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

General Information

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.
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.
Clinical trials phase Long term follow up
Start date (estimated) 2012-07-11
End date (estimated) 2019-12-31
Clinical feature
Label atrophic macular degeneration
Link http://www.ebi.ac.uk/efo/EFO_1001492
Description Dry AMD is most common type of macular degeneration and affects 90% of the people who have the condition. In the dry form, there is a breakdown or thinning of the layer of retinal pigment epithelial cells (RPE) in the macula. No medical or surgical treatment is available for this condition.
Publications

Administrative Information

NCT Number NCT02463344
Other study identifiers
Name 7316-CL-0005
Name ACT MA09-hRPE AMD -001 LTFU
Description (Other Identifier: Sponsor)
Source weblink https://clinicaltrials.gov/ct2/show/NCT02463344
Regulatory body approval
Name Food and Drug Administration (FDA)
Country
United States
Public contact
Country
Sponsors Astellas Pharma Inc.

Cell Line

Stem cell lines obtained from

Recruitment

Recruitment Status Active, not recruiting
Estimated number of participants 11