What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF injections, laser therapy, and photodynamic therapy. Anti-VEGF injections, such as ranibizumab and aflibercept, can cause side effects like eye pain, increased intraocular pressure, and inflammation. Laser therapy may lead to scarring and vision loss in treated areas. Photodynamic therapy can cause temporary vision changes and photosensitivity. Treatments similar to RPESC-RPE-4W, which involve retinal pigment epithelial cell transplantation, have shown medium- to long-term safety and tolerability in initial studies, with potential side effects including immune rejection and the need for immunosuppressive therapy. Regular monitoring by an eye care professional is essential to manage these risks.

What prior FDA approvals exist?

FDA-approved treatments for Age-Related Macular Degeneration (AMD) primarily include anti-VEGF (vascular endothelial growth factor) therapies such as ranibizumab (Lucentis), aflibercept (Eylea), and bevacizumab (Avastin). These treatments work by inhibiting abnormal blood vessel growth and leakage in the retina. While these are the mainstay treatments, the RPESC-RPE-4W trial is exploring the use of stem cell-derived Retinal Pigment Epithelial (RPE) cells, which represents a novel approach aimed at replacing damaged RPE cells to restore retinal function. This type of cell therapy is still under investigation and has not yet received FDA approval.

What other types of research exist?

Promising novel alternatives to RPESC-RPE-4W for AMD patients include: 1) Gene therapy approaches such as RGX-314, which delivers a gene encoding for a therapeutic protein to reduce the need for frequent anti-VEGF injections. 2) Other stem cell therapies like human embryonic stem cell-derived RPE cells, which aim to replace damaged retinal cells. 3) Pharmacological advancements such as faricimab, a bispecific antibody targeting both VEGF and Ang-2, offering potential for improved efficacy and reduced treatment burden.

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Stem Cell Therapy

Stem Cell Therapy for Dry Age-Related Macular Degeneration

Phase 1 & 2

Recruiting

Led By Rajesh C Rao, M.D.

Research Sponsored by Luxa Biotechnology, LLC

Eligibility Criteria Checklist

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

Must have

Ability to understand and give informed consent

Medically suitable to undergo vitrectomy and subretinal injection (>60% on Karnofsky scale)

Must not have

Active major medical conditions limiting ability to participate in the study

Systemic immunosuppressant therapy within past six months

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment where cells from donor eyes are transplanted into patients with dry AMD to see if it is safe and can help improve their vision. Stem cell transplantation may improve visual acuity in patients with dry age-related macular degeneration.

Who is the study for?

This trial is for adults over 55 with dry AMD who can consent and are healthy enough for eye surgery. Men must use contraception, women should be postmenopausal or unable to have children. Excluded are those with major health issues, recent immunosuppressants or clinical trials, certain infections, pregnancy, nursing, or specific allergies.

What is being tested?

The study tests the safety and tolerability of a stem cell-derived treatment called RPESC-RPE-4W in patients with dry age-related macular degeneration (AMD). It involves vitrectomy and subretinal injection procedures to deliver the therapy.

What are the potential side effects?

While not explicitly listed here, potential side effects may include typical risks associated with vitrectomy and subretinal injections such as eye inflammation, infection risk increase, vision changes, bleeding inside the eye area.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I understand the study and agree to participate.

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I am medically fit for eye surgery (>60% Karnofsky).

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I have been diagnosed with dry age-related macular degeneration.

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I am over 55 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have major health issues that would prevent me from joining the study.

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I have not taken immunosuppressant drugs in the last 6 months.

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I am currently receiving chemotherapy for cancer.

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I have had toxoplasmosis, retinal histoplasmosis, or tuberculosis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Safety and tolerability of RPESC-RPE-4W transplantation

Secondary study objectives

Change in GA lesion area

Change in the mean of Best Corrected Visual Acuity (BCVA)

Evidence of structural changes

+1 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: 50,000 cellsExperimental Treatment1 Intervention

Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.

Group II: 250,000 cellsExperimental Treatment1 Intervention

Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.

Group III: 150,000 cellsExperimental Treatment1 Intervention

Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.

0 / 8Eligibility criteria met

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

The most common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF (vascular endothelial growth factor) therapy, photodynamic therapy, and emerging treatments like Retinal Pigment Epithelial (RPE) cell transplantation. Anti-VEGF therapy works by inhibiting abnormal blood vessel growth and leakage in the retina, which helps to prevent vision loss. Photodynamic therapy involves using a light-activated drug to destroy abnormal blood vessels. RPE cell transplantation, such as the RPESC-RPE-4W being studied, aims to replace damaged RPE cells, which are crucial for supporting the photoreceptors in the retina. This is particularly important for AMD patients as it addresses the underlying cellular damage, potentially restoring retinal function and slowing disease progression.