Safety and Tolerability of RPESC-derived RPE Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
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|ClinicalTrials.gov Identifier: NCT04627428|
Recruitment Status : Recruiting
First Posted : November 13, 2020
Last Update Posted : July 26, 2022
|Condition or disease||Intervention/treatment||Phase|
|Dry Age-related Macular Degeneration||Biological: RPESC-RPE-4W||Phase 1 Phase 2|
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 (Clinical Trial)|
|Estimated Enrollment :||18 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase1/2a, Open-Label Study to Evaluate the Safety and Tolerability of RPESC-derived RPE Transplantation as Therapy for Dry Age-related Macular Degeneration (AMD)|
|Actual Study Start Date :||April 5, 2022|
|Estimated Primary Completion Date :||May 31, 2025|
|Estimated Study Completion Date :||May 31, 2025|
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.
- 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
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04627428
|Contact: Jeffrey H Stern, M.D., Ph.D.||firstname.lastname@example.org|
|United States, Michigan|
|University of Michigan Kellogg Eye Center||Recruiting|
|Ann Arbor, Michigan, United States, 48105|
|Contact: Rajesh C Rao, M.D.|
|Principal Investigator:||Rajesh C Rao, M.D.||University of Michigan Kellogg Eye Center|