorders, 43-10 (October 2013)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 43-10 (October 2013) . - p.2341-2352 Titre : Exploring the Manifestations of Anxiety in Children with Autism Spectrum Disorders Type de document : Texte imprimé et/ou numérique Auteurs : Victoria HALLETT, Auteur ; Luc LECAVALIER, Auteur ; Denis G. SUKHODOLSKY, Auteur ; Noreen CIPRIANO, Auteur ; Michael G. AMAN, Auteur ; James T. MCCRACKEN, Auteur ; Christopher J. MCDOUGLE, Auteur ; Elaine TIERNEY, Auteur ; Bryan H. KING, Auteur ; Eric HOLLANDER, Auteur ; Linmarie SIKICH, Auteur ; Joel D. BREGMAN, Auteur ; Evdokia ANAGNOSTOU, Auteur ; Craig DONNELLY, Auteur ; Lily KATSOVICH, Auteur ; Kimberly DUKES, Auteur ; Benedetto VITIELLO, Auteur ; Kenneth D. GADOW, Auteur ; Lawrence SCAHILL, Auteur Article en page(s) : p.2341-2352 Langues : Anglais (eng) Mots-clés : Autism spectrum disorders  Anxiety  Measurement  Clinical Trials Index. décimale : PER Périodiques Résumé : This study explores the manifestation and measurement of anxiety symptoms in 415 children with ASDs on a 20-item, parent-rated, DSM-IV referenced anxiety scale. In both high and low-functioning children (IQ above vs. below 70), commonly endorsed items assessed restlessness, tension and sleep difficulties. Items requiring verbal expression of worry by the child were rarely endorsed. Higher anxiety was associated with functional language, IQ above 70 and higher scores on several other behavioral measures. Four underlying factors emerged: Generalized Anxiety, Separation Anxiety, Social Anxiety and Over-arousal. Our findings extend our understanding of anxiety across IQ in ASD and provide guidance for improving anxiety outcome measurement. En ligne : http://dx.doi.org/10.1007/s10803-013-1775-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=215 [article] Exploring the Manifestations of Anxiety in Children with Autism Spectrum Disorders [Texte imprimé et/ou numérique] / Victoria HALLETT, Auteur ; Luc LECAVALIER, Auteur ; Denis G. SUKHODOLSKY, Auteur ; Noreen CIPRIANO, Auteur ; Michael G. AMAN, Auteur ; James T. MCCRACKEN, Auteur ; Christopher J. MCDOUGLE, Auteur ; Elaine TIERNEY, Auteur ; Bryan H. KING, Auteur ; Eric HOLLANDER, Auteur ; Linmarie SIKICH, Auteur ; Joel D. BREGMAN, Auteur ; Evdokia ANAGNOSTOU, Auteur ; Craig DONNELLY, Auteur ; Lily KATSOVICH, Auteur ; Kimberly DUKES, Auteur ; Benedetto VITIELLO, Auteur ; Kenneth D. GADOW, Auteur ; Lawrence SCAHILL, Auteur . - p.2341-2352. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 43-10 (October 2013) . - p.2341-2352 Mots-clés : Autism spectrum disorders  Anxiety  Measurement  Clinical Trials Index. décimale : PER Périodiques Résumé : This study explores the manifestation and measurement of anxiety symptoms in 415 children with ASDs on a 20-item, parent-rated, DSM-IV referenced anxiety scale. In both high and low-functioning children (IQ above vs. below 70), commonly endorsed items assessed restlessness, tension and sleep difficulties. Items requiring verbal expression of worry by the child were rarely endorsed. Higher anxiety was associated with functional language, IQ above 70 and higher scores on several other behavioral measures. Four underlying factors emerged: Generalized Anxiety, Separation Anxiety, Social Anxiety and Over-arousal. Our findings extend our understanding of anxiety across IQ in ASD and provide guidance for improving anxiety outcome measurement. En ligne : http://dx.doi.org/10.1007/s10803-013-1775-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=215

Genetic and epigenetic signatures associated with plasma oxytocin levels in children and adolescents with autism spectrum disorder / Stephen K. SIECINSKI in Autism Research, 16-3 (March 2023)  

