Purpose of this Study
If you take part in this study, you will visit the clinic at least ten times over about twenty seven months. Most visits will last two days, but some may take three days if needed. You will be placed into one of two groups by chance. One group will receive the study medicine, and the other group will receive a sham injection, which looks like a real injection but has no medicine.
Who Can Participate?
Eligibility
People can join this study if they are older than 8 years or are adults. Both eyes must show signs of a vision disease called retinitis pigmentosa. They cannot have had eye injections in the past.
Age Range
8-110
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study is testing a new medicine called ultevursen. It is designed to help people who have a vision disease called retinitis pigmentosa caused by a specific genetic change called a USH2A exon 13 mutation. Researchers want to learn if this medicine is safe and if it can help slow down or improve vision loss.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene - SB-421a-006 - LUNA
Principal Investigator
Oleg
Alekseev
Protocol Number
PRO00118825
NCT ID
NCT06627179
Phase
II
Enrollment Status
Pending Open to Enrollment