NCT04627428

Brief Summary

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

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for phase_1

Timeline
6mo left

Started Apr 2022

Longer than P75 for phase_1

Geographic Reach
1 country

3 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress90%
Apr 2022Dec 2026

First Submitted

Initial submission to the registry

November 1, 2020

Completed
12 days until next milestone

First Posted

Study publicly available on registry

November 13, 2020

Completed
1.4 years until next milestone

Study Start

First participant enrolled

April 5, 2022

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2026

Last Updated

October 15, 2025

Status Verified

October 1, 2025

Enrollment Period

4.7 years

First QC Date

November 1, 2020

Last Update Submit

October 12, 2025

Conditions

Dry Age-related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (4)

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

    24 months

  • Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry

    24 months

  • Change in GA lesion area

    24 months

  • Evidence of structural changes

    24 months

Study Arms (3)

50,000 cells

EXPERIMENTAL

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

Biological: RPESC-RPE-4W

150,000 cells

EXPERIMENTAL

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

Biological: RPESC-RPE-4W

250,000 cells

EXPERIMENTAL

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

Biological: RPESC-RPE-4W

Interventions

RPESC-RPE-4WBIOLOGICAL

RPESC-RPE-4W

150,000 cells250,000 cells50,000 cells

Eligibility Criteria

Age55 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Retina Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

RECRUITING

Spencer Center for Vision Research

Palo Alto, California, 94303, United States

RECRUITING

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

RECRUITING

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Central Study Contacts

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

CONTACT

05184371111jeffreystern@luxabiotech.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 1, 2020

First Posted

November 13, 2020

Study Start

April 5, 2022

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

December 1, 2026

Last Updated

October 15, 2025

Record last verified: 2025-10

Data Sharing

IPD Sharing
Will not share

Locations

US(3)