Public ISBD [article]  inAutism Research > 16-3 (March 2023) . - p.502-523 Titre : Genetic and epigenetic signatures associated with plasma oxytocin levels in children and adolescents with autism spectrum disorder Type de document : Texte imprimé et/ou numérique Auteurs : Stephen K. SIECINSKI, Auteur ; Stephanie N. GIAMBERARDINO, Auteur ; Marina SPANOS, Auteur ; Annalise C. HAUSER, Auteur ; Jason R. GIBSON, Auteur ; Tara CHANDRASEKHAR, Auteur ; Maria Del Pilar TRELLES, Auteur ; Carol M. ROCKHILL, Auteur ; Michelle L. PALUMBO, Auteur ; Allyson Witters CUNDIFF, Auteur ; Alicia MONTGOMERY, Auteur ; Paige SIPER, Auteur ; Mendy MINJAREZ, Auteur ; Lisa A. NOWINSKI, Auteur ; Sarah MARLER, Auteur ; Lydia C. KWEE, Auteur ; Lauren C. SHUFFREY, Auteur ; Cheryl ALDERMAN, Auteur ; Jordana WEISSMAN, Auteur ; Brooke ZAPPONE, Auteur ; Jennifer E. MULLETT, Auteur ; Hope CROSSON, Auteur ; Natalie HONG, Auteur ; Sheng LUO, Auteur ; Lilin SHE, Auteur ; Manjushri BHAPKAR, Auteur ; Russell DEAN, Auteur ; Abby SCHEER, Auteur ; Jacqueline L. JOHNSON, Auteur ; Bryan H. KING, Auteur ; Christopher J. MCDOUGLE, Auteur ; Kevin B. SANDERS, Auteur ; Soo-Jeong KIM, Auteur ; Alexander KOLEVZON, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur ; Elizabeth R. HAUSER, Auteur ; Linmarie SIKICH, Auteur ; Simon G. GREGORY, Auteur Article en page(s) : p.502-523 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : Abstract Oxytocin (OT), the brain's most abundant neuropeptide, plays an important role in social salience and motivation. Clinical trials of the efficacy of OT in autism spectrum disorder (ASD) have reported mixed results due in part to ASD's complex etiology. We investigated whether genetic and epigenetic variation contribute to variable endogenous OT levels that modulate sensitivity to OT therapy. To carry out this analysis, we integrated genome-wide profiles of DNA-methylation, transcriptional activity, and genetic variation with plasma OT levels in 290 participants with ASD enrolled in a randomized controlled trial of OT. Our analysis identified genetic variants with novel association with plasma OT, several of which reside in known ASD risk genes. We also show subtle but statistically significant association of plasma OT levels with peripheral transcriptional activity and DNA-methylation profiles across several annotated gene sets. These findings broaden our understanding of the effects of the peripheral oxytocin system and provide novel genetic candidates for future studies to decode the complex etiology of ASD and its interaction with OT signaling and OT-based interventions. Lay Summary Oxytocin (OT) is an abundant chemical produced by neurons that plays an important role in social interaction and motivation. We investigated whether genetic and epigenetic factors contribute to variable OT levels in the blood. To this, we integrated genetic, gene expression, and non-DNA regulated (epigenetic) signatures with blood OT levels in 290 participants with autism enrolled in an OT clinical trial. We identified genetic association with plasma OT, several of which reside in known autism risk genes. We also show statistically significant association of plasma OT levels with gene expression and epigenetic across several gene pathways. These findings broaden our understanding of the factors that influence OT levels in the blood for future studies to decode the complex presentation of autism and its interaction with OT and OT-based treatment. En ligne : https://doi.org/10.1002/aur.2884 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=498 [article] Genetic and epigenetic signatures associated with plasma oxytocin levels in children and adolescents with autism spectrum disorder [Texte imprimé et/ou numérique] / Stephen K. SIECINSKI, Auteur ; Stephanie N. GIAMBERARDINO, Auteur ; Marina SPANOS, Auteur ; Annalise C. HAUSER, Auteur ; Jason R. GIBSON, Auteur ; Tara CHANDRASEKHAR, Auteur ; Maria Del Pilar TRELLES, Auteur ; Carol M. ROCKHILL, Auteur ; Michelle L. PALUMBO, Auteur ; Allyson Witters CUNDIFF, Auteur ; Alicia MONTGOMERY, Auteur ; Paige SIPER, Auteur ; Mendy MINJAREZ, Auteur ; Lisa A. NOWINSKI, Auteur ; Sarah