22 recherche sur le mot-clé 'Research Design'

Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents with Autism Spectrum Disorder: Design of Two Phase III Studies (SIGN Trials) / V. CRUTEL in Journal of Autism and Developmental Disorders, 51-8 (August 2021)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 51-8 (August 2021) . - p.2959-2972 Titre : Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents with Autism Spectrum Disorder: Design of Two Phase III Studies (SIGN Trials) Type de document : Texte imprimé et/ou numérique Auteurs : V. CRUTEL, Auteur ; E. LAMBERT, Auteur ; P. F. PENELAUD, Auteur ; C. ALBARRÁN SEVERO, Auteur ; J. FUENTES, Auteur ; A. ROSIER, Auteur ; A. HERVAS, Auteur ; S. MARRET, Auteur ; G. OLIVEIRA, Auteur ; Mara PARELLADA, Auteur ; S. KYAGA, Auteur ; S. GOUTTEFANGEAS, Auteur ; M. BERTRAND, Auteur ; D. RAVEL, Auteur ; B. FALISSARD, Auteur Article en page(s) : p.2959-2972 Langues : Anglais (eng) Mots-clés : Adolescent  Autism Spectrum Disorder/drug therapy  Bumetanide/administration & dosage/therapeutic use  Child  Child, Preschool  Double-Blind Method  Humans  Male  Research Design  Social Behavior  Treatment Outcome  Autism spectrum disorder  Bumetanide  Pediatrics  Randomized controlled trial  for Actelion, Allergan, Almirall, Astellas, AstraZeneca, Bayer, Biotronik, BMS,  Boehringer Ingelheim, Daiichi- Sankyo, Eli Lilly, Genzyme, Gilead, Grunenthal, GSK,  HRA, Janssen, Lundbeck, MSD, Novartis, Otsuka, Pierre Fabre, Roche, Sanofi, Servier,  Stallergene, UCB, ViiV. JF has received research support from Servier and  AIMS-2-Trials project ID 777394. DR is an employee of Neurochlore. GO, SM, AR, AH,  and MP report no conflict of interest. Index. décimale : PER Périodiques Résumé : There are currently no approved pharmacological treatments to improve social reciprocity and limit repetitive and rigid behaviors in autism spectrum disorder (ASD). We describe the design of two Phase III studies evaluating the efficacy/safety of bumetanide oral liquid formulation in ASD. These are international, multicenter, randomized, double-blind, placebo-controlled studies in children and adolescents with ASD aged 7 to 17 years (n?=?200; study 1), or younger children with ASD aged 2 to 6 years (n?=?200; study 2). The primary endpoint of each is change in Childhood Autism Rating Scale 2 total raw score after 6 months. These studies could contribute to the first pharmacological treatment to improve social reciprocity and limit repetitive and rigid behaviors in children and adolescents with ASD. En ligne : http://dx.doi.org/10.1007/s10803-020-04709-8 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=453 [article] Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents with Autism Spectrum Disorder: Design of Two Phase III Studies (SIGN Trials) [Texte imprimé et/ou numérique] / V. CRUTEL, Auteur ; E. LAMBERT, Auteur ; P. F. PENELAUD, Auteur ; C. ALBARRÁN SEVERO, Auteur ; J. FUENTES, Auteur ; A. ROSIER, Auteur ; A. HERVAS, Auteur ; S. MARRET, Auteur ; G. OLIVEIRA, Auteur ; Mara PARELLADA, Auteur ; S. KYAGA, Auteur ; S. GOUTTEFANGEAS, Auteur ; M. BERTRAND, Auteur ; D. RAVEL, Auteur ; B. FALISSARD, Auteur . - p.2959-2972. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 51-8 (August 2021) . - p.2959-2972 Mots-clés : Adolescent  Autism Spectrum Disorder/drug therapy  Bumetanide/administration & dosage/therapeutic use  Child  Child, Preschool  Double-Blind Method  Humans  Male  Research Design  Social Behavior  Treatment Outcome  Autism spectrum disorder  Bumetanide  Pediatrics  Randomized controlled trial  for Actelion, Allergan, Almirall, Astellas, AstraZeneca, Bayer, Biotronik, BMS,  Boehringer Ingelheim, Daiichi- Sankyo, Eli Lilly, Genzyme, Gilead, Grunenthal, GSK,  HRA, Janssen, Lundbeck, MSD, Novartis, Otsuka, Pierre Fabre, Roche, Sanofi, Servier,  Stallergene, UCB, ViiV. JF has received research support from Servier and  AIMS-2-Trials project ID 777394. DR is an employee of Neurochlore. GO, SM, AR, AH,  and MP report no conflict of interest. Index. décimale : PER Périodiques Résumé : There are currently no approved pharmacological treatments to improve social reciprocity and limit repetitive and rigid behaviors in autism spectrum disorder (ASD). We describe the design of two Phase III studies evaluating the efficacy/safety of bumetanide oral liquid formulation in ASD. These are international, multicenter, randomized, double-blind, placebo-controlled studies in children and adolescents with ASD aged 7 to 17 years (n?=?200; study 1), or younger children with ASD aged 2 to 6 years (n?=?200; study 2). The primary endpoint of each is change in Childhood Autism Rating Scale 2 total raw score after 6 months. These studies could contribute to the first pharmacological treatment to improve social reciprocity and limit repetitive and rigid behaviors in children and adolescents with ASD. En ligne : http://dx.doi.org/10.1007/s10803-020-04709-8 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=453

