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

Overview

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

Full Title of Study: “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)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: May 31, 2025

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

Interventions

  • Biological: RPESC-RPE-4W
    • RPESC-RPE-4W

Arms, Groups and Cohorts

  • Experimental: 50,000 cells
    • Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.
  • Experimental: 150,000 cells
    • Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.
  • Experimental: 250,000 cells
    • Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.

Clinical Trial Outcome Measures

Primary Measures

  • Safety and tolerability of RPESC-RPE-4W transplantation
    • Time Frame: 24 months
    • 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 version 5) or greater Adverse Events (AE) related to the cell product and investigational interventions. Any evidence that the cell are contaminated with an infectious agent or serious immune response to the cell product Any evidence that the cells show tumorigenic potential

Secondary Measures

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

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 55 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Luxa Biotechnology, LLC
  • Collaborator
    • National Institutes of Health (NIH)
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Rajesh C Rao, M.D., Principal Investigator, University of Michigan Kellogg Eye Center
  • Overall Contact(s)
    • Jeffrey H Stern, M.D., Ph.D., 05184371111, jeffreystern@luxabiotech.com

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