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

Luxa Biotechnology

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Dry Age-related Macular Degeneration

Treatments: Biological: RPESC-RPE-4W

Study type: Interventional

Funder types: Industry; Other; NIH

Identifiers

NCT04627428

UG3EY031810 (U.S. NIH Grant/Contract)

U01EY030581 (U.S. NIH Grant/Contract)

RPESC-RPE-01

Details and patient eligibility

About

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

Full 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

Enrollment: 18 estimated patients

Sex: All

Ages: 55+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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18 participants in 3 patient groups

50,000 cells

Experimental group

Description:

Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.

Treatment:

Biological: RPESC-RPE-4W

150,000 cells

Experimental group

Description:

Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.

Treatment:

Biological: RPESC-RPE-4W

250,000 cells

Experimental group

Description:

Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.

Treatment:

Biological: RPESC-RPE-4W