Phase 1b/2a Dose Exploration Study to Determine Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Opus Genetics, Inc
Study ID
NCT07681778
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

  • LCA5
  • Leber Congenital Amaurosis
  • Leber Congenital Amaurosis (LCA)

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • OPGx-RDH12 — DRUG
    Experimental gene therapy

Study Details

This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to severe vision loss. The treatment is delivered as a one-time injection (300 µL) into the retina (subretinal space) of the worse-seeing eye, using a method similar to approved gene therapies like Luxturna. The study is designed to evaluate safety and effectiveness at two dose levels (1E11 and 3E11 viral genomes per eye) in small groups of 5 participants. Each group begins cautiously with 2 adults (age ≥18), treated at least one month apart, followed by FDA review before allowing adolescents (ages 12-17) to participate. An independent monitoring committee (IDMC) oversees safety throughout. After 3 adolescents are treated and followed for 3 months, the committee reviews all data to decide whether to move to a higher dose. However, if the lower dose (1E11 vg/eye) shows strong effectiveness in the first group, the study may expand by treating more adolescents at that same dose instead of increasing it further.

Key Dates

Start date
Sep 1, 2026
Status verified
Jun 2026
Primary completion
Dec 31, 2032
Completion
Jul 31, 2034

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: OPGx-RDH12
    Administration of OPGx-RDH12 will occur via a cannula into the subretinal space, using the standard technique for delivery of other adeno-associated virus (AAV) therapies including Luxturna®. A dose of 1E11 vg/eye will be injected sub-retinally one time into the treatment eye. The treatment eye will be the eye with the worst visual function (as determined by visual acuity, full-field sensitivity testing \[FST\] and kinetic perimetry) or the non-dominant eye in cases of bilateral symmetric disease.

Primary Outcome Measure

Number of dose-limiting toxicity (DLT) events at the proposed doses [ Time Frame: 5 Years ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Associated Retina ConsultantsPhoenixArizona85020
Mallory Mintert
[email protected]
Benjamin Bakall, MD (PRINCIPAL_INVESTIGATOR)
Perelman School of Medicine, University of PennsylvaniaPhiladelphiaPennsylvania19104
Tomas Aleman, MD (PRINCIPAL_INVESTIGATOR)
Retina Consultants of Texas & Retina Group Inc.HoustonTexas77056
Kenneth Fan, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Phoenix, AZ

By research site
Associated Retina Consultants· Phoenix, AZPerelman School of Medicine, University of Pennsylvania· Philadelphia, PARetina Consultants of Texas & Retina Group Inc.· Houston, TX

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