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

Dry Age-related Macular Degeneration Clinical Trial

Official 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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04627428
• Other study ID #: RPESC-RPE-01
• Secondary ID: U01EY030581UG3EY
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: April 5, 2022
• Est. completion date: May 31, 2025

Study information

• Verified date: October 2023
• Source: Luxa Biotechnology, LLC
• Contact: Jeffrey H Stern, M.D., Ph.D.
• Phone: 05184371111
• Email: jeffreystern[@]luxabiotech.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 18
• Est. completion date: May 31, 2025
• Est. primary completion date: May 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 55 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Dry Age-related Macular Degeneration
• Macular Degeneration

Intervention

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

Locations

Country	Name	City	State
United States	University of Michigan Kellogg Eye Center	Ann Arbor	Michigan

Sponsors (4)

Lead Sponsor	Collaborator
Luxa Biotechnology, LLC	National Eye Institute (NEI), National Institutes of Health (NIH), Regenerative Research Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 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	24 months
Secondary	Change in the mean of Best Corrected Visual Acuity (BCVA)	Change in visual acuity will be measured by ETDRS chart.	24 months
Secondary	Loss of =10 decibels of ten-degree average visual sensitivity microperimetry	Loss of =10 decibels of ten-degree average visual sensitivity will by measured by microperimetry.	24 months
Secondary	Change in GA lesion area	Change in GA lesion area will be measured.	24 months
Secondary	Evidence of structural changes	Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography	24 months