Antisense Oligonucleotide
Ultevursen for Vision Impairments
Shiley Eye Institute - UC San Diego, San Diego, CA
Targeting 11 different conditionsUltevursen +1 morePhase 2 & 3Waitlist AvailableResearch Sponsored by ProQR TherapeuticsEligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Clinical presentation consistent with RP with Usher syndrome type 2 or nonsyndromic form of RP (NSRP), based on ophthalmic, audiologic, and vestibular examinations.
Must not have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up27 months
Awards & highlights
Study Summary
This trial will test if a new treatment for Retinitis Pigmentosa is safe and effective.
Eligible Conditions
- Eye Diseases
- Usher Syndrome
- Vision Impairment
- Deafblind
- Retinitis Pigmentosa
- Genetic Disorders
- Retinal Disease
- Congenital Eye Disorders
Eligibility Criteria
Inclusion Criteria
You will be eligible if you check “Yes” for the criteria belowSelect...
You have vision and hearing loss caused by a genetic condition called Usher syndrome type 2 or a form of vision loss called nonsyndromic retinitis pigmentosa.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 27 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~27 months
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 moreOther 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 site
Who is running the clinical trial?
ProQR TherapeuticsLead Sponsor
11 Previous Clinical Trials
216 Total Patients Enrolled
6 Trials studying Eye Diseases
96 Patients Enrolled for Eye Diseases
ProQR Clinical Trial ManagerStudy Director
ProQR Therapeutics3 Previous Clinical Trials
36 Total Patients Enrolled
3 Trials studying Eye Diseases
36 Patients Enrolled for Eye Diseases
ProQR Medical MonitorStudy Director
ProQR Therapeutics7 Previous Clinical Trials
96 Total Patients Enrolled
6 Trials studying Eye Diseases
96 Patients Enrolled for Eye Diseases
Media Library
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?
Retina Foundation of the Southwest
University of Miami, Bascom Palmer Eye Institute
Emory Eye Center
Other
How many prior treatments have patients received?
0
Why did patients apply to this trial?
I'm hoping this clinical trial can help me with my usher syndrome. I’ve had rp for roughly 12-13 years and it’s getting bad and affecting my everyday life.
Patient
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 🙏.
Patient
I am 34 years old and I am diagnosed with Usher syndrome 1 year ago. I have problems with night vision. I am applying looking for some opportunity to slow down or improve my condition.
Patient
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?
Patient