Commentary: Leveraging discovery science to advance child and adolescent psychiatric research – a commentary on Zhao and Castellanos 2016 / Maarten MENNES in Journal of Child Psychology and Psychiatry, 57-3 (March 2016)  

Public ISBD [article]  inJournal of Child Psychology and Psychiatry > 57-3 (March 2016) . - p.440-442 Titre : Commentary: Leveraging discovery science to advance child and adolescent psychiatric research – a commentary on Zhao and Castellanos 2016 Type de document : Texte imprimé et/ou numérique Auteurs : Maarten MENNES, Auteur Article en page(s) : p.440-442 Langues : Anglais (eng) Mots-clés : Research design Index. décimale : PER Périodiques Résumé : ‘Big Data’ and ‘Population Imaging’ are becoming integral parts of inspiring research aimed at delineating the biological underpinnings of psychiatric disorders. The scientific strategies currently associated with big data and population imaging are typically embedded in so-called discovery science, thereby pointing to the hypothesis-generating rather than hypothesis-testing nature of discovery science. In this issue, Yihong Zhao and F. Xavier Castellanos provide a compelling overview of strategies for discovery science aimed at progressing our understanding of neuropsychiatric disorders. In particular, they focus on efforts in genetic and neuroimaging research, which, together with extended behavioural testing, form the main pillars of psychopathology research. En ligne : http://dx.doi.org/10.1111/jcpp.12538 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=282 [article] Commentary: Leveraging discovery science to advance child and adolescent psychiatric research – a commentary on Zhao and Castellanos 2016 [Texte imprimé et/ou numérique] / Maarten MENNES, Auteur . - p.440-442. Langues : Anglais (eng) in Journal of Child Psychology and Psychiatry > 57-3 (March 2016) . - p.440-442 Mots-clés : Research design Index. décimale : PER Périodiques Résumé : ‘Big Data’ and ‘Population Imaging’ are becoming integral parts of inspiring research aimed at delineating the biological underpinnings of psychiatric disorders. The scientific strategies currently associated with big data and population imaging are typically embedded in so-called discovery science, thereby pointing to the hypothesis-generating rather than hypothesis-testing nature of discovery science. In this issue, Yihong Zhao and F. Xavier Castellanos provide a compelling overview of strategies for discovery science aimed at progressing our understanding of neuropsychiatric disorders. In particular, they focus on efforts in genetic and neuroimaging research, which, together with extended behavioural testing, form the main pillars of psychopathology research. En ligne : http://dx.doi.org/10.1111/jcpp.12538 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=282

Commentary: The construct validity of 'camouflaging' in autism: psychometric considerations and recommendations for future research - reflection on Lai et al. (2020) / Z. J. WILLIAMS in Journal of Child Psychology and Psychiatry, 63-1 (January 2022)  

