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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

Study 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

Find a Location

Who is running the clinical trial?

ProQR TherapeuticsLead Sponsor
11 Previous Clinical Trials
216 Total Patients Enrolled
ProQR Clinical Trial ManagerStudy DirectorProQR Therapeutics
3 Previous Clinical Trials
36 Total Patients Enrolled
ProQR Medical MonitorStudy DirectorProQR Therapeutics
7 Previous Clinical Trials
96 Total Patients Enrolled

Media Library

Ultevursen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT05158296 — Phase 2 & 3
Vision Impairment Research Study Groups: Ultevursen 180/60 µg, Ultevursen 60/60 µg, Sham-procedure
Vision Impairment 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
Vision Impairment Patient Testimony for trial: Trial Name: NCT05158296 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can new patients sign up for this clinical trial?

"Unfortunately, this specific study is not recruiting at the moment. According to clinicaltrials.gov, it was last edited on September 15th, 2022 and originally posted December 8th, 2021. There are 1258 other trials that may be of interest and are currently looking for patients."

Answered by AI

How many different hospitals or research facilities are conducting this experiment?

"Patients are being accepted at 11 different locations for this clinical trial. Some of these sites include Columbia University in New york, New York, Shiley Eye Institute - UC San Diego in San Diego, California, and University of California, San Francisco in San Francisco, Florida."

Answered by AI

What are the goals that this clinical trial is hoping to achieve?

"The primary goal of this experiment, which will be conducted over the course of 18 months and pit treatment against a sham procedure, is to evaluate changes in BCVA from baseline. Additionally, we will also be looking at Cmax of ultevursen in serum, change from baseline in ellipsoid zone (EZ) area and width as imaged by spectral domain optical coherence tomography (SD-OCT), and exposure of QR-421a in serum."

Answered by AI

Who else is applying?

What state do they live in?
Georgia
Texas
Puerto Rico
Other
How old are they?
18 - 65
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
What site did they apply to?
Shiley Eye Institute - UC San Diego
Columbia University
University of Miami, Bascom Palmer Eye Institute
Other
How many prior treatments have patients received?
0

Why did patients apply to this trial?

Want to preserve eyesight have crb1 rp. I'm hoping this clinical trial can help me with my usher syndrome.
PatientReceived 2+ prior treatments
I’ve had rp for roughly 12-13 years and it’s getting bad and affecting my everyday life.
PatientReceived 2+ prior treatments
To safe some of my vision and to bring hope for other people who are going through the same issues..
PatientReceived 2+ prior treatments
I really hope that you can give me the opportunity to fight this condition and try to keep the remaining vision i have and hopefully re gain mu vision 🙏.
PatientReceived 2+ prior treatments

What questions have other patients asked about this trial?

Will I receive help for my vision? How long does the whole trial take? Will it improve my vision?
PatientReceived 2+ prior treatments
~19 spots leftby Dec 2024