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Gene Therapy Trial Report

Summary

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients


NCTID NCT05616793 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Leber Congenital Amaurosis-Type 5
Disease Ontology Term DOID:0110215
Compound Name OPGx-LCA5
Compound Description AAV8.hLCA5
Sponsor Opus Genetics, Inc
Funder Type Industry
Recruitment Status
Enrollment Count 22 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant LCA5
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 1E10 vg/eye (up to 300ul)
Dose 2 3E10 vg/eye
Dose 3 1E11 vg/eye

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-11-07
Completion Date 2028-06-15
Last Update 2026-02-19

Participation Criteria


Eligible Age >=4 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates First participant enrolled in run-in period for planned adaptive Phase 3 trial

Resources/Links