Public ISBD [article]  inJournal of Child Psychology and Psychiatry > 63-1 (January 2022) . - p.118-121 Titre : Commentary: The construct validity of 'camouflaging' in autism: psychometric considerations and recommendations for future research - reflection on Lai et al. (2020) Type de document : Texte imprimé et/ou numérique Auteurs : Z. J. WILLIAMS, Auteur Article en page(s) : p.118-121 Langues : Anglais (eng) Mots-clés : Autistic Disorder  Humans  Mental Health  Psychometrics  Research Design  Research Personnel Index. décimale : PER Périodiques Résumé : Research on the construct of 'camouflaging' in autism and its sociodemographic/clinical correlates has far outpaced the work being done to establish the construct validity of camouflaging and its distinction from other similar constructs. The imprecision with which camouflaging is defined and measured has serious implications for future research on this topic, and unless additional effort is made to produce reliable and valid measurements of this construct, researchers will not be able to meaningfully assess important questions such as whether the effort of camouflaging one's behavior contributes to increased mental health difficulties. By reviewing the psychometric strengths and weaknesses of various operationalizations of camouflaging, this commentary highlights a pressing need for further measure validation in this area. Specific methodological guidance is provided for researchers interested in rigorously testing the validity of putative camouflaging measures. En ligne : http://dx.doi.org/10.1111/jcpp.13468 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=457 [article] Commentary: The construct validity of 'camouflaging' in autism: psychometric considerations and recommendations for future research - reflection on Lai et al. (2020) [Texte imprimé et/ou numérique] / Z. J. WILLIAMS, Auteur . - p.118-121. Langues : Anglais (eng) in Journal of Child Psychology and Psychiatry > 63-1 (January 2022) . - p.118-121 Mots-clés : Autistic Disorder  Humans  Mental Health  Psychometrics  Research Design  Research Personnel Index. décimale : PER Périodiques Résumé : Research on the construct of 'camouflaging' in autism and its sociodemographic/clinical correlates has far outpaced the work being done to establish the construct validity of camouflaging and its distinction from other similar constructs. The imprecision with which camouflaging is defined and measured has serious implications for future research on this topic, and unless additional effort is made to produce reliable and valid measurements of this construct, researchers will not be able to meaningfully assess important questions such as whether the effort of camouflaging one's behavior contributes to increased mental health difficulties. By reviewing the psychometric strengths and weaknesses of various operationalizations of camouflaging, this commentary highlights a pressing need for further measure validation in this area. Specific methodological guidance is provided for researchers interested in rigorously testing the validity of putative camouflaging measures. En ligne : http://dx.doi.org/10.1111/jcpp.13468 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=457

Is autism a unitary biological entity? A revised and extended response to "A radical change in our autism research strategy is needed: Back to prototypes" (Mottron, 2021, Autism Research) / L. WATERHOUSE in Autism Research, 14-10 (October 2021)  

Public ISBD [article]  inAutism Research > 14-10 (October 2021) . - p.2241-2242 Titre : Is autism a unitary biological entity? A revised and extended response to "A radical change in our autism research strategy is needed: Back to prototypes" (Mottron, 2021, Autism Research) Type de document : Texte imprimé et/ou numérique Auteurs : L. WATERHOUSE, Auteur Article en page(s) : p.2241-2242 Langues : Anglais (eng) Mots-clés : Analysis of Variance  Autism Spectrum Disorder  Autistic Disorder  Humans  Research Design Index. décimale : PER Périodiques En ligne : http://dx.doi.org/10.1002/aur.2602 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=450 [article] Is autism a unitary biological entity? A revised and extended response to "A radical change in our autism research strategy is needed: Back to prototypes" (Mottron, 2021, Autism Research) [Texte imprimé et/ou numérique] / L. WATERHOUSE, Auteur . - p.2241-2242. Langues : Anglais (eng) in Autism Research > 14-10 (October 2021) . - p.2241-2242 Mots-clés : Analysis of Variance  Autism Spectrum Disorder  Autistic Disorder  Humans  Research Design Index. décimale : PER Périodiques En ligne : http://dx.doi.org/10.1002/aur.2602 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=450

A radical change in our autism research strategy is needed: Back to prototypes / L. MOTTRON in Autism Research, 14-10 (October 2021)  

