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Gene Therapy Trial Report

Summary

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)


NCTID NCT07681778 (View at clinicaltrials.gov)
Description 🔄
Development Status 🔄 Active
Indication Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA), LCA5
Disease Ontology Term 🔄 DOID:0110330
Drug Product Name 🔄 OPGx-RDH12
Drug Product Description 🔄 AAV8-RK1-hoptRDH12
Sponsor Opus Genetics, Inc
Funder Type Industry
Recruitment Status 🔄
Enrollment Count 10 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 RDH12
Therapeutic Modality 🔄 Gene transfer
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Functional gene replacement
Route of Administration 🔄 Subretinal
Drug Product Type 🔄 Viral vector
Target Tissue/Cell 🔄 Photoreceptors
Gene Delivery System Type 🔄 Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV8
Dose 1 🔄 Low dose: 1E11 vg/eye
Dose 2 🔄 High dose: 3E11 vg/eye

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2026-06-24
Completion Date 2034-07
Last Update 🔄 2026-10-30

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Developed with support from the RDH12 Alliance, FDA cleared IND application in October 2026

Resources/Links