Psychopharmacology Clinical Trial Recruitment Challenges in Neurogenetic Syndromes: Lessons from an Open-Label Trial of Fluoxetine in Down Syndrome

<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.