NCT05616793

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients

An Open Label, Dose Exploration, Safety and Tolerability Study of a Subretinal Injection of an OPGx-001 Gene Vector to Participants With LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) With OCncurrent Non-Interventional Follow-Up of Untreated Patients

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 22 (estimated)
• Sponsor: Opus Genetics, Inc · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is a non-randomized, open-label, phase 1/2 dose-escalation study evaluating untreated patients for 6 months and with three doses of OPGx-001 for the treatment of LCA5-IRD. Enrollment will begin with a low-dose of OPGx-001 delivered via single, unilateral subretinal injection (Cohort 1) and proceed to an intermediate dose (Cohort 2) and subsequent high dose (Cohort 3). Escalation to each next cohort will proceed only after review of all data and upon recommendation by an independent data monitoring committee (IDMC). Concurrently, 16 untreated patients will be assessed for 6 months prior to treatment to study the natural course of LCA5-IRD.

Conditions

• LCA5

Interventions

Type	Name	Description
BIOLOGICAL	AAV8.hLCA5	Adeno-associated virus vector expressing human LCA5 gene

Timeline

• Start date: 2023-06-15
• Primary completion: 2028-06-15
• Completion: 2028-06-15
• First posted: 2022-11-15
• Last updated: 2026-02-19

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study