Erickson Craig A, Kaufmann Walter E, Budimirovic Dejan B, Lachiewicz Ave, Haas-Givler Barbara, Miller Robert M, Weber Jayne Dixon, Abbeduto Leonard, Hessl David, Hagerman Randi J, Berry-Kravis Elizabeth
Division of Child & Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine, Cincinnati, OH 45219, USA.
Brain Sci. 2018 Dec 15;8(12):224. doi: 10.3390/brainsci8120224.
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.
使用脆性X综合征动物模型的临床前研究已经产生了几种能够挽救多种表型的药物。然而，将这些治疗方法应用于患有该疾病的人类尚未成功，这揭示了动物模型和人体试验设计的各种局限性。作为国家脆性X基金会临床试验委员会的成员，我们已经在临床前开发、从临床前到人类项目的过渡、家庭参与和多中心试验规划等层面讨论了各种建议。我们的建议基于这样一种愿景，即有效的新治疗方法将存在于创新、严谨且可重复的研究以及利益相关者参与的交叉点上。
