Safety and Tolerability of RPE Stem Cell-derived RPE(RPESC-RPE) Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: RPESC-RPE-4W.
- Who it may be relevant to
- Registry conditions: Dry Age-related Macular Degeneration. Basic parameters: from 55 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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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)
Overview
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
- Biological RPESC-RPE-4W
RPESC-RPE-4W
Primary outcome measures
- Safety and tolerability of RPESC-RPE-4W transplantation [Time frame: 24 months]
Secondary outcome measures (4)
- 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]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- Retina Vitreous Associates Medical Group — Beverly Hills
- Spencer Center for Vision Research — Palo Alto
- University of Michigan Kellogg Eye Center — Ann Arbor
Identifiers
NCT: NCT04627428 · RPESC-RPE-01 · U01EY030581 · UG3EY031810