OCU410 for Age-Related Macular Degeneration

(ArMaDa Trial)

Not currently recruiting at 14 trial locations

Overseen By Michael J Borne, M.D

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ocugen

Disqualifiers: Gene therapy, Cell therapy, Wet AMD, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called OCU410 for individuals with geographic atrophy, a condition related to dry age-related macular degeneration (AMD) that leads to vision loss. Researchers aim to determine the safety and effectiveness of OCU410 by evaluating different doses. Participants will receive either a low, medium, or high dose of OCU410, or no treatment at all for comparison. The study seeks individuals aged 50 or older who have been diagnosed with dry AMD and experience vision problems due to geographic atrophy. As a Phase 1, Phase 2 trial, this research focuses on understanding how OCU410 works in people and measuring its effectiveness in an initial group, offering participants a chance to contribute to groundbreaking advancements in AMD treatment.

Will I have to stop taking my current medications?

The trial requires a washout period (time without taking certain medications) of at least 3 months for those who had prior treatment with approved drugs for AMD, like Izerway® or Syfovre®. For other medications, the protocol does not specify if you need to stop taking them.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that OCU410 is very safe. Studies have found no serious side effects linked to the treatment. In past trials, participants did not experience any major health problems caused by OCU410. The treatment is easy for patients to handle, which is encouraging for anyone considering joining a clinical trial. This safety information should provide comfort to prospective participants. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Unlike the standard treatments for age-related macular degeneration, which often involve regular injections into the eye to inhibit blood vessel growth, OCU410 is unique because it is delivered via a one-time subretinal injection. This gene therapy approach aims to address the root cause of the degeneration by targeting specific genes involved in the disease process. Researchers are excited about OCU410 because it has the potential to offer a more lasting solution, reducing the need for frequent treatments and possibly providing better long-term outcomes for patients.

What evidence suggests that OCU410 might be an effective treatment for age-related macular degeneration?

Research has shown that OCU410 may help treat geographic atrophy, a condition related to dry age-related macular degeneration (AMD). In earlier studies, patients who used OCU410 preserved the eye tissue around damaged areas better after six months. This suggests that OCU410 might slow vision loss in people with AMD. In this trial, participants will receive different doses of OCU410—low, medium, and high—to evaluate its effectiveness and safety. Trials so far have found OCU410 to be safe, with no serious side effects reported. These findings offer hope that OCU410 could effectively help preserve vision in AMD patients. ^{2 3 6 7 8}

Who Is on the Research Team?

Murthy Chavali, Ph.D

Principal Investigator

Ocugen

Are You a Good Fit for This Trial?

This trial is for people aged 50 or older with a specific eye condition called Geographic Atrophy due to Dry Age-Related Macular Degeneration. Participants must have certain levels of vision and specific characteristics in their eye imaging. Those who've had recent investigational treatments, gene or cell therapies, or treatment with Syfovre are excluded.

Inclusion Criteria

Your total GA area is between 2.5 and 17.5 mm2, equivalent to 1-7 disk areas (DA).

I am 50 years old or older.

Your visual acuity is at least 24 letters or higher according to the ETDRS chart (equivalent to 20/320 on a Snellen scale).

Exclusion Criteria

I haven't used experimental drugs or devices in the last year.

My vision loss is not due to AMD but another condition like Stargardt disease.

I have been treated with Syfovre before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1 Treatment

Open-label, dose-ranging/dose-escalating study with a 3+3 design enrolling up to 18 subjects. Participants receive a single subretinal injection of OCU410.

12 months

Multiple visits for dose escalation and monitoring

Phase 2 Treatment

Randomized dose-expansion cohort with 45 subjects in a 1:1:1 ratio to two treatment arms or control. Participants receive a single subretinal injection of OCU410.

12 months

Multiple visits for treatment and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of visual acuity and immune response.

12 months

What Are the Treatments Tested in This Trial?

Interventions

• OCU410

Trial Overview The study tests the safety and effectiveness of OCU410 in treating Geographic Atrophy. It's a two-phase study involving up to 63 subjects across multiple centers, aiming to see how well this new intervention works compared to current standards.

How Is the Trial Designed?

Group I: Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL): Experimental Treatment 1 Intervention

Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.

Group II: Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL): Experimental Treatment 1 Intervention

Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.

Group III: Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL): Experimental Treatment 1 Intervention

High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.

Group IV: Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm Experimental Treatment 1 Intervention

Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.

Group V: Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm Experimental Treatment 1 Intervention

Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.

Group VI: Control Arm Active Control 1 Intervention

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

Who Is Running the Clinical Trial?

Ocugen

Lead Sponsor

Trials

12

Recruited

1,100+

Citations

1. ir.ocugen.com ir.ocugen.com/news-releases/news-release-details/ocugen-announces-compelling-preliminary-data-ocu410-single-dose

Ocugen Announces Compelling Preliminary Data for OCU410 ... OCU410 treatment showed increasing preservation of retinal tissue around the GA lesions of treated eyes over six months, which also compared ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06018558

Study to Assess the Safety and Efficacy of OCU410 for ... This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD).

3. ir.ocugen.com ir.ocugen.com/news-releases/news-release-details/ocugen-inc-announces-dosing-completion-phase-2-armada-clinical

Press Release - Investors - Ocugen The ArMaDa Phase 1/2 clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two ...

4. iovs.arvojournals.org iovs.arvojournals.org/article.aspx?articleid=2807599

Phase 1/2 OCU410: The Age-related Macular Degeneration ... Conclusions : OCU410 has shown to be safe in all nine treated patients. Preliminary efficacy analysis of three patients completing 6 month follow up shows that ...

5. ophthalmologytimes.com ophthalmologytimes.com/view/asrs-2025-phase-1-2-study-update-on-ocu-410-for-geographic-atrophy

ASRS 2025: Phase 1/2 study update on OCU-410 for ... The study's safety profile was particularly noteworthy, with no serious adverse events reported during the 12-month follow-up period.

6. ir.ocugen.com ir.ocugen.com/news-releases/news-release-details/data-and-safety-monitoring-board-reviews-interim-safety-data

Press Release - Investors - Ocugen OCU410 has a very favorable safety and tolerability profile; No serious adverse events related to the study drug have been reported, ...

7. packgene.com packgene.com/frontier/250212-2/

Ocugen, Inc. Announces Dosing Completion in the Phase ... Announces Dosing Completion in the Phase 2 ArMaDa Clinical Trial for OCU410—a Multifunctional Modifier Gene Therapy for the Treatment of ...

8. ctv.veeva.com ctv.veeva.com/study/study-to-assess-the-safety-and-efficacy-of-ocu410-for-geographic-atrophy

Study to Assess the Safety and Efficacy of OCU410 for ... A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration.