Safety and tolerability of a single retina gene therapy for RDH12-related LCA

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 is a multicenter, open-label, dose-escalation Phase 1b/2a study of OPGx-RDH12 delivered by unilateral subretinal injection. Two dose levels (1E11 and 3E11 viral genomes per eye) are tested in up to two cohorts of five participants each, focusing on safety and preliminary effectiveness in RDH12-related LCA. Initial enrollment includes adults (18+) with potential expansion to adolescents (12-17) after a safety review by an independent monitoring committee and regulatory feedback. The treatment eye is selected based on worse visual function, and a standard ocular delivery technique used with approved gene therapies guides administration. An independent data monitoring committee oversees safety throughout the trial.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age ≥18 years (adult participants) or 12-17 years (adolescent participants) at the time of consent/assent.
* Provide written informed consent and/or assent prior to any study procedures.
* Willing to adhere to the clinical protocol and follow directions of the Investigator regarding post-surgery restrictions.
* Are a good candidate for surgery, per the Investigator's judgment.
* Have LCA with autosomal-recessive RDH12 mutation(s), confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory. Historic testing, up to 15 years prior to date of consent, may be considered.
* Clinical diagnosis of LCA with RDH12 mutation(s), in the judgment of the Investigator.
* BCVA 20/200 (1.0 logarithm of the minimum angle of resolution \[logMAR\]) or worse for the sentinel adult in each cohort; BCVA 20/40 (0.5 logMAR) or worse for all subsequent participants in each cohort.

Exclusion Criteria:

* Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year after IMP administration.
* Men who are unwilling to use effective contraception from Screening through 180 days after IMP administration.
* Have an ocular infection, a pre-existing eye condition, or a complicating systemic disease that could preclude the planned surgery or any future ocular surgery. This includes individuals who are immunocompromised and/or on continuous systemic immunosuppressive therapy.
* Have a past or current condition that may preclude participation in the study, interfere with outcome measure testing or test results, or otherwise make the potential participant unsuitable for the study.
* Have previously received gene therapy of any kind.
* In either eye, have undergone intraocular surgery within 90 days prior to planned IMP administration or have active inflammation at Screening resulting from prior ocular surgery.
* Have used any investigational device or investigational drug within 90 days (or 5 half-lives of the drug, whichever is longer) prior to planned IMP administration or intend to participate in another drug or device study during the same period as the current study.
* Have received or plan to receive a vaccination within 6 weeks prior to or 6 weeks after IMP administration. Note: For the influenza vaccine, the exclusionary period is shorter: 2 weeks prior to and 5 weeks after IMP administration (i.e., during steroid treatment).
* Have received anticoagulant therapy within 2 weeks prior to planned IMP administration.
* Currently use medications that are potentially neuroprotective/beneficial or retinotoxic.
* Are incapable of performing visual function testing (e.g., FST), with or without assistance, for reason other than poor vision.
* Have any contraindication to a course of oral steroids, in the opinion of the Investigator.
* Have a known history of hypersensitivity to constituents or excipients in the pharmaceutical formulation of the IMP.
* Have a known or active infection of human immunodeficiency virus (HIV) or hepatitis B or C virus.
* Have a known or active infection of herpes simplex virus with ocular manifestations.
* Are an employee of the Sponsor or a relative of the Investigator or investigative site staff.

Where it's running

• Retina Consultants of Texas & Retina Group Inc.
  Houston, Texas, United States Recruiting
• Associated Retina Consultants
  Phoenix, Arizona, United States Not yet recruiting
• Perelman School of Medicine, University of Pennsylvania
  Philadelphia, Pennsylvania, United States Not yet recruiting
• Retina Foundation of the Southwest
  Dallas, Texas, United States Not yet recruiting

Study contacts

• Study coordinator: Study Director
• Email: ct.gov_inquiries@opusgtx.com
• Phone: 984-884-6030

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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.