In short
Clinical trials are investigating ULTEVURSEN in people with retinitis pigmentosa linked to mutations in exon 13 of the USH2A gene. These studies are looking at efficacy, safety, and tolerability, and they compare ULTEVURSEN with a sham procedure. The trials focus on adult subjects with this inherited eye disease.
Key points
- ULTEVURSEN is being studied in Phase 2 clinical trials for retinitis pigmentosa linked to mutations in exon 13 of the USH2A gene. Both studies are interventional, randomized, double-masked, and sham-controlled. The main goal is to assess efficacy, safety, and tolerability over 24 months. The key measurement is the change in EZ width on SD-OCT imaging. One trial was withdrawn, and one was authorised.
Clinical trials overview
Two interventional studies are investigating ULTEVURSEN in people with retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. Both studies are Phase 2 trials, which means they are designed to learn more about the treatment in a patient group and to continue checking safety and tolerability.
The two studies have the same main goal: to evaluate efficacy after 24 months of treatment. Efficacy means whether the treatment helps improve or slow the disease in the way the study is trying to measure.
Study design and control groups
Both trials are randomized, double-masked, and sham-controlled. Randomized means participants are assigned to groups by chance, double-masked means neither the participants nor the study staff know who is in which group, and sham-controlled means one group receives a procedure that looks like treatment but does not include the active injection.
The intervention groups include ULTEVURSEN at 60 micrograms and 180 micrograms, both given by intravitreal use, which means into the eye. The sham group undergoes a procedure that closely mimics the active injection but with no penetration of the globe.
Who was studied
The target population in both trials is subjects with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene. This means the studies are focused on a specific inherited form of eye disease rather than all types of vision loss.
The source data do not list more detailed eligibility rules, such as age limits or vision requirements, so only the condition and genetic target can be confirmed from the trial records provided.
Main outcomes and measurements
The primary outcome in both trials is the annualized percent change from baseline in EZ width measured by spectral-domain optical coherence tomography (SD-OCT) up to Month 24. Baseline means the starting point before treatment, and EZ width is a retinal measurement used to follow changes over time.
SD-OCT is an imaging test that gives detailed pictures of the retina, the light-sensitive layer at the back of the eye. In simple terms, the studies are using eye scans to see whether ULTEVURSEN changes a specific part of the retina over 24 months.
Trial status and enrollment
One study, NCT05158296, is listed as Withdrawn and planned to enroll 75 participants. The other study, NCT06627179, is listed as Authorised and planned to enroll 81 participants.
Even though the studies are similar in design, the status shows that they are at different points in the research process. This helps readers understand that trial records can change over time, and not every planned study continues to completion.
Key terms explained
- Retinitis pigmentosa is a group of inherited eye diseases that can slowly reduce vision over time.
- USH2A gene is the gene named in these trials, and the studies focus on people with mutations in exon 13 of this gene.
- Sham procedure means a procedure that looks like treatment but does not include the active study injection.
- Tolerability means how well people can handle the study treatment during the trial.
- Enrollment is the planned number of people who may join a study.