RetinoStat® Safety for Age-Related Macular Degeneration

Not currently recruiting at 2 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Oxford BioMedica

Disqualifiers: Did not receive RetinoStat, 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 evaluates the long-term safety of RetinoStat®, a new treatment for neovascular age-related macular degeneration, a condition that affects the eyes and can cause vision loss. The trial involves individuals who previously received RetinoStat® in an earlier study, providing an opportunity to help researchers assess the treatment's safety over time. Only those who participated in that specific earlier study can join. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants a unique opportunity to contribute to groundbreaking research.

Do I need to stop my current medications for the RetinoStat® trial?

The trial information does not specify whether you need to stop taking your current medications.

Is there any evidence suggesting that RetinoStat is likely to be safe for humans?

Research has shown that RetinoStat® has been tested for safety in treating age-related macular degeneration. In earlier studies, patients handled RetinoStat well. The findings indicate that the LentiVector® platform, a component of RetinoStat, delivers the treatment safely and effectively.

Reports from these studies showed that serious side effects were rare, suggesting that RetinoStat is generally safe for patients. This trial aims to ensure it remains safe over the long term. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike standard treatments for age-related macular degeneration (AMD) that typically involve repeated injections of anti-VEGF drugs into the eye, RetinoStat® offers a unique approach. This investigational treatment uses a gene therapy vector to deliver a protein directly to the retina, potentially offering a long-lasting solution with just a single administration. Researchers are excited about RetinoStat® because it targets the underlying causes of AMD at the genetic level, which could lead to more sustained improvements in vision and reduce the burden of frequent treatments.

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

Research has shown that RetinoStat® is a promising new treatment for neovascular age-related macular degeneration (AMD). In earlier studies, patients who received this treatment found it safe and well-tolerated, marking important initial steps. RetinoStat® delivers a gene directly to the eye to halt the growth of abnormal blood vessels that cause vision loss in AMD. Early results suggest this method could lead to better long-term outcomes compared to traditional treatments. Although more research is needed, its mechanism offers hope for potential effectiveness in treating AMD. ^{1 6 7 8 9}

Who Is on the Research Team?

Peter Campochiaro, MD

Principal Investigator

John Hopkins University Hospital

Are You a Good Fit for This Trial?

This trial is specifically for patients who were previously enrolled in the RS1/001/10 study and received a subretinal injection of RetinoStat®. It's designed to follow up on those individuals to assess long-term safety related to their treatment for age-related macular degeneration.

Inclusion Criteria

I have received a RetinoStat injection in my eye.

Must have been enrolled in Protocol RS1/001/10

Exclusion Criteria

Did not receive RetinoStat as part of the RS1/001/10 protocol

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants received RetinoStat® in a previous study

Long Term Follow-up

Participants are monitored for long-term safety and changes in visual acuity

14 years

What Are the Treatments Tested in This Trial?

Interventions

• RetinoStat

Trial Overview The focus of this study is on the long-term safety profile of RetinoStat®, an experimental gene therapy aimed at treating neovascular age-related macular degeneration, which was administered in a prior clinical trial.

How Is the Trial Designed?

Group I: Long Term Follow up Experimental Treatment 1 Intervention

Long Term follow up of patients who received RetinoStat in a previous study.

Who Is Running the Clinical Trial?

Oxford BioMedica

Lead Sponsor

Trials

13

Recruited

1,100+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12271004/

Real-World 5-Year Outcomes of Age-Related Macular ... Patients treated in Dutch FRB! clinics have good long-term outcomes with a 2.3-letter higher mean VA at the 60-month timepoint compared to FRB! clinics in the ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT01301443

Phase I Dose Escalation Safety Study of RetinoStat in ... The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, RetinoStat, designed to treat neovascular age-related ...

3. biosignaling.biomedcentral.com biosignaling.biomedcentral.com/articles/10.1186/s12964-025-02246-4

Gene therapy for age-related macular degeneration Clinical observations reveal a gradual decline in visual acuity among wet AMD patients undergoing anti-angiogenic therapy. This deterioration is ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2352304221000295

Age-related macular degeneration: Epidemiology, genetics ... Age-related macular degeneration (AMD) is a complex eye disorder and is the leading cause of incurable blindness worldwide in the elderly.

5. aaojournal.org aaojournal.org/article/s0161-6420(16)30092-6/fulltext

Five-Year Outcomes with Anti–Vascular Endothelial ... To describe outcomes 5 years after initiating treatment with bevacizumab or ranibizumab for neovascular age-related macular degeneration (AMD).

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12090701/

Gene therapy for age-related macular degeneration Age-related macular degeneration (AMD) is a major cause of vision loss, primarily affecting the central region of the retina and leading to ...

7. iovs.arvojournals.org iovs.arvojournals.org/article.aspx?articleid=2332049

Results from the Phase I GEM study, evaluating safety ... - IOVS ... (RetinoStat®) for the treatment of wet age-related macular degeneration (AMD) ... data demonstrates that the LentiVector® platform safely and efficiently ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT01678872

A Follow-up Study to Evaluate the Safety of RetinoStat® in ... ... Safety of RetinoStat® in Patients With Age-Related Macular Degeneration. Conditions. Age-Related Macular Degeneration. Age-Related Macular Degeneration. Age ...

9. withpower.com withpower.com/trial/phase-1-macular-degeneration-7-2012-48a68

RetinoStat® Safety for Age-Related Macular Degeneration It's designed to follow up on those individuals to assess long-term safety related to their treatment for age-related macular degeneration. Inclusion Criteria.