Kimaya R Gracias, Laura Sarnie, Michelle Palumbo, Caitlin Ravichandran, Christopher J McDougle, Robyn P Thom
{"title":"神经遗传综合征的精神药理学临床试验招募挑战:来自氟西汀治疗唐氏综合征的开放标签试验的经验教训。","authors":"Kimaya R Gracias, Laura Sarnie, Michelle Palumbo, Caitlin Ravichandran, Christopher J McDougle, Robyn P Thom","doi":"10.1177/10445463251379822","DOIUrl":null,"url":null,"abstract":"<p><p><b><i>Background:</i></b> Despite the increased risk for psychopathology in individuals with neurogenetic syndromes, very few psychopharmacology trials have been conducted in these populations. <b><i>Objectives:</i></b> The objective of this perspectives article is to describe recruitment challenges and potential solutions for psychopharmacology trials in neurogenetic syndromes. <b><i>Methods:</i></b> We describe recruitment challenges and lessons learned from an open-label trial of fluoxetine for the treatment of depression in adults with Down syndrome (DS). These challenges are contrasted with a successful open-label trial of buspirone for the treatment of anxiety in Williams syndrome. <b><i>Results:</i></b> Factors that contributed to recruitment challenges include limited research on the clinical presentation of depression in DS and the relatively small target population. This experience highlights the importance of foundational research studies on the phenomenology of target symptoms and burden of disease, as well as disseminating that information to the patient/family community. Partnership with a local family organization with close ties to the patient population can assist in overcoming recruitment barriers. <b><i>Conclusion:</i></b> The successes and challenges of early psychopharmacology clinical trials in neurogenetic syndromes should be considered for future trials.</p>","PeriodicalId":15277,"journal":{"name":"Journal of child and adolescent psychopharmacology","volume":" ","pages":""},"PeriodicalIF":2.2000,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Psychopharmacology Clinical Trial Recruitment Challenges in Neurogenetic Syndromes: Lessons from an Open-Label Trial of Fluoxetine in Down Syndrome.\",\"authors\":\"Kimaya R Gracias, Laura Sarnie, Michelle Palumbo, Caitlin Ravichandran, Christopher J McDougle, Robyn P Thom\",\"doi\":\"10.1177/10445463251379822\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p><b><i>Background:</i></b> Despite the increased risk for psychopathology in individuals with neurogenetic syndromes, very few psychopharmacology trials have been conducted in these populations. <b><i>Objectives:</i></b> The objective of this perspectives article is to describe recruitment challenges and potential solutions for psychopharmacology trials in neurogenetic syndromes. <b><i>Methods:</i></b> We describe recruitment challenges and lessons learned from an open-label trial of fluoxetine for the treatment of depression in adults with Down syndrome (DS). These challenges are contrasted with a successful open-label trial of buspirone for the treatment of anxiety in Williams syndrome. <b><i>Results:</i></b> Factors that contributed to recruitment challenges include limited research on the clinical presentation of depression in DS and the relatively small target population. This experience highlights the importance of foundational research studies on the phenomenology of target symptoms and burden of disease, as well as disseminating that information to the patient/family community. Partnership with a local family organization with close ties to the patient population can assist in overcoming recruitment barriers. <b><i>Conclusion:</i></b> The successes and challenges of early psychopharmacology clinical trials in neurogenetic syndromes should be considered for future trials.</p>\",\"PeriodicalId\":15277,\"journal\":{\"name\":\"Journal of child and adolescent psychopharmacology\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":2.2000,\"publicationDate\":\"2025-09-22\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of child and adolescent psychopharmacology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/10445463251379822\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of child and adolescent psychopharmacology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/10445463251379822","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PEDIATRICS","Score":null,"Total":0}
Psychopharmacology Clinical Trial Recruitment Challenges in Neurogenetic Syndromes: Lessons from an Open-Label Trial of Fluoxetine in Down Syndrome.
Background: Despite the increased risk for psychopathology in individuals with neurogenetic syndromes, very few psychopharmacology trials have been conducted in these populations. Objectives: The objective of this perspectives article is to describe recruitment challenges and potential solutions for psychopharmacology trials in neurogenetic syndromes. Methods: We describe recruitment challenges and lessons learned from an open-label trial of fluoxetine for the treatment of depression in adults with Down syndrome (DS). These challenges are contrasted with a successful open-label trial of buspirone for the treatment of anxiety in Williams syndrome. Results: Factors that contributed to recruitment challenges include limited research on the clinical presentation of depression in DS and the relatively small target population. This experience highlights the importance of foundational research studies on the phenomenology of target symptoms and burden of disease, as well as disseminating that information to the patient/family community. Partnership with a local family organization with close ties to the patient population can assist in overcoming recruitment barriers. Conclusion: The successes and challenges of early psychopharmacology clinical trials in neurogenetic syndromes should be considered for future trials.
期刊介绍:
Journal of Child and Adolescent Psychopharmacology (JCAP) is the premier peer-reviewed journal covering the clinical aspects of treating this patient population with psychotropic medications including side effects and interactions, standard doses, and research on new and existing medications. The Journal includes information on related areas of medical sciences such as advances in developmental pharmacokinetics, developmental neuroscience, metabolism, nutrition, molecular genetics, and more.
Journal of Child and Adolescent Psychopharmacology coverage includes:
New drugs and treatment strategies including the use of psycho-stimulants, selective serotonin reuptake inhibitors, mood stabilizers, and atypical antipsychotics
New developments in the diagnosis and treatment of ADHD, anxiety disorders, schizophrenia, autism spectrum disorders, bipolar disorder, eating disorders, along with other disorders
Reports of common and rare Treatment Emergent Adverse Events (TEAEs) including: hyperprolactinemia, galactorrhea, weight gain/loss, metabolic syndrome, dyslipidemia, switching phenomena, sudden death, and the potential increase of suicide. Outcomes research.