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NCT07681778 · ClinicalTrials.gov registry record · Phase 1
Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)
A Phase 1 study, sponsored by Opus Genetics.
- Not yet
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT07681778 is a Phase 1 study that has not yet begun recruiting, run by Opus Genetics. The registered enrollment target is 10 participants.
The verdict
NCT07681778, a Phase 1 study, has not yet begun recruiting, sponsored by Opus Genetics.
- NOT YET RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
Study 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.
Interventions
- DRUG OPGx-RDH12
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2026-09-01 |
| Est. Completion | 2034-07 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07681778
The ClinicalTrials.gov registry entry for NCT07681778 describes a study currently listed as not yet recruiting, categorized as Phase 1. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Opus Genetics, which has 3 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which OPGx-RDH12 is the first listed.
NCT07681778 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT07681778 about?
NCT07681778 is a clinical study titled "Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)". 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 (...
What is the current status of trial NCT07681778?
This trial is currently not yet recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2026-09-01. Estimated completion is 2034-07.
What interventions are being tested in trial NCT07681778?
The interventions under investigation include: OPGx-RDH12 (DRUG).
Who is sponsoring clinical trial NCT07681778?
This trial is sponsored by Opus Genetics, which has 3 total clinical trials registered on ClinicalTrials.gov.
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