Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
Primary Purpose
Dry Age-related Macular Degeneration
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
RPESC-RPE-4W
Sponsored by
About this trial
This is an interventional treatment trial for Dry Age-related Macular Degeneration
Eligibility Criteria
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.
Sites / Locations
- University of Michigan Kellogg Eye CenterRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
50,000 cells
150,000 cells
250,000 cells
Arm Description
Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.
Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.
Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Change in the mean of Best Corrected Visual Acuity (BCVA)
Change in visual acuity will be measured by ETDRS chart.
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.
Change in GA lesion area
Change in GA lesion area will be measured.
Evidence of structural changes
Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography
Full Information
NCT ID
NCT04627428
First Posted
November 1, 2020
Last Updated
October 3, 2023
Sponsor
Luxa Biotechnology, LLC
Collaborators
National Institutes of Health (NIH), National Eye Institute (NEI), Regenerative Research Foundation
1. Study Identification
Unique Protocol Identification Number
NCT04627428
Brief Title
Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
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)
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 5, 2022 (Actual)
Primary Completion Date
May 31, 2025 (Anticipated)
Study Completion Date
May 31, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Luxa Biotechnology, LLC
Collaborators
National Institutes of Health (NIH), National Eye Institute (NEI), Regenerative Research Foundation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.
Detailed 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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dry Age-related Macular Degeneration
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
18 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
50,000 cells
Arm Type
Experimental
Arm Description
Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.
Arm Title
150,000 cells
Arm Type
Experimental
Arm Description
Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.
Arm Title
250,000 cells
Arm Type
Experimental
Arm Description
Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.
Intervention Type
Biological
Intervention Name(s)
RPESC-RPE-4W
Intervention Description
RPESC-RPE-4W
Primary Outcome Measure Information:
Title
Safety and tolerability of RPESC-RPE-4W transplantation
Description
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
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Change in the mean of Best Corrected Visual Acuity (BCVA)
Description
Change in visual acuity will be measured by ETDRS chart.
Time Frame
24 months
Title
Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry
Description
Loss of ≥10 decibels of ten-degree average visual sensitivity will by measured by microperimetry.
Time Frame
24 months
Title
Change in GA lesion area
Description
Change in GA lesion area will be measured.
Time Frame
24 months
Title
Evidence of structural changes
Description
Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jeffrey H Stern, M.D., Ph.D.
Phone
05184371111
Email
jeffreystern@luxabiotech.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rajesh C Rao, M.D.
Organizational Affiliation
University of Michigan Kellogg Eye Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Michigan Kellogg Eye Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rajesh C Rao, M.D.
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
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