Research With Retinal Cells Derived From Stem Cells for Myopic Macular Degeneration

General Information

Summary Pathologic myopia is a major cause of legal blindness worldwide. In myopic macular degeneration (MMD), there is degeneration of the retinal pigment epithelial (RPE) layer, and associated photoreceptors, resulting in vision loss. There is currently no standard treatment for MMD. Transplantation of intact sheets of RPE and suspensions of isolated individual RPE cells as well as autologous translocation of RPE cells has been attempted as treatment for AMD. Human photoreceptors are comprised of two cell types-rods and cones. Both have a close relationship with the outermost retinal cells, the retinal pigmented epithelium (RPE). The RPE is located between the choroid and the photoreceptors. The RPE maintains photoreceptor function by recycling photopigments,delivering, metabolizing and storing vitamin A, phagocytosing rod photoreceptor outer segments, transporting iron and small molecules between retina and choroid, maintaining Bruch's membrane and absorbing stray light to allow better image resolution. In essence, the RPE layer is critical to the function and health of photoreceptors and the retina as a whole. Human PRE (hRPE) transplantation may be a viable option for treatment of degenerative diseases of the retina. MA09-hRPE cells are fully differentiated human RPE cells derived from embryonic stem cells. Transplanted hRPE cells prepared by Advanced Cell Technology have been studied in rodent models of macular degenerative disease. The data suggests that the subretinal injection of ACT's hRPE cell products rescues, or at least delays, loss of visual function in two animal models of retinal degenerative diseases. The main purpose of this study is to evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with Myopic Macular Degeneration (MMD). Another objective is to evaluate potential efficacy endpoints to be used in future studies of RPE cellular therapy.
Clinical trials phase Phases 1/2
Start date (estimated) 2013-03-01
End date (estimated) 2016-06-30
Clinical feature
Label NON RARE IN EUROPE: Myopic macular degeneration
Description A rare, genetic macular disorder characterised by severe near-sightedness resulting from continual elongation of the eyeball. As the eyeball stretches the sclera and retina thin and the macula can tear, causing bleeding beneath the retina. It is a major cause of irreversible vision loss.

Administrative Information

NCT number NCT02122159
ICTRP weblink
Other study identifiers
Source weblink
Regulatory body approval
Name Food and Drug Administration (FDA)
United States
Public contact
Public email
First name Steven
Last name Schwartz
Phone +1 310-206-7474
City Los Angeles
United States
Sponsors University of California, Los Angeles


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 (Study to be revised. Future plans to be determined.)