The National Fragile X Foundation (NFXF) formed the Clinical Trials Committee to address both the limitations with animal models and human trial design with the goal of improving Fragile X syndrome research and the patient experience. A peer-reviewed paper has just been published that discusses a variety of recommendations at the level of preclinical development, the transition from preclinical to human projects, family involvement, and multi-site trial planning. The recommendations are made with the vision that effective new treatment will lie at the intersection of innovation, rigorous and reproducible research, and stakeholder involvement.

Why is this important to families living with Fragile X?

The NFXF developed a Clinical Trials Committee (CTC) structure made up of Fragile X Clinical and Research Consortium (FXCRC) members, Fragile X syndrome clinicians, expert Fragile X syndrome trialists, outcome measure experts in the field, and family stakeholders to assist and support treatment developments. It is a “one-stop point of contact to interface with industry, academic, and other partners seeking to move forward Fragile X syndrome-focused new treatment development at any stage.

With an increasing number of current and potential trials in Fragile X syndrome research, it will be critical to, “protect patients and also optimize Fragile X syndrome participant resources.” To achieve this, the CTC reviews preclinical data in early stages of drug development and subsequently help with recommendations regarding trial designs, outcome measures, and development strategy for agents showing promise on appropriate rigor preclinical and/or early phase clinical studies.

This paper provides peer-reviewed guidance to researchers so they can correctly design their studies.

Working together, we will have better research and will beat Fragile X.

A special thank you to the authors: Craig A. Erickson, Walter E. Kaufman, Ave Lachiewicz, Barbara Haas-Givler, Robert M. Miller, Jayne Dixon Weber, Leonard Abbeduto, David Hessl, Randi Hagerman, and Elizabeth Berry-Kravis.