Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients

General Information

Summary The purpose of this study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced Stargardt's Macular Dystrophy (SMD) 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 SMD 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 (three patients), 100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells transplanted (three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted (one patient). Three patients with severe to moderate vision 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 Outcomes. 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) 2019-06-21
Clinical feature
Label Stargardt disease
Link http://purl.obolibrary.org/obo/DOID_0050817
Description An age related macular degeneration that is characterized by progressive vision loss usually to the point of legal blindness.

Administrative Information

NCT number NCT02445612
ICTRP weblink https://trialsearch.who.int/Trial2.aspx?TrialID=NCT02445612
Other study identifiers
Name 7316-CL-0004
Description (Other Identifier: Sponsor)
Source weblink https://clinicaltrials.gov/ct2/show/NCT02445612
Public contact
Email astellas.registration@astellas.com
Public email astellas.registration@astellas.com
Last name Study Director: Global Therapeutic Area Head & Chief Medical Officer, Astellas Institute for Regenerative Medicine
United States
Sponsors Astellas Institute for Regenerative Medicine


Source pluripotent stem cell lines
Which differentiated cell type is used
Label retinal pigment epithelial cell
Link http://purl.obolibrary.org/obo/CL_0002586
Description An epithelial cell of the retinal pigmented epithelium.


Recruitment Status Completed
Estimated number of participants 13