What is the mechanism of action of this treatment?

Leber Congenital Amaurosis (LCA) treatments, particularly gene therapy, work by delivering a functional copy of the defective gene directly to retinal cells. For example, the OPGx-001 trial uses viral vectors to introduce a healthy LCA5 gene into the retina. This mechanism is vital as it targets the root cause of the disease, aiming to restore normal gene function and potentially halt or reverse vision loss, offering significant and lasting improvements for patients.

What side effects are associated with this type of intervention?

Gene therapy for Leber Congenital Amaurosis, such as the delivery of the functional LCA5 gene to retinal cells, can have several side effects. Commonly reported side effects include mild to moderate inflammation, which can be managed with corticosteroids. Other potential side effects include retinal detachment, increased intraocular pressure, and cataract formation. In some cases, there may be an immune response to the viral vector used to deliver the gene. Careful dose optimization and monitoring are essential to minimize these risks.

What prior FDA approvals exist?

The FDA has approved gene therapy for Leber Congenital Amaurosis, specifically targeting mutations in the RPE65 gene. Luxturna (voretigene neparvovec-rzyl) is an FDA-approved gene therapy that delivers a functional copy of the RPE65 gene to retinal cells, improving vision in patients with LCA caused by RPE65 mutations. This treatment is similar in approach to the OPGx-001 trial, which aims to deliver a functional LCA5 gene to retinal cells.

What other types of research exist?

Promising alternatives to OPGx-001 for LCA patients include: 1) AAV-based gene therapies targeting other LCA-related genes such as RPE65, CEP290, and GUCY2D, which are in various stages of clinical trials. 2) CRISPR/Cas9 gene editing techniques aimed at correcting specific mutations causing LCA. 3) Stem cell-based therapies that aim to replace damaged retinal cells. These approaches are being actively researched and have shown potential in early studies.

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Gene Therapy

Gene Therapy for Leber Congenital Amaurosis

Phase 1 & 2

Recruiting

Research Sponsored by Opus Genetics, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

Study Summary

This trial tests a gene therapy to help people with an inherited eye disorder that causes vision loss.

Who is the study for?

Adults with inherited retinal degeneration due to LCA5 gene mutations, who have visual acuity less than 20/80 and detectable photoreceptors. Candidates must be able to undergo surgery, follow post-surgery instructions, use effective contraception if of childbearing potential, and commit to the study protocol.Check my eligibility

What is being tested?

The trial is testing a subretinal gene therapy called OPGx-001 for safety and initial effectiveness in treating vision loss caused by LCA5-associated retinal degeneration. It involves surgical delivery of the AAV8.hLCA5 gene therapy directly into the retina.See study design

What are the potential side effects?

Potential side effects may include typical risks associated with eye surgery such as discomfort or infection at the injection site, inflammation inside the eye, changes in intraocular pressure or cataract formation. Gene therapy-specific reactions could also occur.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in retinal thickness (as measured by OCT)

Incidence of dose limiting toxicities

Number of adverse events related to OPGx-001

+1 more

Secondary outcome measures

Change from baseline to month 12 in best corrected visual acuity (BCVA)

Change from baseline to month 12 in dark-adapted transient pupillary light reflexes (TPLR)

Change from baseline to month 12 in oculomotor control and fixation stability

+2 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: Dose Group 3Experimental Treatment1 Intervention

Single, unilateral subretinal administration of a high dose of OPGx-001 to adult participants at least 18 years of age

Group II: Dose Group 2Experimental Treatment1 Intervention

Single, unilateral subretinal administration of an intermediate dose of OPGx-001 to adult participants at least 18 years of age

Group III: Dose Group 1Experimental Treatment1 Intervention

Single, unilateral subretinal administration of a low dose of OPGx-001 to adult participants at least 18 years of age

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Leber Congenital Amaurosis (LCA) treatments, particularly gene therapy, work by delivering a functional copy of the defective gene directly to retinal cells. For example, the OPGx-001 trial uses viral vectors to introduce a healthy LCA5 gene into the retina. This mechanism is vital as it targets the root cause of the disease, aiming to restore normal gene function and potentially halt or reverse vision loss, offering significant and lasting improvements for patients.

Find a Location

Who is running the clinical trial?

Opus Genetics, IncLead Sponsor

Media Library

AAV8.hLCA5 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05616793 — Phase 1 & 2

Leber Congenital Amaurosis Research Study Groups: Dose Group 1, Dose Group 2, Dose Group 3

Leber Congenital Amaurosis Clinical Trial 2023: AAV8.hLCA5 Highlights & Side Effects. Trial Name: NCT05616793 — Phase 1 & 2

AAV8.hLCA5 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05616793 — Phase 1 & 2

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