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

Investigation of Safety and Effects of Gene Therapy Administered Beneath the Retina for LCA5-Associated Retinal Degeneration

Recruiting
4 years or above
All
Phase 1/2
22 participants needed
1 Location

Brief description of study

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 of study

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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: LCA5
  • Age: 4 years or above
  • Gender: All

Inclusion Criteria:

  1. Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures.
  2. Are willing to adhere to the clinical protocol and able to perform testing procedures.
  3. In part A participants must be 13 years of age or older at consent, for Part B, participants must be 4 years of age or older at consent with the ability to conduct the MLoMT.
  4. Carry disease-causing biallelic LCA5 gene mutations determined by a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (historic testing up to 15 years from date of consent can be considered).
  5. Visual acuity: BCVA < 20/80 on the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity chart (modified for low vision participants) in the eye to be treated
  6. Show evidence of detectable photoreceptors by Spectral Domain Optical Coherence Tomography (SD-OCT)
  7. Participant is a good candidate for surgery per investigator judgement
  8. Participant agrees to follow direction of investigator regarding restrictions post-surgery (Part A only).

Exclusion Criteria:

  1. Women who are pregnant or individuals (women of childbearing potential and men) unwilling to use effective contraception for the duration of the study, including barrier methods for the first year after investigational product (IP) administration (Part A only).
  2. Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery. This includes individuals who are immunocompromised.
  3. History of intraocular surgery for either eye within 6 months prior to planned IP administration (Part A only).
  4. Have previously received gene therapy.
  5. Have used any investigational drug or device within 90 days or 5 estimated half-lives of treatment, whichever is longer or plan to participate in another study of drug or device during the study period.
  6. History of disease which may preclude the participant from participation, or which may interfere with outcome measure testing or test results.
  7. Incapable of performing visual function testing (e.g., FST testing) for reasons other than poor vision.
  8. Any absolute contraindication to a course of oral steroids.
  9. Any other condition that would not allow the potential participant to complete follow-up examinations during the study and, in the opinion of the Investigator, makes the potential participant unsuitable for the study.

This study investigates the safety and initial effects of a gene therapy for patients with a type of inherited eye disease called LCA5-associated inherited retinal degeneration (LCA5-IRD). The purpose is to understand how this condition progresses over time and to see how patients respond to different doses of an investigational gene therapy.

Participants in this study will receive a subretinal injection of the investigational gene therapy. The study is divided into different arms, with some participants receiving varying doses of the therapy, while others are observed without treatment to study the natural progression of the disease. The study will monitor participants' eye health and vision over time to assess safety and effects.

  • Who can participate: Participants must be at least 13 years old for part A and 4 years old for part B, with specific genetic mutations in the LCA5 gene. They must have a certain level of vision and be able to undergo testing procedures.
  • Study details: Participants will undergo subretinal injections of the investigational therapy and be monitored for safety and effects. Some participants will not receive treatment initially to observe the natural disease progression.
  • Study timelines: The study will last 6 months.
Updated on 25 Aug 2026. Study ID: 22-1661

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