General Information |
| Summary |
This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD). |
| Description |
This study is a Phase I/II, open-label, non randomized, sequential, multi-center clinical trial. There will be 5 cohorts, the 4 low vision cohorts will contain 3 patients, the better vision cohort will contain 4 patients. The enrolled cohorts will be as follows:
Three SMD patients- 50,000 MA09-hRPE cells transplanted
Three SMD patients- 100,000 MA09-hRPE cells transplanted
Four Better Vison SMD patients- 100,000 MA09-hRPE cells transplanted
Three SMD patients- 150,000 MA09-hRPE cells transplanted
Three SMD patients- 200,000 MA09-hRPE cells transplanted
Patients will be enrolled sequentially, and within each cohort of 3 patients, each patient's clinical course over the first 6 weeks following cell transplantation will be reviewed by an independent (DSMB) before enrollment is opened for the next 2 patients. A full safety assessment of all 3 patients in each cohort will be made by the DSMB when the 3rd patient in each cohort completes 4 weeks of follow-up, and before the first patient in the next cohort receives a cell transplant. The exception is the better vision group where all patients may be enrolled once DSMB approval has been received.
Each cohort will be enrolled sequentially in turn, with the exception of the better vision cohort which may be enrolled in parallel with the other cohorts.
The day of the cell implantation will be Day 0, and patients will remain in the study until the last visit at 12 months. |
| Clinical trials phase |
Phases 1/2 |
| Start date (estimated) |
2011-06-16 |
| End date (estimated) |
2015-08-10 |
| 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. |
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| Publications |
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Administrative Information |
| NCT number |
NCT01345006 |
| ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT01345006 |
| Other study identifiers |
| Name |
ACT SMD 01 MA09-hRPE |
| Description |
(Other Identifier: Sponsor) |
| Name |
ACT MA09-hRPE SMD-001 |
| Description |
(Other Identifier: Sponsor) |
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| Source weblink |
http://www.clinicaltrials.gov/ct2/show/NCT01345006 |
| 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.; 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. |
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Recruitment |
| Recruitment Status |
Completed |
| Estimated number of participants |
13 |
