Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

Last updated: June 26, 2024
Sponsor: Opus Genetics, Inc
Overall Status: Active - Recruiting

Phase

1/2

Condition

Macular Degeneration

Retina

Myopic Macular Degeneration

Treatment

AAV8.hLCA5

Clinical Study ID

NCT05616793
OPGx-LCA5-1001
  • Ages > 13
  • All Genders

Study Summary

The goal of this clinical trial is 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Are willing and able to provide written informed consent (ICF) and, whereappropriate, willing to sign an assent prior to any study procedures.

  2. Are willing to adhere to the clinical protocol and able to perform testingprocedures.

  3. Participants must be at least 13 years of age at consent.

  4. Carry disease-causing biallelic LCA5 gene mutations determined by a ClinicalLaboratory Improvement Amendments (CLIA) certified laboratory.

  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 betreated

  6. Show evidence of detectable photoreceptors by Spectral Domain Optical CoherenceTomography (SD-OCT)

  7. Participant is a good candidate for surgery per investigator judgement

  8. Participant agrees to follow direction of investigator regarding restrictionspost-surgery.

Exclusion

Exclusion Criteria:

  1. Individuals of childbearing potential (male and female) who are pregnant orunwilling to use effective contraception for the duration of the study, includingbarrier methods for the first year after investigational product (IP)administration.

  2. Pre-existing eye conditions or complicating systemic diseases that would precludethe planned surgery. This includes individuals who are immunocompromised.

  3. History of intraocular surgery for either eye within 6 months prior to planned IPadministration.

  4. Have previously received gene therapy.

  5. Have used any investigational drug or device within 90 days or 5 estimatedhalf-lives of treatment, whichever is longer or plan to participate in another studyof drug or device during the study period.

  6. History of disease which may preclude the participant from participation, or whichmay interfere with outcome measure testing or test results.

  7. Incapable of performing visual function testing (e.g., FST testing) for reasonsother 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 completefollow-up examinations during the study and, in the opinion of the Investigator,makes the potential participant unsuitable for the study.

Study Design

Total Participants: 15
Treatment Group(s): 1
Primary Treatment: AAV8.hLCA5
Phase: 1/2
Study Start date:
June 15, 2023
Estimated Completion Date:
January 30, 2028

Study Description

This is a non-randomized, open-label, phase 1/2 dose-escalation study evaluating 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).

Connect with a study center

  • University of Pennsylvania Perelman School of Medicine

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

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