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) |
2013-02-25 |
End date (estimated) |
2019-08-19 |
Clinical feature |
Label |
age related macular degeneration |
Link |
http://purl.obolibrary.org/obo/DOID_10871 |
Description |
A degeneration of macula and posterior pole that is characterized by a loss of vision in the center of the visual field (the macula) resulting from damage to the retina and resulting in blurring of the sharp central vision.; OMIM mapping confirmed by DO. [SN]. |
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Publications |
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Administrative Information |
NCT number |
NCT02463344 |
ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT02463344 |
Other study identifiers |
Name |
ACT MA09-hRPE AMD -001 LTFU |
Description |
(Other Identifier: Sponsor) |
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Source weblink |
https://clinicaltrials.gov/ct2/show/NCT02463344 |
Regulatory body approval |
Name |
Food and Drug Administration (FDA) |
Country |
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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 |
Country |
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Sponsors |
Astellas Institute for Regenerative Medicine |
Cells |
Source pluripotent stem cell lines |
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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. |
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Recruitment |
Recruitment Status |
Completed |
Estimated number of participants |
11 |