Actively Recruiting
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
Led by Opus Genetics, Inc · Updated on 2026-02-19
22
Participants Needed
2
Research Sites
261 weeks
Total Duration
On this page
Sponsors
O
Opus Genetics, Inc
Lead Sponsor
U
University of Pennsylvania
Collaborating Sponsor
AI-Summary
What this Trial Is About
The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Funding Source- FDA Office of Orphan Products Development (OOPD).
CONDITIONS
Official Title
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willing and able to provide written informed consent and assent when appropriate
- Willing to follow study procedures and testing requirements
- At least 13 years old for Part A; at least 4 years old for Part B with ability to perform specific vision tests
- Confirmed biallelic disease-causing mutations in the LCA5 gene by a certified laboratory
- Best corrected visual acuity worse than 20/80 in the eye to be treated
- Detectable photoreceptors shown by spectral domain optical coherence tomography
- Considered a suitable candidate for surgery by the investigator
- Agree to follow post-surgery restrictions if applicable (Part A only)
You will not qualify if you...
- Pregnant women or individuals unwilling to use effective contraception during the study (Part A only)
- Pre-existing eye or systemic conditions that prevent planned surgery, including immunocompromised status
- History of intraocular surgery within 6 months before planned treatment (Part A only)
- Previous gene therapy treatment
- Use of investigational drugs or devices within 90 days or 5 half-lives, or participation in another study
- Conditions that prevent participation or interfere with testing
- Unable to perform visual function tests except due to poor vision
- Contraindication to oral steroids
- Any condition making follow-up examinations impossible or unsuitable according to the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
University of Pennsylvania Perelman School of Medicine
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
2
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
Research Team
T
Tomas Aleman, M.D.
CONTACT
M
Mariejel Weber
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
OTHER
Number of Arms
4
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here