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Tests treatment safety and results for LCA5
Official title Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
ClinicalTrials.gov ID: NCT05616793
What this study is testing
What is AAV8.hLCA5?
AAV8.hLCA5 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for lca5.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 2: a mid-size study of how well it works
- Time commitment: about 6 months
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures.
- Are willing to adhere to the clinical protocol and able to perform testing procedures.
- 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...
- Carry disease-causing biallelic LCA5 gene mutations determined by a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (historic...
- Visual acuity: BCVA \< 20/80 on the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity chart (modified for low vision participants)...
You likely can't join if
- Women who are pregnant or individuals (women of childbearing potential and men) unwilling to use effective contraception for the duration of the...
- Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery. This includes individuals who are...
- History of intraocular surgery for either eye within 6 months prior to planned IP administration (Part A only).
- Have previously received gene therapy.
- 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...
- History of disease which may preclude the participant from participation, or which may interfere with outcome measure testing or test results.
See the full eligibility criteria
- Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures.
- Are willing to adhere to the clinical protocol and able to perform testing procedures.
- 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.
- 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).
- 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
- Show evidence of detectable photoreceptors by Spectral Domain Optical Coherence Tomography (SD-OCT)
- Participant is a good candidate for surgery per investigator judgement
- Participant agrees to follow direction of investigator regarding restrictions post-surgery (Part A only).
- 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...
- Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery. This includes individuals who are immunocompromised.
- History of intraocular surgery for either eye within 6 months prior to planned IP administration (Part A only).
- Have previously received gene therapy.
- 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.
- History of disease which may preclude the participant from participation, or which may interfere with outcome measure testing or test results.
- Incapable of performing visual function testing (e.g., FST testing) for reasons other than poor vision.
- Any absolute contraindication to a course of oral steroids.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Philadelphia, Pennsylvania, United States
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 6 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Philadelphia, Pennsylvania, United States; Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.