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NCT Number: NCT04627428

Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With 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.

Recruiting

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Key information

Conditions

Dry Age-related Macular Degeneration

Age range

55 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Retina Vitreous Associates Medical Group, Beverly Hills, California, United States

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • Allergy or hypersensitivity to dilation drops or fluorescein.
  • Active major medical conditions limiting ability to participate in the study.
  • Active malignancy or treatment with chemotherapy.
  • Systemic immunosuppressant therapy within past six months.
  • History of toxoplasmosis, retinal histoplasmosis or tuberculosis.
  • Receipt of investigational product (IP) in a clinical trial within prior six months.
  • 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.
  • Pregnant or nursing females.

Treatment and study plan

RPESC-RPE-4W

Biological

RPESC-RPE-4W

Primary outcomes

  1. 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 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

Secondary outcomes

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

    Time frame: 24 months

    Change in visual acuity will be measured by ETDRS chart.

  2. 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.

  3. Change in GA lesion area

    Time frame: 24 months

    Change in GA lesion area will be measured.

  4. Evidence of structural changes

    Time frame: 24 months

    Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography

Study contacts

Contact information is provided by the study sponsor or research team.

Jeffrey H Stern, M.D., Ph.D.

CONTACT

[email protected]

05184371111

Sponsors and collaborators

Lead sponsor

Luxa Biotechnology, LLC

Industry

Collaborators

  • California Institute for Regenerative Medicine (CIRM)
  • National Eye Institute (NEI)
  • National Institutes of Health (NIH)
  • Regenerative Research Foundation

Registry information

Official study title

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)

Important dates

Study start
2022
Primary completion
2026
Study completion
2026
First posted
Nov 13, 2020
Registry last updated
Oct 15, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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