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

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age55+
SponsorLuxa Biotechnology, LLC

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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).

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • RPESC-RPE-4W

    Biological/Vaccine

    RPESC-RPE-4W

Treatment groups

18 Participants
are divided into 3 treatment groups
Group A: 50,000 cellsExperimental treatment 1 intervention
Group B: 150,000 cellsExperimental treatment 1 intervention
Group C: 250,000 cellsExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
24 months

Secondary outcomes

1

Change in the mean of Best Corrected Visual Acuity (BCVA)

Change in visual acuity will be measured by ETDRS chart.

Time frame
24 months
2

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.

Time frame
24 months
3

Change in GA lesion area

Change in GA lesion area will be measured.

Time frame
24 months
4

Evidence of structural changes

Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography

Time frame
24 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Luxa Biotechnology, LLC

Lead sponsor

National Institutes of Health (NIH)

Collaborator

National Eye Institute (NEI)

Collaborator

Regenerative Research Foundation

Collaborator

California Institute for Regenerative Medicine (CIRM)

Collaborator