MARLER, Auteur ; Lydia C. KWEE, Auteur ; Lauren C. SHUFFREY, Auteur ; Cheryl ALDERMAN, Auteur ; Jordana WEISSMAN, Auteur ; Brooke ZAPPONE, Auteur ; Jennifer E. MULLETT, Auteur ; Hope CROSSON, Auteur ; Natalie HONG, Auteur ; Sheng LUO, Auteur ; Lilin SHE, Auteur ; Manjushri BHAPKAR, Auteur ; Russell DEAN, Auteur ; Abby SCHEER, Auteur ; Jacqueline L. JOHNSON, Auteur ; Bryan H. KING, Auteur ; Christopher J. MCDOUGLE, Auteur ; Kevin B. SANDERS, Auteur ; Soo-Jeong KIM, Auteur ; Alexander KOLEVZON, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur ; Elizabeth R. HAUSER, Auteur ; Linmarie SIKICH, Auteur ; Simon G. GREGORY, Auteur . - p.502-523. Langues : Anglais (eng) in Autism Research > 16-3 (March 2023) . - p.502-523 Index. décimale : PER Périodiques Résumé : Abstract Oxytocin (OT), the brain's most abundant neuropeptide, plays an important role in social salience and motivation. Clinical trials of the efficacy of OT in autism spectrum disorder (ASD) have reported mixed results due in part to ASD's complex etiology. We investigated whether genetic and epigenetic variation contribute to variable endogenous OT levels that modulate sensitivity to OT therapy. To carry out this analysis, we integrated genome-wide profiles of DNA-methylation, transcriptional activity, and genetic variation with plasma OT levels in 290 participants with ASD enrolled in a randomized controlled trial of OT. Our analysis identified genetic variants with novel association with plasma OT, several of which reside in known ASD risk genes. We also show subtle but statistically significant association of plasma OT levels with peripheral transcriptional activity and DNA-methylation profiles across several annotated gene sets. These findings broaden our understanding of the effects of the peripheral oxytocin system and provide novel genetic candidates for future studies to decode the complex etiology of ASD and its interaction with OT signaling and OT-based interventions. Lay Summary Oxytocin (OT) is an abundant chemical produced by neurons that plays an important role in social interaction and motivation. We investigated whether genetic and epigenetic factors contribute to variable OT levels in the blood. To this, we integrated genetic, gene expression, and non-DNA regulated (epigenetic) signatures with blood OT levels in 290 participants with autism enrolled in an OT clinical trial. We identified genetic association with plasma OT, several of which reside in known autism risk genes. We also show statistically significant association of plasma OT levels with gene expression and epigenetic across several gene pathways. These findings broaden our understanding of the factors that influence OT levels in the blood for future studies to decode the complex presentation of autism and its interaction with OT and OT-based treatment. En ligne : https://doi.org/10.1002/aur.2884 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=498

Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program / Suma JACOB in Molecular Autism, 13 (2022)  

Public ISBD [article]  inMolecular Autism > 13 (2022) . - 25 p. Titre : Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program Type de document : Texte imprimé et/ou numérique Auteurs : Suma JACOB, Auteur ; Evdokia ANAGNOSTOU, Auteur ; Eric HOLLANDER, Auteur ; Roger JOU, Auteur ; Nora MCNAMARA, Auteur ; Linmarie SIKICH, Auteur ; Russell TOBE, Auteur ; Declan MURPHY, Auteur ; James MCCRACKEN, Auteur ; Elizabeth ASHFORD, Auteur ; Christopher CHATHAM, Auteur ; Susanne CLINCH, Auteur ; Janice SMITH, Auteur ; Kevin SANDERS, Auteur ; Lorraine MURTAGH, Auteur ; Jana NOELDEKE, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur Article en page(s) : 25 p. Langues : Anglais (eng) Mots-clés : Autism Spectrum Disorder/complications/drug therapy  Autistic Disorder/complications/drug therapy  Benzodiazepines  Humans  Pyridines  Quality of Life  Randomized Controlled Trials as Topic  Triazoles  