Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Patients With AMD

Completed

• Conditions: Age-Related Macular Degeneration
• Interventions: Biological: MA09-hRPE

• Registration Number: NCT02463344
• Lead Sponsor: Astellas Institute for Regenerative Medicine

Brief Summary

The purpose of his study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry Age-Related Macular Degeneration (AMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.

Detailed Description

This study is a long term, follow up to the ACT MA09-hRPE 001 phase I/II trial. The phase I/II trial (referred to as the core protocol) was an open-label, non randomized, dose escalation, multi-center trial. Thirteen AMD patients were treated in this trial. Ten patients with profound vision loss (visual acuity \<= 20/400) received a single subretinal injection of MA09-hRPE cells, starting at a dose of 50,000 MA09-hRPE cells transplanted (three patients), 100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells (three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted (one patient). Three patients with severe to moderate loss (visual acuity \<= 20/100) received a dose of 100,000 MA09-hRPE cells. All patients who participated in the core protocol are eligible for participation in this follow-up protocol. The first visit of this long term follow-up protocol will correspond to the last visit of the core protocol, and will take place at 12 months post cell implantation. Informed consent will be obtained at this visit. Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more frequently as clinically indicated). Follow-up will include obtaining information about ophthalmological findings and events of special interest as defined in the Primary Outcome. At the last visit of this follow-up study, whether at 60 months post-transplant or at early discontinuation, patients will be invited to participate in a safety surveillance study for an additional 10 years under a separate protocol which will continue to monitor the long term risks of MA09-hRPE cell transplantation.

Study Timeline

• Study Start: 2013-02-25
• Study First Submitted: 2015-05-13
• Study First Submitted QC: 2015-06-02
• Study First Posted: 2015-06-04
• Primary Completion: 2019-08-19
• Study Completion: 2019-08-19
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-29
• Last Update Posted: 2024-10-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

Inclusion Criteria

• Must have been treated with hESC-RPE cell transplant in the core protocol.
• Able to understand and willing to sign the informed consent to participate in the follow up study.

Exclusion Criteria

• There are no exclusion criteria.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
MA09-hRPE	MA09-hRPE	Experimental: Subretinal injection of MA09-hRPE * Cohort 1 50,000 cells * Cohort 2 100,000 cells * Cohort 2a Better Vision 100,000 cells * Cohort 3 150,000 cells * Cohort 4 200,000 cells

Primary Outcome Measures

Name	Time	Method
Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product	4 years	This will include obtaining information about ophthalmological findings and Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, pregnancy in a female subject or the partner of a male subject and pregnancy outcome, any Adverse Event (AE) that causes a subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer, ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, and unexpected, clinically significant AEs possibly related to the cell transplant procedure or the investigational product (MA09-hRPE cells).

Secondary Outcome Measures

Name	Time	Method
Incidence of graft failure or rejection	4 years	Evidence of graft failure or rejection will consist of: Presence of retinal edema, cystoid macular edema, retinal white dots, retinal hemorrhage, serous retinal detachment, subretinal exudates, subretinal fibrosis, or vascular and/or optic disc leakage, elevated intraocular pressure or hypotony. Evidence of unanticipated and persistent or increasing non-infectious ocular inflammation (e.g., vasculitis, retinitis, choroiditis, vitritis, pars planitis, anterior segment inflammation/uveitis).
Number of patients with changes in ocular examinations or images	4 years	The number of patients with clinically significant absolute values or changes from baseline in Intra-ocular pressure (IOP) and Best Corrected Visual Acuity (BCVA) will be summarized.

Trial Locations

Locations (4)

Mass Eye and Ear; 🇺🇸 Boston, Massachusetts, United States

Jules Stein Eye Institute, UCLA School of Medicine; 🇺🇸 Los Angeles, California, United States

Bascom Palmer Eye Institute; 🇺🇸 Miami, Florida, United States

Wills Eye Institute-Mid Atlantic Retina; 🇺🇸 Philadelphia, Pennsylvania, United States