General Information |
| Summary |
The purpose of this study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced Stargardt's Macular Dystrophy (SMD) 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 SMD 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 (three patients), 100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells transplanted (three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted (one patient). Three patients with severe to moderate vision 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 Outcomes. 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) |
2012-07-11 |
| End date (estimated) |
2019-06-21 |
| 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 |
NCT02445612 |
| ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT02445612 |
| Other study identifiers |
| Name |
ACT MA09-hRPE 001(SMD)LTFU |
| Description |
(Other Identifier: Sponsor) |
|
| Source weblink |
https://clinicaltrials.gov/ct2/show/NCT02445612 |
| 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 |
