Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
Public ClinicalTrials.gov record NCT07681778. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1b/2a Open-Label, Dose-Exploration Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-RDH12 Administered in Participants With Leber Congenital Amaurosis With Autosomal-Recessive Retinol Dehydrogenase 12 Mutations
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT07681778
- Recruitment status
- Not yet recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 10 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 31, 2026
- Primary completion
- Nov 30, 2032
- Completion
- Jun 30, 2034
- Last update posted
- Jul 14, 2026
2026 – 2034
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Associated Retina Consultants | Phoenix | Arizona | 85020 | — |
| Perelman School of Medicine, University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| Retina Consultants of Texas & Retina Group Inc. | Houston | Texas | 77056 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07681778, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 14, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07681778 live on ClinicalTrials.gov.