What side effects are associated with this type of intervention?

Common treatments for Fundus Flavimaculatus (Stargardt disease) include visual cycle modulators, anti-VEGF injections, and oral supplements. Known side effects of these treatments can include ocular discomfort, increased intraocular pressure, and systemic effects such as hypertension. Given the investigational nature of OCU410ST, it is likely that the side effects would be similar to those observed in other treatments for macular degeneration.

What prior FDA approvals exist?

Fundus Flavimaculatus, a variant of Stargardt Disease, currently has limited FDA-approved treatments. The investigational drug OCU410ST is being studied for its efficacy in treating Stargardt Disease. Historically, treatments for similar retinal conditions have included therapies like verteporfin for choroidal neovascularization in age-related macular degeneration and ranibizumab for macular edema due to retinal vein occlusion. These treatments focus on managing symptoms and slowing disease progression rather than curing the underlying genetic cause.

What other types of research exist?

Promising alternatives to OCU410ST for Stargardt Disease include fenretinide, a visual cycle inhibitor, and research into lutein and zeaxanthin supplements. Additionally, there is interest in the potential use of levodopa, a drug used for Parkinson's disease, which may reduce the incidence of age-related macular degeneration and could be explored for Stargardt Disease.

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Gene Therapy

OCU410ST for Stargardt Disease (GARDian Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Ocugen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months (screening to 12 months post ocu410st administration)

Awards & highlights

No Placebo-Only Group

Summary

This trial tests OCU410ST, a treatment using a harmless virus to deliver a helpful gene into the eye, for children and young adults with Stargardt disease. The gene helps manage fat buildup, stress, and inflammation in the eye.

Who is the study for?

This clinical trial is for individuals aged 18-65 with Stargardt Disease, specifically those who have a vision acuity of 50 letters or less on the ETDRS chart. Participants must have confirmed genetic mutations in ABCA4 and evidence of macular lesions consistent with the disease. They should also have some remaining retinal layer detectable by OCT imaging.

What is being tested?

The study tests OCU410ST's safety and effectiveness for treating Stargardt Disease. It's a two-phase trial involving up to 42 participants across multiple centers, aiming to see how well this treatment works and if it's safe.

What are the potential side effects?

Since this is an early phase trial (Phase 1/2), specific side effects are not yet fully known but will be closely monitored throughout the study to ensure patient safety.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months (screening to 12 months post ocu410st administration)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months (screening to 12 months post ocu410st administration)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months (screening to 12 months post ocu410st administration) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change Using Qualitative and quantitative assessments of autofluorescence pattern (FAF)

Ophthalmic Safety: Change From Baseline in BCVA (Best Corrected Visual Acuity)

Ophthalmic Safety: Change in the Intraocular Pressure (mmHg)

+3 more

Secondary study objectives

Change in laboratory parameters for Hematology

Change in laboratory parameters for Serum Chemistry

Humoral and cellular immune response

+1 more

Other study objectives

Change in Quality-of-life measure using NEI VFQ-25 (Adult subjects only)

Changes in macular thickness on Spectra Domain Optical Coherence Tomography (SD-OCT)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

9Treatment groups

Experimental Treatment

Active Control

Group I: Experimental: Phase1 Dose Escalation- Medium Dose (7.5×10E10 vg/mL):Experimental Treatment1 Intervention

Medium Dose (7.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410ST in the Medium dose concentration.

Group II: Experimental: Phase1 Dose Escalation- Low Dose (3.75×10E10 vg/mL):Experimental Treatment1 Intervention

Low Dose (3.75×10E10 vg/mL): Subjects will receive a subretinal injection of 200 µL of OCU410ST in the low dose concentration.

Group III: Experimental: Phase1 Dose Escalation- High Dose (2.25×10E11 vg/mL):Experimental Treatment1 Intervention

High Dose (2.25×10E11 vg/mL): Subjects will receive a subretinal injection of OCU410ST in the high dose concentration.

Group IV: Experimental: Phase 2 Dose Expansion: Dose 2 from Phase 1-Randomized Pediatric ArmExperimental Treatment1 Intervention

Subjects will receive a subretinal injection of OCU410ST with Lower Dose than Maximum tolerated dose (MTD) from Phase 1

Group V: Experimental: Phase 2 Dose Expansion: Dose 2 from Phase 1-Randomized Adult ArmExperimental Treatment1 Intervention

Subjects will receive a subretinal injection of OCU410ST with Lower Dose than Maximum tolerated dose (MTD) from Phase 1

Group VI: Experimental: Phase 2 Dose Expansion: Dose 1 from Phase 1-Randomized Pediatric ArmExperimental Treatment1 Intervention

Subjects will receive a subretinal injection of OCU410ST with Maximum tolerated dose (MTD) from Phase 1.

Group VII: Experimental: Phase 2 Dose Expansion: Dose 1 from Phase 1-Randomized Adult ArmExperimental Treatment1 Intervention

Subjects will receive a subretinal injection of OCU410ST with Maximum tolerated dose (MTD) from Phase 1.

Group VIII: No Intervention- Randomized Control Adult ArmActive Control1 Intervention

No Intervention Control Arm: Subject will not receive any active study intervention

Group IX: No Intervention- Randomized Control Pediatric ArmActive Control1 Intervention

No Intervention Control Arm: Subject will not receive any active study intervention

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Fundus Flavimaculatus, or Stargardt Disease, is often treated with investigational drugs like OCU410ST, which aim to slow or halt the progression of retinal degeneration. These treatments typically work by targeting the underlying genetic mutations that cause the accumulation of toxic byproducts in the retinal pigment epithelium (RPE). By reducing the buildup of these toxic substances, these therapies help preserve photoreceptor function and prevent further vision loss. This is crucial for patients as it can maintain their visual acuity and quality of life for a longer period.

Optic nerve head reactive retinal astrocytic tumor treated with photodynamic therapy.Intravitreal bevacizumab injection alone or combined with macular photocoagulation compared to macular photocoagulation as primary treatment of diabetic macular edema.Phase ii, randomized, placebo-controlled, 90-day study of emixustat hydrochloride in geographic atrophy associated with dry age-related macular degeneration.