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Antisense Oligonucleotide

Ultevursen for Vision Impairments

Phase 2 & 3
Waitlist Available
Research Sponsored by ProQR Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female, ≥ 18 years of age OR a minor (12 to < 18 years) with permission from a parent or legal guardian. The lower age limit for pediatric populations is subject to local regulatory and ethics committee requirements.
An adult willing to comply with the protocol, follow study instructions, attend study visits as required and willing and able to complete all study assessments. OR A minor able to complete all study assessments and comply with the protocol and has a parent or caregiver willing and able to follow study instructions, and attend study visits with the subject as required.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 27 months
Awards & highlights

Summary

This trial will test if a new treatment for Retinitis Pigmentosa is safe and effective.

Who is the study for?
This trial is for adults and minors with parental consent who have Retinitis Pigmentosa due to USH2A gene mutations. Participants must have a certain level of vision in both eyes, no treatment history with genetic or stem-cell therapy, and not be allergic to the study medication components.Check my eligibility
What is being tested?
The trial tests Ultevursen's effectiveness, safety, and tolerability when injected into the eye compared to a sham procedure. It targets patients with specific genetic mutations affecting their vision.See study design
What are the potential side effects?
While side effects are not detailed here, similar treatments may cause eye discomfort or inflammation, temporary visual disturbances post-injection, potential allergic reactions, or increased intraocular pressure.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 or older, or between 12 and 17 with parental consent.
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I am willing and able to follow the study's requirements and attend all visits.
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I have RP with Usher syndrome type 2 or a form of RP without other symptoms, confirmed by eye and ear tests.
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I have a specific genetic mutation in the USH2A gene.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~27 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 27 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Mean change from baseline in BCVA
Secondary outcome measures
Change from baseline in Full-field Stimulus Threshold (FST)
Change from baseline in Low Luminance Visual Acuity (LLVA)
Change from baseline in Microperimetry
+6 more
Other outcome measures
Change from baseline in mobility course score

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Ultevursen 60/60 µgExperimental Treatment1 Intervention
60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
Group II: Ultevursen 180/60 µgExperimental Treatment1 Intervention
180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
Group III: Sham-procedurePlacebo Group1 Intervention
Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Gene therapy for Retinitis Pigmentosa (RP) aims to correct or compensate for the genetic mutations causing the disease. Treatments like Ultevursen, which target mutations in exon 13 of the USH2A gene, work by delivering a functional copy of the gene or modifying the existing gene to restore normal function. This approach is significant for RP patients because it addresses the root cause of the disease at the molecular level, potentially halting or reversing the progression of vision loss. By targeting specific genetic mutations, gene therapy offers a personalized treatment option that can lead to more effective and lasting outcomes compared to traditional therapies.

Find a Location

Who is running the clinical trial?

ProQR TherapeuticsLead Sponsor
11 Previous Clinical Trials
216 Total Patients Enrolled
4 Trials studying Retinitis Pigmentosa
57 Patients Enrolled for Retinitis Pigmentosa
ProQR Clinical Trial ManagerStudy DirectorProQR Therapeutics
3 Previous Clinical Trials
36 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
36 Patients Enrolled for Retinitis Pigmentosa
ProQR Medical MonitorStudy DirectorProQR Therapeutics
7 Previous Clinical Trials
96 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
36 Patients Enrolled for Retinitis Pigmentosa

Media Library

Ultevursen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT05158296 — Phase 2 & 3
Retinitis Pigmentosa Research Study Groups: Ultevursen 60/60 µg, Ultevursen 180/60 µg, Sham-procedure
Retinitis Pigmentosa Clinical Trial 2023: Ultevursen Highlights & Side Effects. Trial Name: NCT05158296 — Phase 2 & 3
Ultevursen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05158296 — Phase 2 & 3
Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT05158296 — Phase 2 & 3
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