Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy (SMD)

General Information

Summary The purpose of this study is: To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD Group. When-MA09-hRPE cell transplantation to evaluate the safety of surgical procedures. In future studies intended to assess the number of transplanted hRPE cells. In the past, MA09-hRPE cell therapy used in the study was to evaluate the validity of the potential. Homologous retinal pigment epithelial cells derived from embryonic stem cells, future studies of drugs that are used in representing the potential validity to evaluate the optimal dose.
Description Not Provided
Clinical trials phase Phase 1
Start date (estimated) 2012-09-01
End date (estimated) 2015-06-30
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 NCT01625559
ICTRP weblink https://trialsearch.who.int/Trial2.aspx?TrialID=NCT01625559
Other study identifiers
Name CHA_CTP_0903
Source weblink https://clinicaltrials.gov/ct2/show/study/NCT01625559
Regulatory body approval
Name Korea Food and Drug Administration (KFDA)
Country
South Korea
Public contact
Email info@chabio.com
First name Wonkyung
Last name Song
Country
South Korea
Address freetext CHA Bundang Medical Center
Sponsors CHABiotech CO., Ltd

Cells

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.; This extended description was generated by ChatGPT and reviewed by the CellGuide team, who added references, and by the CL editors, who approved it for inclusion in CL. It may contain information that applies to only to some subtypes and species, and so should not be considered definitional. Retinal pigment epithelial (RPE) cells form a single layer of cells at the back of the eye sandwiched between the neurosensory retina and the choroid, playing a significant role in maintaining vision health. These pigment-laden cells are highly specialized and perform an array of metabolic and transport functions essential for the maintenance of the photoreceptor cells (rods and cones) in the retina. The pigmentation of RPE cells actively aids in the absorption of excess light and the prevention of light scattering, thus enhancing the eye's optical properties. The retinal pigment epithelium forms a key part of the blood/retina barrier. The cells have long sheet-like microvilli on their apical membrane that project into the light-sensitive outer segments of the photoreceptors, forming a close structural interaction. The basolateral membrane of the RPE interacts with the underlying Bruch’s membrane, which separates the RPE cells from fenestrated endothelium of the choriocapillaris. RPE cells support the photoreceptor by providing them with oxygen and nutrients (such as glucose, retinol and fatty acids) and removing waste products. They also recycle the visual pigment, in a process called the "visual cycle", where the RPE cells play a vital role in the regeneration of visual pigment (11-cis retinol) following the absorption of light. This is essential for the maintenance of photoreceptor excitability. Beyond this, RPE cells take part in the phagocytosis process, where they digest the shed ends of photoreceptor outer segments, thus, preventing the build-up of waste residue that could otherwise harm retinal health. They also secrete various factors, including growth factors required to maintain the structural integrity of choriocapillaris endothelium and photoreceptors, as well as immunosuppressive factors that play an important role in establishing the immune privilege of the eye.

Recruitment

Recruitment Status Unknown Status
Estimated number of participants 3