Study to Evaluate Sub-retinal Transplantation of Retinal Pigmented Epithelial Cells in Patients With Dry AMD (PORTRAY)

General Information

Summary To evaluate the safety of 3 regimens of short-term, low-dose systemic IMT as rejection prophylaxis prior to and/or following transplant of MA09-hRPE cells.
Description This study will be a Phase 2, double-masked, randomized, parallel group, sham surgery/placebo control, multi-center trial. Subjects will be randomized in a 3:1 ratio to either the treatment or control group respectively. Subjects randomized to the treatment group will receive transplantation with 200,000 MA09-hRPE (human embryonic stem cell derived retinal pigmented epithelial)cells in one eye. Subjects randomized to the control group will have a sham surgery without transplantation of MA09-hRPE cells. The study eye must meet all eligibility criteria. If both eyes meet all eligibility criteria, then the study eye will be the eye with the worst Best Corrected Visual Acuity (BCVA) score at screening. If both eyes have identical BCVA scores, then the study eye will be chosen by the Investigator and the subject. There will be 3 cohorts, each with a different regimen of low-dose IMT [tacrolimus and mycophenolate mofetil (MMF)]. Subjects will be randomized to treatment or control within cohorts, defined by severity of BCVA in the study eye at Screening. Enrollment in Cohort 1 and 2 will be concurrent. Enrollment into Cohort 3 will begin once Cohort 2 is fully enrolled.
Clinical trials phase Phase 2
Start date (estimated) 2015-08-24
End date (estimated) 2017-05-05
Clinical feature
Label age related macular degeneration
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].

Administrative Information

NCT number NCT02563782
ICTRP weblink
Other study identifiers
Name 7317-CL-0002
Description ( Other Identifier: Sponsor )
Source weblink
Regulatory body approval
Name Food and Drug Administration (FDA)
United States
Public contact
Public email
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
Description An epithelial cell of the retinal pigmented epithelium.


Recruitment Status Withdrawn
Comment recruitment status Changes to the study design and the cell line