NCT02463344

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

Long Term Follow up to A Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Advanced Dry AMD

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 11 (actual)
• Sponsor: Astellas Institute for Regenerative Medicine · Industry
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Age-Related Macular Degeneration

Interventions

Type	Name	Description
BIOLOGICAL	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

Timeline

• Start date: 2013-02-25
• Primary completion: 2019-08-19
• Completion: 2019-08-19
• First posted: 2015-06-04
• Last updated: 2024-10-31

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study