We are thrilled to launch RUSH1F, the Usher 1F natural history study in partnership with Foundation Fighting Blindness, the world’s leading private funding source for retinal degenerative research. Additional funding for the project will be provided by the Marjorie C. Adams Trust. We are excited and grateful to Foundation Fighting Blindness for the opportunity to partner to achieve this critical milestone for patients with Usher 1F. 

The RUSH1F natural history study will follow 40 individuals with USH1F who are 8 years of age or older, assessing changes in their vision and retinal structure over four years. The primary goals of the study are to identify outcome measures for future clinical trials of emerging therapies. The Jaeb Center for Health Research in Tampa, Florida, will serve as the coordinating center for the study. Dr. Katarina Stingl, University of Tübingen in Germany, will serve as the chair of the international multicenter clinical trial group. “Genetic diseases with multiple sensory impairment such as Usher syndrome are the most common cause of combined deafness and blindness in adults. It is very important that in this upcoming trial we are able to provide clinical knowledge about this very rare disease. Only by means of international cooperation will we be able to progress to future research that can try to save the vision of affected persons with rare diseases,” says Dr. Stingl. 

“We are delighted to partner with the Usher 1F Collaborative and the Marjorie C. Adams Trust to launch a natural history study that will ultimately help advance clinical research for emerging therapies,” says Todd Durham, PhD, vice president of clinical development at the Foundation Fighting Blindness. “In a short period, the Usher 1F Collaborative has been very successful in moving the field forward through their funding of translational research to develop preclinical models of Usher 1F. It is exciting for us to be a part of a family-driven collaboration to expand disease knowledge and the potential for getting more treatments into the clinic.”

The overall cost of the USH1F natural history study will be more than $2 million with Usher 1F Collaborative providing $1 million in support and the Marjorie C. Adams Trust contributing $115,000 in funding. Foundation Fighting Blindness will provide more than $1 million to cover the remaining costs.

Investigators at 10 centers began enrolling study participants in May 2021. Information regarding the trial can be found at clinicaltrials.gov (NCT04765345) and the study protocol can be accessed on the Consortium’s public website. Individuals with USH1F who are interested in participating in this study are encouraged to contact staff at the Jaeb Center by email at ffb@jaeb.org or by phone at 813-975-8690. We encourage everyone with Usher 1F who meets the criteria to sign up for the study so that we will have the required data for a future clinical trial for everyone with Usher 1F.