Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degene… (NCT07681778) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
United States10 participantsStarted 2026-09-01
Plain-language summary
This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to severe vision loss. The treatment is delivered as a one-time injection (300 µL) into the retina (subretinal space) of the worse-seeing eye, using a method similar to approved gene therapies like Luxturna. The study is designed to evaluate safety and effectiveness at two dose levels (1E11 and 3E11 viral genomes per eye) in small groups of 5 participants. Each group begins cautiously with 2 adults (age ≥18), treated at least one month apart, followed by FDA review before allowing adolescents (ages 12-17) to participate. An independent monitoring committee (IDMC) oversees safety throughout. After 3 adolescents are treated and followed for 3 months, the committee reviews all data to decide whether to move to a higher dose. However, if the lower dose (1E11 vg/eye) shows strong effectiveness in the first group, the study may expand by treating more adolescents at that same dose instead of increasing it further.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Age ≥18 years (adult participants) or 12-17 years (adolescent participants) at the time of consent/assent.
* Provide written informed consent and/or assent prior to any study procedures.
* Willing to adhere to the clinical protocol and follow directions of the Investigator regarding post-surgery restrictions.
* Are a good candidate for surgery, per the Investigator's judgment.
* Have LCA with autosomal-recessive RDH12 mutation(s), confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory. Historic testing, up to 15 years prior to date of consent, may be considered.
* Clinical diagnosis of LCA with RDH12 mutation(s), in the judgment of the Investigator.
* BCVA 20/200 (1.0 logarithm of the minimum angle of resolution \[logMAR\]) or worse for the sentinel adult in each cohort; BCVA 20/40 (0.5 logMAR) or worse for all subsequent participants in each cohort.
Exclusion Criteria:
* Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year after IMP administration.
* Men who are unwilling to use effective contraception from Screening through 180 days after IMP administration.
* Have an ocular infection, a pre-existing eye condition, or a complicating systemic disease that could preclude the planned surgery or any future ocular surgery. This includes individuals who are immunocompromised and/or on continuous systemic immunosuppressive therapy.
*…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of dose-limiting toxicity (DLT) events at the proposed doses
Timeframe: 5 Years
2
Number and severity of procedure-related AEs
Timeframe: 5 Years
3
Number and severity of AEs related to OPGx-RDH12
Timeframe: 5 Years
4
Qualitative assessment of cross-sectional spectral-domain optical coherence tomography (SD-OCT), fundus photography, and fundus autofluorescence (FAF) images