Clinical trial NCT04627428: Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Dry Age-related Macular Degeneration
Lead sponsor: Luxa Biotechnology, LLC
Sponsor class: INDUSTRY
Collaborators: name: National Institutes of Health (NIH)
name: National Eye Institute (NEI)
name: Regenerative Research Foundation
First posted: 2020-11-13
Enrollment: 18
Sex: ALL
Minimum age (years): 55
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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

Eligibility criteria: 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.

Primary outcomes:
measure: Safety and tolerability of RPESC-RPE-4W transplantation
timeFrame: 24 months
Secondary outcomes:
measure: Change in the mean of Best Corrected Visual Acuity (BCVA)
timeFrame: 24 months
measure: Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry
timeFrame: 24 months
measure: Change in GA lesion area
timeFrame: 24 months
measure: Evidence of structural changes
timeFrame: 24 months
Other outcomes:
Locations:
city: Ann Arbor
country: United States
facility: University of Michigan Kellogg Eye Center
state: Michigan
Study officials:
affiliation: University of Michigan Kellogg Eye Center
name: Rajesh C Rao, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Spencer Center for Vision Research at Stanford University
name: Theodore Leng, MD, FACS
role: PRINCIPAL_INVESTIGATOR
affiliation: Retina-Vitreous Associates Medical Group
name: David S Liao, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: