UniRare Now Enrolling USH1B Patients!
UniRare is a natural history study of vision loss progression. It is a critical and necessary step in supporting the approval of upcoming clinical trials and future therapies. Enrollments have already started at several clinics across the country, and more clinics will begin enrolling throughout the year.
This study, organized and led by the FFB, is prioritizing enrollment for Usher syndrome type 1B. The study will last for 4 years, and participants will only need to visit their local clinic once a year for non-invasive testing; similar to the imaging and testing that occurs at routine retinal specialist visits.
Why is this study so important for finding a treatment?
Natural history studies help researchers and organizations like the FDA understand how vision loss progresses over time. By collecting data on how quickly vision worsens, we can measure how well treatments work in clinical studies. This information is crucial for deciding if a treatment is effective and if it will ultimately progress through clinical trials and be approved by the FDA.
We have talked to many scientists and companies who are trying to find new treatments for retinal diseases. One of the biggest challenges they face is the lack of information on how these diseases progress naturally. Without this information they will not invest their time and money into discovering new treatments and funding clinical trials.
UniRare information packet (Downloadable PDF)
The PDF packet (click on image) will provide information about the study and answer common questions, but please reach out if you have any additional questions. To view the official study directly, visit: Universal Rare Gene Study at the NIH clinicaltrials.gov website.
Next Steps - how to enroll
On the PDF above, or on the clinicaltrials.gov website, find and contact your nearest participating clinic to see if they have begun enrolling, or when they will. Ask to be put on their list and/or follow up periodically
If your location is enrolling, you'll want to schedule a “Registry Appointment”
The initial "Registry Appointment" cost will be covered by your insurance, all other appointments associated with the study are free. If you need to travel from afar to reach the nearest participating clinic, those expenses will be covered as well.
If you have questions, contact your retinal specialist or Uni-Rare Coordinating Center, 813-975-8690, ffb@jaeb.org
We need your participation
This study is a vital step in the journey toward treatments that can slow down, stop, or even restore vision loss. Clinical trials like Nacuity, Endogena, SparingVision - for retinitis pigmentosa are just around the corner, and trials specifically targeting USH1B, are not far behind. Alberto Auricchio from Naples will begin enrolling USH1B patients this year for a new USH1B trial in Italy, and Atsena (Shannon Boye) hopes to start clinical trials in the United States in 2-3 years. However, without data from natural history studies to show that these treatments work, clinical trials will not make it past the middle phases of testing, and they will be – and have been – canceled because of it.
We strongly encourage anyone who wants to find a treatment for Usher syndrome to enroll and participate in this study. Without the support and involvement from our rare community, whom these treatments are intended for, it will be impossible to discover and fund treatments.
It's important to note that participating in this study does not exclude you from also being accepted and participating in clinical trials. In fact, in some cases, being part of a natural history study can increase your chances of being accepted into a clinical trial, depending on the enrollment criteria.
We hope you’ll consider joining the UniRare study to help us find treatments for USH1B. We also need your help spreading this information by sharing with other families and organizations, and through your social media channels. If you have any questions, do not hesitate to reach out: justin@savesightnow.org