@article{5167de00f8a7464db211acad7d448b53,
title = "Best practices in fragile X syndrome treatment development",
abstract = "Preclinical studies using animal models of fragile X syndrome have yielded several agents that rescue a wide variety of phenotypes. However, translation of these treatments to humans with the disorder has not yet been successful, shedding light on a variety of limitations with both animal models and human trial design. As members of the Clinical Trials Committee of the National Fragile X Foundation, we have discussed a variety of recommendations at the level of preclinical development, transition from preclinical to human projects, family involvement, and multi-site trial planning. Our recommendations are made with the vision that effective new treatment will lie at the intersection of innovation, rigorous and reproducible research, and stakeholder involvement.",
keywords = "Best practices, Clinical trials, Fragile X syndrome, Treatment development",
author = "Erickson, {Craig A.} and Kaufmann, {Walter E.} and Budimirovic, {Dejan B.} and Ave Lachiewicz and Barbara Haas-Givler and Miller, {Robert M.} and {Dixon Weber}, Jayne and Leonard Abbeduto and David Hessl and Hagerman, {Randi J.} and Elizabeth Berry-Kravis",
note = "Funding Information: Such multi-site work should build upon existing FXS centers and clinics with content expertise. Luckily, over a decade of multi-site trial projects in our field has laid a foundation for large-scale trials that has united clinical centers with content expertise with best trial practices and the standard operating procedures of good clinical research. While initial site qualification efforts and trainings should involve FXS content expertise, ongoing support of fidelity of study execution across sites is essential. There are also great opportunities for centralized future potential support of recruitment and retention efforts in large-scale projects given the infrastructure within the FXCRC supported by the National Fragile X Foundation. Publisher Copyright: {\textcopyright} 2018 by the authors. Licensee MDPI, Basel, Switzerland.",
year = "2018",
month = dec,
doi = "10.3390/brainsci8120224",
language = "English (US)",
volume = "8",
journal = "Brain Sciences",
issn = "2076-3425",
publisher = "Multidisciplinary Digital Publishing Institute (MDPI)",
number = "12",
}