Transplanting stem cells to treat dry age-related macular degeneration

A Phase1/2a, Open-Label Study to Evaluate the Safety and Tolerability of RPE Stem Cell-derived RPE (RPESC-RPE) Transplantation as Therapy for Dry Age-related Macular Degeneration (AMD)

This study is testing a new stem cell treatment for people with dry age-related macular degeneration to see if it is safe and well-tolerated.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: 55 Years and up
• Sex: All
• Sponsor: Luxa Biotechnology, LLC (Industry-sponsored)
• Drugs / interventions: chemotherapy
• Locations: 3 sites (Beverly Hills, California and 2 other locations)
• Trial ID: NCT04627428 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and tolerability of RPESC-RPE-4W, a therapy derived from allogeneic RPE stem cells, for patients suffering from dry age-related macular degeneration (AMD). The study is a Phase 1/2a open-label dose-escalation trial that aims to enroll 18 subjects who will receive subretinal injections of the stem cell-derived RPE cells. Participants will be monitored for any adverse effects and overall tolerability of the treatment. The goal is to assess whether this innovative approach can provide a new therapeutic option for individuals with this condition.

Who should consider this trial

Good fit: Ideal candidates are adults over 55 years old with a clinical diagnosis of dry AMD who are medically suitable for vitrectomy.

Not a fit: Patients with active major medical conditions, malignancies, or those on systemic immunosuppressants may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a novel therapy that may improve vision or slow the progression of dry AMD.

How similar studies have performed: While this approach is innovative, similar studies involving stem cell therapies for retinal conditions have shown promise, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

1. Clinical diagnosis of dry AMD.
2. Ability to understand and give informed consent.
3. Adult male or female \>55 years of age.
4. Medically suitable to undergo vitrectomy and subretinal injection (\>60% on Karnofsky scale).
5. Postmenopausal if female (expected to be common for the age limitation), or the female partner of a male subject unable to father children.
6. If male, willing to use barrier and spermicidal contraception during the study.

Exclusion Criteria:

1. Allergy or hypersensitivity to dilation drops or fluorescein.
2. Active major medical conditions limiting ability to participate in the study.
3. Active malignancy or treatment with chemotherapy.
4. Systemic immunosuppressant therapy within past six months.
5. History of toxoplasmosis, retinal histoplasmosis or tuberculosis.
6. Receipt of investigational product (IP) in a clinical trial within prior six months.
7. Any other medical condition, which, in the Investigator's judgment, will interfere with the subject's ability to comply with the protocol, compromises subject safety, or interferes with the interpretation of the study results.
8. Pregnant or nursing females.

Where this trial is running

Beverly Hills, California and 2 other locations

• Retina Vitreous Associates Medical Group — Beverly Hills, California, United States (Recruiting)
• Spencer Center for Vision Research — Palo Alto, California, United States (Recruiting)
• University of Michigan Kellogg Eye Center — Ann Arbor, Michigan, United States (Recruiting)

Study contacts

• Principal investigator: Rajesh C Rao, M.D. — University of Michigan Kellogg Eye Center
• Study coordinator: Jeffrey H Stern, M.D., Ph.D.

Conditions Dry Age-related Macular Degeneration