Public ISBD [article]  inAutism Research > 14-10 (October 2021) . - p.2213-2220 Titre : A radical change in our autism research strategy is needed: Back to prototypes Type de document : Texte imprimé et/ou numérique Auteurs : L. MOTTRON, Auteur Article en page(s) : p.2213-2220 Langues : Anglais (eng) Mots-clés : Autism Spectrum Disorder  Autistic Disorder/diagnosis/epidemiology  Humans  Prevalence  Reproducibility of Results  Research Design  diagnostic  polythetic criteria  prototype  reliability  type 2 error Index. décimale : PER Périodiques Résumé : The evolution of autism diagnosis, from its discovery to its current delineation using standardized instruments, has been paralleled by a steady increase in its prevalence and heterogeneity. In clinical settings, the diagnosis of autism is now too vague to specify the type of support required by the concerned individuals. In research, the inclusion of individuals categorically defined by over-inclusive, polythetic criteria in autism cohorts results in a population whose heterogeneity runs contrary to the advancement of scientific progress. Investigating individuals sharing only a trivial resemblance produces a large-scale type-2 error (not finding differences between autistic and dominant population) rather than detecting mechanistic differences to explain their phenotypic divergences. The dimensional approach of autism proposed to cure the disease of its categorical diagnosis is plagued by the arbitrariness of the dimensions under study. Here, we argue that an emphasis on the reliability rather than specificity of diagnostic criteria and the misuse of diagnostic instruments, which ignore the recognition of a prototype, leads to confound autism with the entire range of neurodevelopmental conditions and personality variants. We propose centering research on cohorts in which individuals are selected based on their expert judged prototypicality to advance the theoretical and practical pervasive issues pertaining to autism diagnostic thresholds. Reversing the current research strategy by giving more weight to specificity than reliability should increase our ability to discover the mechanisms of autism. LAY SUMMARY: Scientific research into the causes of autism and its mechanisms is carried out on large cohorts of people who are less and less different from the general population. This historical trend may explain the poor harvest of results obtained. Services and intervention are provided according to a diagnosis that now encompasses extremely different individuals. Last, we accept as a biological reality the constant increase over the years in the proportion of autistic people among the general population. These drifts are made possible by the attribution of a diagnosis of autism to people who meet vague criteria, rather than to people who experienced clinicians recognize as autistic. We propose to change our research strategy by focusing on the study of the latter, fewer in number, but more representative of the "prototype" of autism. To do this, it is necessary to clearly distinguish the population on which the research is carried out from that to which we provide support. People must receive services according to their needs, and not according to the clarity of their diagnosis. En ligne : http://dx.doi.org/10.1002/aur.2494 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=450 [article] A radical change in our autism research strategy is needed: Back to prototypes [Texte imprimé et/ou numérique] / L. MOTTRON, Auteur . - p.2213-2220. Langues : Anglais (eng) in Autism Research > 14-10 (October 2021) . - p.2213-2220 Mots-clés : Autism Spectrum Disorder  Autistic Disorder/diagnosis/epidemiology  Humans  Prevalence  Reproducibility of Results  Research Design  diagnostic  polythetic criteria  prototype  reliability  type 2 error Index. décimale : PER Périodiques Résumé : The evolution of autism diagnosis, from its discovery to its current delineation using standardized instruments, has been paralleled by a steady increase in its prevalence and heterogeneity. In clinical settings, the diagnosis of autism is now too vague to specify the type of support required by the concerned individuals. In research, the inclusion of individuals categorically defined by over-inclusive, polythetic criteria in autism cohorts results in a population whose heterogeneity runs contrary to the advancement of scientific progress. Investigating individuals sharing only a trivial resemblance produces a large-scale type-2 error (not finding differences between autistic and dominant population) rather than detecting mechanistic differences to explain their phenotypic divergences. The dimensional approach of autism proposed to cure the disease of its categorical diagnosis is plagued by the arbitrariness of the dimensions under study. Here, we argue that an emphasis on the reliability rather than specificity of diagnostic criteria and the misuse of diagnostic instruments, which ignore the recognition of a prototype, leads to confound autism with the entire range of neurodevelopmental conditions and personality variants. We propose centering research on cohorts in which individuals are selected based on their expert judged prototypicality to advance the theoretical and practical pervasive issues pertaining to autism diagnostic thresholds. Reversing the current research strategy by giving more weight to specificity than reliability should increase our ability to discover the mechanisms of autism. LAY SUMMARY: Scientific research into the causes of autism and its mechanisms is carried out on large cohorts of people who are less and less different from the general population. This historical trend may explain the poor harvest of results obtained. Services and intervention are provided according to a diagnosis that now encompasses extremely different individuals. Last, we accept as a biological reality the constant increase over the years in the proportion of autistic people among the general population. These drifts are made possible by the attribution of a diagnosis of autism to people who meet vague criteria, rather than to people who experienced clinicians recognize as autistic. We propose to change our research strategy by focusing on the study of the latter, fewer in number, but more representative of the "prototype" of autism. To do this, it is necessary to clearly distinguish the population on which the research is carried out from that to which we provide support. People must receive services according to their needs, and not according to the clarity of their diagnosis. En ligne : http://dx.doi.org/10.1002/aur.2494 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=450

Response to "A Radical Change in Our Autism Research Strategy is Needed: Back to Prototypes" by Mottron et al. (2021) / John N. CONSTANTINO in Autism Research, 14-10 (October 2021)  

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Response to "A radical change in our autism research strategy is needed: Back to prototypes" by Mottron et al. (2021) / Deborah A. FEIN in Autism Research, 14-10 (October 2021)  

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Commentary: Critical considerations for studying low-functioning autism / Bhismadev CHAKRABARTI in Journal of Child Psychology and Psychiatry, 58-4 (April 2017)  

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Commentary: The best and worst of times – the prospects for magnetic resonance imaging (MRI) of developmental psychopathologies – a commentary on Horga et al. (2014) / Francisco Xavier CASTELLANOS in Journal of Child Psychology and Psychiatry, 55-6 (June 2014)  

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Practitioner Review: Engaging fathers – recommendations for a game change in parenting interventions based on a systematic review of the global evidence / Catherine PANTER-BRICK in Journal of Child Psychology and Psychiatry, 55-11 (November 2014)  

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