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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
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
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 moreTrial 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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I cannot take oral steroids for medical reasons.I haven't used any experimental drugs or devices recently and don't plan to join another study soon.I haven't had eye surgery in the last 6 months.I have undergone gene therapy before.I am 18 years old or older.I do not have eye conditions or other diseases that would prevent surgery.I am unable to do vision tests for reasons other than poor eyesight.My doctor thinks I am a good candidate for surgery.I have LCA5 gene mutations confirmed by a certified lab.I am not pregnant and willing to use effective birth control during and for a year after the study.
Research Study Groups:
This trial has the following groups:- Group 1: Dose Group 1
- Group 2: Dose Group 2
- Group 3: Dose Group 3
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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