Dry Age-related Macular Degeneration
Conditions
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
Interventions
BIOLOGICALRPESC-RPE-4W
RPESC-RPE-4W
Sponsors
National Institutes of Health (NIH)
National Eye Institute (NEI)
Regenerative Research Foundation
California Institute for Regenerative Medicine (CIRM)
Luxa Biotechnology, LLC
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
55 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in the mean of Best Corrected Visual Acuity (BCVA) | 24 months | Change in visual acuity will be measured by ETDRS chart. |
| 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. |
| Change in GA lesion area | 24 months | Change in GA lesion area will be measured. |
| Evidence of structural changes | 24 months | Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography |
Countries
United States
Contacts
Primary ContactJeffrey H Stern, M.D., Ph.D.
Outcome results
None listed