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ULTEVURSEN

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.

Table of contents

Clinical trials overview

Two interventional studies are investigating ULTEVURSEN in people with retinitis pigmentosa due to mutations in exon 13 of the USH2A gene.[1][2] 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.[1][2]

The two studies have the same main goal: to evaluate efficacy after 24 months of treatment.[1][2] Efficacy means whether the treatment helps improve or slow the disease in the way the study is trying to measure.[1][2]

Study design and control groups

Both trials are randomized, double-masked, and sham-controlled.[1][2] 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.[1][2]

The intervention groups include ULTEVURSEN at 60 micrograms and 180 micrograms, both given by intravitreal use, which means into the eye.[1][2] The sham group undergoes a procedure that closely mimics the active injection but with no penetration of the globe.[1][2]

Who was studied

The target population in both trials is subjects with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene.[1][2] This means the studies are focused on a specific inherited form of eye disease rather than all types of vision loss.[1][2]

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.[1][2]

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.[1][2] Baseline means the starting point before treatment, and EZ width is a retinal measurement used to follow changes over time.[1][2]

SD-OCT is an imaging test that gives detailed pictures of the retina, the light-sensitive layer at the back of the eye.[1][2] In simple terms, the studies are using eye scans to see whether ULTEVURSEN changes a specific part of the retina over 24 months.[1][2]

Trial status and enrollment

One study, NCT05158296, is listed as Withdrawn and planned to enroll 75 participants.[1] The other study, NCT06627179, is listed as Authorised and planned to enroll 81 participants.[2]

Even though the studies are similar in design, the status shows that they are at different points in the research process.[1][2] This helps readers understand that trial records can change over time, and not every planned study continues to completion.[1][2]

Key terms explained

  • Retinitis pigmentosa is a group of inherited eye diseases that can slowly reduce vision over time.[1][2]

  • USH2A gene is the gene named in these trials, and the studies focus on people with mutations in exon 13 of this gene.[1][2]

  • Sham procedure means a procedure that looks like treatment but does not include the active study injection.[1][2]

  • Tolerability means how well people can handle the study treatment during the trial.[1][2]

  • Enrollment is the planned number of people who may join a study.[1][2]

Trial ID Phase Condition studied Status Enrollment
NCT05158296 Phase 2 Retinitis Pigmentosa due to mutations in exon 13 of the USH2A gene Withdrawn 75
NCT06627179 Phase 2 Retinitis Pigmentosa due to mutations in exon 13 of the USH2A gene Authorised 81

FAQ

  • What condition is being studied in ULTEVURSEN trials? The trials are studying retinitis pigmentosa, a group of inherited eye diseases that affect vision over time. In these studies, the condition is linked to mutations in exon 13 of the USH2A gene.
  • Who can take part in these studies? The target population is subjects with retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. The trial data do not give more detailed participation rules in the information provided.
  • What phase are the ULTEVURSEN trials? Both trials are Phase 2 studies. Phase 2 usually means the treatment is being studied in a larger group to learn more about its effects and safety.
  • What are the trials trying to measure? The main outcome is the annualized percent change from baseline in EZ width measured by SD-OCT up to Month 24. EZ width is a retinal measurement, and SD-OCT is an imaging test that takes detailed pictures of the eye.
  • Are the trials comparing ULTEVURSEN with anything else? Yes. The studies are double-masked, randomized, and sham-controlled. That means some people receive ULTEVURSEN and others receive a sham procedure that looks like treatment but does not involve the active injection.

Ongoing Clinical Trials on ULTEVURSEN

  • Study of ultevursen treatment in patients with retinitis pigmentosa caused by mutations in USH2A gene exon 13

    Recruiting

    2 1
    Investigated drugs:
    Belgium Denmark France Germany Italy The Netherlands

  • Study on the Effectiveness and Safety of Ultevursen for Patients with Retinitis Pigmentosa Due to USH2A Gene Mutations

    Not yet recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy The Netherlands

Glossary

  • Retinitis pigmentosa: A group of inherited eye diseases that slowly damage the retina and can lead to vision loss.
  • USH2A gene: A gene linked to a form of inherited retinitis pigmentosa when it has certain mutations.
  • Exon 13: A specific part of the USH2A gene. The trials focus on people with mutations in this part.
  • Phase 2: A study stage that looks more closely at how well a treatment works and how safe it is in a larger group.
  • Interventional study: A study where researchers give a treatment or procedure and compare results between groups.
  • Randomized: People are assigned to study groups by chance, not by choice.
  • Double-masked: Neither the participants nor the study staff know who gets the active treatment, which helps reduce bias.
  • Sham-controlled: One group receives a procedure that looks like treatment but does not include the active study drug.
  • Spectral-domain optical coherence tomography (SD-OCT): An eye imaging test that gives detailed pictures of the retina.
  • EZ width: A measurement seen on retinal imaging that the trials use to track changes over time.
  • Baseline: The starting point before treatment or study follow-up begins.
  • Enrollment: The number of participants planned for a study.

References