Autism spectrum disorder  Balovaptan  Placebo response  V1aduct  Vanilla  aV1ation  attended advisory boards for Fraser, Minnesota Independence College & Community,  and F. Hoffmann-La Roche Ltd. EA has received grant support from F. Hoffmann-La  Roche Ltd and SynapDx  royalties from APPI, Springer, and Wiley  has acted as a  consultant for Quadrant, F. Hoffmann-La Roche Ltd, and SynapDx  has received  honorarium for a webinar from AIDE  has a patent on anxiety meter  and has  received a study drug and in kind supports from AMO Pharma. EH has received  research grants from the Department of Defense, Food and Drug Administration, GW  Pharma, and F. Hoffmann-La Roche Ltd  and editorial stipends from Elsevier  and  served on scientific advisory boards for GW Pharma and F. Hoffmann-La Roche Ltd.  RJ’s institution received payments for clinical trial agreements from Roche  Translational & Clinical Research Center, Inc. and Genentech, Inc. NM has  provided research support to F. Hoffmann-La Roche Ltd  and consulted for Shire  grant support has been received from Forest Research Institute, Genentech,  Lundbeck, Pfizer, F. Hoffmann-La Roche Ltd, Shire, Sunovion, and Zynerba. LS has  received funding from the National Institute of Child Health and Human  Development  was paid by F. Hoffmann-La Roche Ltd as a clinical research site  investigator for involvement in conducting the VANILLA and aV1ation studies  has  attended an advisory board for F. Hoffmann-La Roche Ltd  and has a patent pending  for new formulation of intranasal oxytocin by Duke University  part of her salary  is paid by the Duke Clinical Research Institute where she is providing thought  leadership for trials sponsored by Tris Pharmaceuticals. RT has received grant  support from Janssen and F. Hoffmann-La Roche Ltd  and has attended advisory  boards for F. Hoffmann-La Roche Ltd. DM receives funding support from the  EU/EFPIA/SFARI/Autistica/AUTISM SPEAKS Innovative Medicines Initiative 2 Joint  Undertaking (AIMS-2-TRIALS Grant No. 777394)  has received grant support from F  Hoffmann-La Roche and Shire  royalties from Springer and Wiley  and has attended  advisory boards for F Hoffmann-La Roche and Servier. JM has received research  support from F. Hoffmann-La Roche Ltd  has attended advisory boards for GW  Pharmaceuticals and F. Hoffmann-La Roche Ltd  and was a consultant for Octopharma  and TRIS Pharmaceuticals. JV-VW has received research support from NIH, the  Simons Foundation, Health Canada, F. Hoffmann-La Roche Ltd, Janssen, Acadia, and  Zynerba  honoraria for lectures at the  American Academy of Child and Adolescent Psychiatry, Karolinska Institute, Mount  Sinai, National Institute of Neurological Disease and Stroke, Florida Atlantic  University, UCLA, Stanford University, Child Mind Institute, and Pennsylvania  State University  has attended advisory boards for Roche  serves on the medical  and/or scientific advisory boards for Autism Speaks, the Simons Foundation Autism  Research Initiative, and the Brain Behavior Research Foundation  and serves as  the co-chair of the Autism and Intellectual Disability Committee for the American  Academy of Child and Adolescent Psychiatry. EAs is an employee of F. Hoffmann-La  Roche Ltd, and has stock options in F. Hoffmann-La Roche Ltd. CC and SC are  employees of F. Hoffmann-La Roche Ltd, and have stocks in F. Hoffmann-La Roche  Ltd. JS, JN, KS, and LM are employees of F. Hoffmann-La Roche Ltd and have stocks  and stock options in F. Hoffmann-La Roche Ltd. Index. décimale : PER Périodiques Résumé : BACKGROUND: Autism spectrum disorder (ASD) is a common and heterogeneous neurodevelopmental condition that is characterized by the core symptoms of social communication difficulties and restricted and repetitive behaviors. At present, there is an unmet medical need for therapies to ameliorate these core symptoms in order to improve quality of life of autistic individuals. However, several