Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
- Conditions
- Dry Age-related Macular Degeneration
- Interventions
- Registration Number
- NCT04627428
- Lead Sponsor
- Luxa Biotechnology, LLC
- 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
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 18
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description 150,000 cells RPESC-RPE-4W Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye. 50,000 cells RPESC-RPE-4W Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye. 250,000 cells RPESC-RPE-4W Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.
- Primary Outcome Measures
Name Time Method Safety and tolerability of RPESC-RPE-4W transplantation 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
- Secondary Outcome Measures
Name Time Method Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry 24 months Loss of ≥10 decibels of ten-degree average visual sensitivity will by measured by microperimetry.
Evidence of structural changes 24 months Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography
Change in the mean of Best Corrected Visual Acuity (BCVA) 24 months Change in visual acuity will be measured by ETDRS chart.
Change in GA lesion area 24 months Change in GA lesion area will be measured.
Trial Locations
- Locations (1)
University of Michigan Kellogg Eye Center
🇺🇸Ann Arbor, Michigan, United States