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NCT05616793 · ClinicalTrials.gov registry record · Phase 1
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
A Phase 1 study of LCA5, sponsored by Opus Genetics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 22
- Enrollment target
- 2
- Study locations
NCT05616793: Recruiting Phase 1 study of LCA5, sponsored by Opus Genetics.
NCT05616793 is a Phase 1 study of LCA5 that is actively recruiting participants, run by Opus Genetics. The registered enrollment target is 22 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (63% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05616793, a Phase 1 study of LCA5, is actively recruiting participants, sponsored by Opus Genetics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 22 participants
- Enrollment target
- 2
- Study locations
Study Summary
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).
Primary Outcome
Number of DLT events
Conditions Studied
Interventions
- BIOLOGICAL AAV8.hLCA5
Study Locations (2)
Pennsylvania
- University of Pennsylvania Perelman School of Medicine - Philadelphia
Texas
- Retina Foundation of the Southwest - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2023-06-15 |
| Est. Completion | 2028-06-15 |
| Phase | Phase 1 |
What NCT05616793 shows while recruiting
NCT05616793 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (63% lower).
The record links to 1 condition, with LCA5 appearing as the primary indexed condition, and to 1 intervention - of which AAV8.hLCA5 is the first listed.
NCT05616793 reports a single indexed study location in Pennsylvania, Texas.
Frequently Asked Questions
What is clinical trial NCT05616793 about?
NCT05616793 is a clinical study titled "Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients". 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 Offic...
What is the current status of trial NCT05616793?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 22 participants. The study started on 2023-06-15. Estimated completion is 2028-06-15.
What conditions does trial NCT05616793 study?
This clinical trial studies the following conditions: LCA5.
What interventions are being tested in trial NCT05616793?
The interventions under investigation include: AAV8.hLCA5 (BIOLOGICAL).
Who is sponsoring clinical trial NCT05616793?
This trial is sponsored by Opus Genetics, which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05616793 being conducted?
This trial has 2 study locations across Pennsylvania, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05616793's enrollment target sits among peer trials
22 1601st of 2000 higher than 386 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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