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

NCT ID: NCT04627428

Last Updated: 2025-10-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-04-05

Study Completion Date

2026-12-01

Brief Summary

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

Detailed Description

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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

Conditions

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Dry Age-related Macular Degeneration

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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50,000 cells

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

Group Type EXPERIMENTAL

RPESC-RPE-4W

Intervention Type BIOLOGICAL

RPESC-RPE-4W

150,000 cells

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

Group Type EXPERIMENTAL

RPESC-RPE-4W

Intervention Type BIOLOGICAL

RPESC-RPE-4W

250,000 cells

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

Group Type EXPERIMENTAL

RPESC-RPE-4W

Intervention Type BIOLOGICAL

RPESC-RPE-4W

Interventions

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RPESC-RPE-4W

RPESC-RPE-4W

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Minimum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

National Eye Institute (NEI)

NIH

Sponsor Role collaborator

Regenerative Research Foundation

UNKNOWN

Sponsor Role collaborator

California Institute for Regenerative Medicine (CIRM)

OTHER

Sponsor Role collaborator

Luxa Biotechnology, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Rajesh C Rao, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Michigan Kellogg Eye Center

Theodore Leng, MD, FACS

Role: PRINCIPAL_INVESTIGATOR

Spencer Center for Vision Research at Stanford University

David S Liao, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Retina-Vitreous Associates Medical Group

Locations

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Retina Vitreous Associates Medical Group

Beverly Hills, California, United States

Site Status RECRUITING

Spencer Center for Vision Research

Palo Alto, California, United States

Site Status RECRUITING

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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

Role: CONTACT

05184371111

Facility Contacts

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David S Liao, MD, PhD

Role: primary

Theodore Leng, MD, FACS

Role: primary

Rajesh C Rao, M.D.

Role: primary

Other Identifiers

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U01EY030581

Identifier Type: NIH

Identifier Source: secondary_id

View Link

UG3EY031810

Identifier Type: NIH

Identifier Source: secondary_id

View Link

RPESC-RPE-01

Identifier Type: -

Identifier Source: org_study_id

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