Dry Age-related Macular DegenerationPhase 1/2NCT04627428
Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
A Phase 1/2 study testing RPESC-RPE-4W in people with Dry Age-related Macular Degeneration, run by Luxa Biotechnology, LLC at 3 sites in United States.
Trial summary
The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD. Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Dry Age-related Macular Degeneration
- What is being tested?
- RPESC-RPE-4W
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 3 sitesUnited States
- Study dates
- 5 Apr 2022to 1 Dec 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105
Rajesh C Rao, M.D.
- Recruiting
Retina Vitreous Associates Medical Group
Beverly Hills, California, 90211
David S Liao, MD, PhD
- Recruiting
Spencer Center for Vision Research
Palo Alto, California, 94303
Theodore Leng, MD, FACS
About this study
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)
The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.
Read the 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
- Conditions
- Dry Age-related Macular Degeneration
- Being tested
- RPESC-RPE-4W
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RPESC-RPE-4WBiological
RPESC-RPE-4W
| Study group | Role | What they receive |
|---|---|---|
| 50,000 cells | Experimental | Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye. Receives: Biological: RPESC-RPE-4W |
| 150,000 cells | Experimental | Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye. Receives: Biological: RPESC-RPE-4W |
| 250,000 cells | Experimental | Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye. Receives: Biological: RPESC-RPE-4W |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 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
Measured: 24 months
Secondary outcomes (4)
Change in the mean of Best Corrected Visual Acuity (BCVA)
Change in visual acuity will be measured by ETDRS chart.
Measured: 24 months
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.
Measured: 24 months
Change in GA lesion area
Change in GA lesion area will be measured.
Measured: 24 months
Evidence of structural changes
Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2020
- Study start5 Apr 2022
- Primary completion1 Dec 2026 (estimated)
- Study completion1 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Dry Age-related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Luxa Biotechnology, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Institutes of Health (NIH), National Eye Institute (NEI), Regenerative Research Foundation, California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Sponsor
- Investigators
- Rajesh C Rao, M.D., Principal investigator, University of Michigan Kellogg Eye CenterTheodore Leng, MD, FACS, Principal investigator, Spencer Center for Vision Research at Stanford UniversityDavid S Liao, MD, PhD, Principal investigator, Retina-Vitreous Associates Medical Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04627428
- Sponsor's protocol ID
- RPESC-RPE-01
- Other IDs
- U01EY030581, UG3EY031810
- Status last verified
- Oct 2025
- First submitted
- 1 Nov 2020
- Last updated
- 15 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Rao RC, Arduini BL, Borden S, Sareen D, Svendsen C, Lee P, Ryan C, Kodati S, Nyaiburi C, Wolsieffer K, Oh E, Park S, Ford G, Dionne K, Temple S, Stern J. Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes. Cell Stem Cell. 2025 Nov 6;32(11):1659-1670.e4. doi: 10.1016/j.stem.2025.08.012. Epub 2025 Sep 16.
PubMed 40961946
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04627428
- Acronym / sponsor ID
- RPESC-RPE-01
- Conditions
- Dry Age-related Macular Degeneration
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Luxa Biotechnology, LLCIndustry (pharma, biotech, devices)
- Collaborators
- National Institutes of Health (NIH), National Eye Institute (NEI), Regenerative Research Foundation, California Institute for Regenerative Medicine (CIRM)
- Study start
- 5 Apr 2022 (actual)
- Primary completion
- 1 Dec 2026 (estimated)
- Study completion
- 1 Dec 2026 (estimated)
- First posted
- 13 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 50,000 cells | Experimental | Biological: RPESC-RPE-4WSix patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye. |
| 150,000 cells | Experimental | Biological: RPESC-RPE-4WSix patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye. |
| 250,000 cells | Experimental | Biological: RPESC-RPE-4WSix patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye. |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RPESC-RPE-4WRPESC-RPE-4W | Biological | 150,000 cells, 250,000 cells, 50,000 cells |
Primary outcome
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 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
4 secondary outcomes
Change in the mean of Best Corrected Visual Acuity (BCVA)
Time frame: 24 months
Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry
Time frame: 24 months
Change in GA lesion area
Time frame: 24 months
Evidence of structural changes
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2025.
- Derived from this study: PubMed 40961946Rao RC, Arduini BL, Borden S, Sareen D, Svendsen C, Lee P, Ryan C, Kodati S, Nyaiburi C, Wolsieffer K, Oh E, Park S, Ford G, Dionne K, Temple S, Stern J. Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes. Cell Stem Cell. 2025 Nov 6;32(11):1659-1670.e4. doi: 10.1016/j.stem.2025.08.012. Epub 2025 Sep 16.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Rajesh C Rao, M.D. principal investigator
University of Michigan Kellogg Eye Center
Theodore Leng, MD, FACS principal investigator
Spencer Center for Vision Research at Stanford University
David S Liao, MD, PhD principal investigator
Retina-Vitreous Associates Medical Group
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing RPESC-RPE-4W in people with Dry Age-related Macular Degeneration, run by Luxa Biotechnology, LLC at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jeffrey H Stern, M.D., Ph.D. (05184371111, jeffreystern@luxabiotech.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of RPESC-RPE-4W transplantation (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04627428, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2025.