← Back to Search

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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Summary

This trial is testing a new gene therapy injected under the retina to help people with a specific genetic eye condition that causes vision loss. The therapy aims to replace faulty genes with healthy ones to improve vision. Mutations in the RPE65 gene are one of the causes of RP and Leber congenital amaurosis (LCA), a form of RP present at birth.

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.
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.
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 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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in retinal thickness
Secondary study objectives
Change from baseline to month 12 in retinal sensitivity
Change from baseline to month 12 transient pupillary light reflexes (TPLR)
Other study objectives
Chromatic referential looking
Full-field chromatic sensitivity testing (FST)
Kinetic perimetry
+2 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: Dose Group 3Experimental Treatment1 Intervention
A single, unilateral, subretinal injection of high dose (1E11 vg/eye) OPGx-001 is injected into two LCA5 adults (18 yo or above) in a sentinel fashion. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, a subsequent LCA5 adolescent (13-17) may be treated in a sentinel fashion, with a unilateral, subretinal injection of OPGx-001. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, two remaining adolescents (13-17) are eligible to be treated with a single, unilateral, subretinal injection of a high dose of OPGx-001. Total cohort size is 5 (2 adults and 3 adolescents).
Group II: Dose Group 2Experimental Treatment1 Intervention
A single, unilateral, subretinal injection of an intermediate dose (3E10 vg/eye) OPGx-001 is injected into two LCA5 adults (18 yo or above) in a sentinel fashion. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, a subsequent LCA5 adolescent (13-17) may be treated in a sentinel fashion, with a unilateral, subretinal injection of OPGx-001. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, two remaining adolescents (13-17) are eligible to be treated with a single, unilateral, subretinal injection of an intermediate dose of OPGx-001. Total cohort size is 5 (2 adults and 3 adolescents).
Group III: Dose Group 1Experimental Treatment1 Intervention
A single, unilateral, subretinal injection of low dose (1E10 vg/eye) OPGx-001 is injected into two LCA5 adults (18 yo or above) in a sentinel fashion. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, a subsequent LCA5 adolescent (13-17) may be treated in a sentinel fashion, with a unilateral, subretinal injection of OPGx-001. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, two remaining adolescents (13-17) are eligible to be treated with a single, unilateral, subretinal injection of a low dose of OPGx-001. Total cohort size is 5 (2 adults and 3 adolescents).

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?

University of PennsylvaniaOTHER
2,063 Previous Clinical Trials
42,711,897 Total Patients Enrolled
1 Trials studying Leber Congenital Amaurosis
15 Patients Enrolled for Leber Congenital Amaurosis
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
~2 spots leftby Dec 2024