challenges are currently faced by the ASD community relating to the development of pharmacotherapies, namely in the conduct of clinical trials. Balovaptan is a V1a receptor antagonist that has been investigated to improve social communication difficulties in individuals with ASD. In this viewpoint, we draw upon our recent first-hand experiences of the balovaptan clinical development program to describe current challenges of ASD trials. DISCUSSION POINTS: The balovaptan trials were conducted in a wide age range of individuals with ASD with the added complexities associated with international trials. When summarizing all three randomized trials of balovaptan, a placebo response was observed across several outcome measures. Placebo response was predicted by greater baseline symptom severity, online recruitment of participants, and less experienced or non-academic trial sites. We also highlight challenges relating to selection of outcome measures in ASD, the impact of baseline characteristics, and the role of expectation bias in influencing trial results. CONCLUSION: Taken together, the balovaptan clinical development program has advanced our understanding of the key challenges facing ASD treatment research. The insights gained can be used to inform and improve the design of future clinical trials with the collective aim of developing efficacious therapies to support individuals with ASD. En ligne : http://dx.doi.org/10.1186/s13229-022-00505-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=491 [article] Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program [Texte imprimé et/ou numérique] / Suma JACOB, Auteur ; Evdokia ANAGNOSTOU, Auteur ; Eric HOLLANDER, Auteur ; Roger JOU, Auteur ; Nora MCNAMARA, Auteur ; Linmarie SIKICH, Auteur ; Russell TOBE, Auteur ; Declan MURPHY, Auteur ; James MCCRACKEN, Auteur ; Elizabeth ASHFORD, Auteur ; Christopher CHATHAM, Auteur ; Susanne CLINCH, Auteur ; Janice SMITH, Auteur ; Kevin SANDERS, Auteur ; Lorraine MURTAGH, Auteur ; Jana NOELDEKE, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur . - 25 p. Langues : Anglais (eng) in Molecular Autism > 13 (2022) . - 25 p. Mots-clés : Autism Spectrum Disorder/complications/drug therapy  Autistic Disorder/complications/drug therapy  Benzodiazepines  Humans  Pyridines  Quality of Life  Randomized Controlled Trials as Topic  Triazoles  Autism spectrum disorder  Balovaptan  Placebo response  V1aduct  Vanilla  aV1ation  attended advisory boards for Fraser, Minnesota Independence College & Community,  and F. Hoffmann-La Roche Ltd. EA has received grant support from F. Hoffmann-La  Roche Ltd and SynapDx  royalties from APPI, Springer, and Wiley  has acted as a  consultant for Quadrant, F. Hoffmann-La Roche Ltd, and SynapDx  has received  honorarium for a webinar from AIDE  has a patent on anxiety meter  and has  received a study drug and in kind supports from AMO Pharma. EH has received  research grants from the Department of Defense, Food and Drug Administration, GW  Pharma, and F. Hoffmann-La Roche Ltd  and editorial stipends from Elsevier  and  served on scientific advisory boards for GW Pharma and F. Hoffmann-La Roche Ltd.  RJ’s institution received payments for clinical trial agreements from Roche  Translational & Clinical Research Center, Inc. and Genentech, Inc. NM has  provided research support to F. Hoffmann-La Roche Ltd  and consulted for Shire  grant support has been received from Forest Research Institute, Genentech,  Lundbeck, Pfizer, F. Hoffmann-La Roche Ltd, Shire, Sunovion, and Zynerba. LS has  received funding from the National Institute of Child Health and Human  Development  was paid by F. Hoffmann-La Roche Ltd as a clinical research site  investigator for involvement in conducting the VANILLA and aV1ation studies  has  attended an advisory board for F. Hoffmann-La Roche Ltd  and has a patent pending  for new formulation of intranasal oxytocin by Duke University  part of