Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)

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)

The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.

Study Overview

• Status: Recruiting
• Conditions: Dry Age-related Macular Degeneration
• Intervention / Treatment: Biological: RPESC-RPE-4W

Detailed Description

RPESC-RPE-4W is Allogeneic RPE stem cell (RPESC)-derived RPE cells (RPESC-RPE) isolated from the RPE layer of human cadaveric eyes are transplanted under the macular.

This first-in-human Phase 1/2a open-label dose-escalation interventional study plans to enroll a total of 18 subjects

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Jeffrey H Stern, M.D., Ph.D.; Phone Number: 05184371111; Email: jeffreystern@luxabiotech.com

Study Locations

• United States
  • California
    • Beverly Hills, California, United States, 90211
      • Recruiting
      • Retina Vitreous Associates Medical Group
      • Contact: David S Liao, MD, PhD
    • Palo Alto, California, United States, 94303
      • Recruiting
      • Spencer Center for Vision Research
      • Contact: Theodore Leng, MD, FACS
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Recruiting
      • University of Michigan Kellogg Eye Center
      • Contact: Rajesh C Rao, M.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 55 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 50,000 cells; Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.	Biological: RPESC-RPE-4W; RPESC-RPE-4W
Experimental: 150,000 cells; Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.	Biological: RPESC-RPE-4W; RPESC-RPE-4W
Experimental: 250,000 cells; Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.	Biological: RPESC-RPE-4W; RPESC-RPE-4W

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability of RPESC-RPE-4W transplantation	The transplantation of RPESC-RPE-4W cells will be considered safe and tolerated in the absence of: Decrease in visual acuity (VA) of more than 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (or to worse than counting fingers at three feet) from baseline; Any Grade 2 (CTCAE v5.0) or greater non-ocular Adverse Events (AE) related to the RPESC-RPE-4W investigational product (IP) or investigational interventions; Intraocular inflammation indicating contamination with an infectious agent or serious immune response greater than moderate severity related to the RPESC-RPE-4W investigational product or investigational interventions; Tumor formation > 1 mm size related to the IP or investigational interventions	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in the mean of Best Corrected Visual Acuity (BCVA)	Change in visual acuity will be measured by ETDRS chart.	24 months
Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry	Loss of ≥10 decibels of ten-degree average visual sensitivity will by measured by microperimetry.	24 months
Change in GA lesion area	Change in GA lesion area will be measured.	24 months
Evidence of structural changes	Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography	24 months

Collaborators and Investigators

• Sponsor: Luxa Biotechnology, LLC
• Collaborators: National Eye Institute (NEI); National Institutes of Health (NIH); California Institute for Regenerative Medicine (CIRM); Regenerative Research Foundation
• Investigators: Principal Investigator: Rajesh C Rao, M.D., University of Michigan Kellogg Eye Center; Principal Investigator: Theodore Leng, MD, FACS, Spencer Center for Vision Research at Stanford University; Principal Investigator: David S Liao, MD, PhD, Retina-Vitreous Associates Medical Group

Study record dates

Study Major Dates

• Study Start (Actual): April 5, 2022
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026

Study Registration Dates

• First Submitted: November 1, 2020
• First Submitted That Met QC Criteria: November 8, 2020
• First Posted (Actual): November 13, 2020

Study Record Updates

• Last Update Posted (Estimated): October 15, 2025
• Last Update Submitted That Met QC Criteria: October 12, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration
• Other Study ID Numbers: RPESC-RPE-01; U01EY030581 (U.S. NIH Grant/Contract); UG3EY031810 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No