NCT05616793 Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
| NCT ID | NCT05616793 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Opus Genetics, Inc |
| Condition | LCA5 |
| Study Type | INTERVENTIONAL |
| Enrollment | 22 participants |
| Start Date | 2023-06-15 |
| Primary Completion | 2028-06-15 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 22 participants in total. It began in 2023-06-15 with a primary completion date of 2028-06-15.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and 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. Funding Source- FDA Office of Orphan Products Development (OOPD).
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05616793 clinical trial?
This trial is open to participants of all sexes, aged 4 Years or older, studying LCA5. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05616793 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05616793 currently recruiting?
Yes, NCT05616793 is actively recruiting participants. Contact the research team at ct.gov_inquiries@opusgtx.com for enrollment information.
Where is the NCT05616793 trial being conducted?
This trial is being conducted at Philadelphia, United States, Dallas, United States.
Who is sponsoring the NCT05616793 clinical trial?
NCT05616793 is sponsored by Opus Genetics, Inc. The trial plans to enroll 22 participants.