her salary  is paid by the Duke Clinical Research Institute where she is providing thought  leadership for trials sponsored by Tris Pharmaceuticals. RT has received grant  support from Janssen and F. Hoffmann-La Roche Ltd  and has attended advisory  boards for F. Hoffmann-La Roche Ltd. DM receives funding support from the  EU/EFPIA/SFARI/Autistica/AUTISM SPEAKS Innovative Medicines Initiative 2 Joint  Undertaking (AIMS-2-TRIALS Grant No. 777394)  has received grant support from F  Hoffmann-La Roche and Shire  royalties from Springer and Wiley  and has attended  advisory boards for F Hoffmann-La Roche and Servier. JM has received research  support from F. Hoffmann-La Roche Ltd  has attended advisory boards for GW  Pharmaceuticals and F. Hoffmann-La Roche Ltd  and was a consultant for Octopharma  and TRIS Pharmaceuticals. JV-VW has received research support from NIH, the  Simons Foundation, Health Canada, F. Hoffmann-La Roche Ltd, Janssen, Acadia, and  Zynerba  honoraria for lectures at the  American Academy of Child and Adolescent Psychiatry, Karolinska Institute, Mount  Sinai, National Institute of Neurological Disease and Stroke, Florida Atlantic  University, UCLA, Stanford University, Child Mind Institute, and Pennsylvania  State University  has attended advisory boards for Roche  serves on the medical  and/or scientific advisory boards for Autism Speaks, the Simons Foundation Autism  Research Initiative, and the Brain Behavior Research Foundation  and serves as  the co-chair of the Autism and Intellectual Disability Committee for the American  Academy of Child and Adolescent Psychiatry. EAs is an employee of F. Hoffmann-La  Roche Ltd, and has stock options in F. Hoffmann-La Roche Ltd. CC and SC are  employees of F. Hoffmann-La Roche Ltd, and have stocks in F. Hoffmann-La Roche  Ltd. JS, JN, KS, and LM are employees of F. Hoffmann-La Roche Ltd and have stocks  and stock options in F. Hoffmann-La Roche Ltd. Index. décimale : PER Périodiques Résumé : BACKGROUND: Autism spectrum disorder (ASD) is a common and heterogeneous neurodevelopmental condition that is characterized by the core symptoms of social communication difficulties and restricted and repetitive behaviors. At present, there is an unmet medical need for therapies to ameliorate these core symptoms in order to improve quality of life of autistic individuals. However, several challenges are currently faced by the ASD community relating to the development of pharmacotherapies, namely in the conduct of clinical trials. Balovaptan is a V1a receptor antagonist that has been investigated to improve social communication difficulties in individuals with ASD. In this viewpoint, we draw upon our recent first-hand experiences of the balovaptan clinical development program to describe current challenges of ASD trials. DISCUSSION POINTS: The balovaptan trials were conducted in a wide age range of individuals with ASD with the added complexities associated with international trials. When summarizing all three randomized trials of balovaptan, a placebo response was observed across several outcome measures. Placebo response was predicted by greater baseline symptom severity, online recruitment of participants, and less experienced or non-academic trial sites. We also highlight challenges relating to selection of outcome measures in ASD, the impact of baseline characteristics, and the role of expectation bias in influencing trial results. CONCLUSION: Taken together, the balovaptan clinical development program has advanced our understanding of the key challenges facing ASD treatment research. The insights gained can be used to inform and improve the design of future clinical trials with the collective aim of developing efficacious therapies to support individuals with ASD. En ligne : http://dx.doi.org/10.1186/s13229-022-00505-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=491

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