In collaboration with Applied Genetic Technologies Corporation (AGTC) and Biogen, the Foundation is seeking males with X-linked retinitis pigmentosa (XLRP) for participation in a survey through the My Retina Tracker Registry (MRTR). The goal is to better understand the impact of vision loss on activities of daily living and perspectives on the risks and benefits of potential treatments.
The results of the survey will inform a Patient-Focused Drug Development Meeting, organized by the Foundation and attended by representatives of the U.S. Food and Drug Administration and affected adults and caregivers of affected minors. The meeting will be scheduled for some time in mid-2022.
The Foundation is seeking 300 males 18-years of age and older with XLRP, registered in MRTR, and who live in the US. Only males are being sought for the survey because XLRP is more predominant and severe in the male population. The participant must have a mutation in the gene RPGR identified as the cause of XLRP. Approximately 10-20 percent of the survey participants will include caregivers of people with XLRP.
Those who have received an experimental treatment (e.g., gene therapy or a cell-based therapy) in a clinical trial or use a retinal prosthesis are not eligible to participate in the survey.
The survey should take about 25 minutes to complete and is designed to be accessible to people with low vision and those who use computer screen readers. Participation in this survey will NOT impact your ability to take part in any other research programs, surveys, or clinical trials.
“The survey is an excellent opportunity for people with XLRP and their caregivers to share their expectations of the benefits of emerging therapies,” says Todd Durham., PhD, senior vice president, clinical development and outcomes research, Foundation Fighting Blindness. “The information collected helps all partners involved in therapy development to gain a better understanding of the role of vision on activities of daily living and understand the needs of the targeted population.”
If you are already registered in MRTR as an XLRP affected man or the caregiver of an affected male child with a variant in the RPGR gene, log in to your account to take the survey.
Questions? E-mail [email protected].