Gene therapy for inherited retinal degeneration caused by LCA5 mutations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety and preliminary efficacy of a gene therapy called OPGx-001 delivered via subretinal injection in patients with inherited retinal degeneration due to mutations in the LCA5 gene. It is a non-randomized, open-label study that will escalate doses across three cohorts, starting with a low dose and moving to intermediate and high doses based on safety data reviewed by an independent monitoring committee. The goal is to assess how well this treatment can improve vision in affected individuals.

Why it matters

Who can join: the full rules

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.

Where it's running

• University of Pennsylvania Perelman School of Medicine
  Philadelphia, Pennsylvania, United States Recruiting
• Retina Foundation of the Southwest
  Dallas, Texas, United States Recruiting

Study contacts

• Study coordinator: Tomas Aleman, M.D.
• Email: ct.gov_inquiries@opusgtx.com
• Phone: 1 (215) 662-6396

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• OPGx-BEST1 subretinal gene therapy for Best vitelliform macular dystrophy and autosomal-recessive bestrophinopathy

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  Recruiting at 4 sites Phase 1/2 Opus Genetics, Inc · Los Angeles, California and 4 other locations · NCT07185256

• Phentolamine eye drops for adults with reduced night vision after keratorefractive surgery

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  Recruiting at 20 sites Phase 3 Opus Genetics, Inc · Chandler, Arizona and 19 other locations · NCT07140783

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.