A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)

General Information

Summary The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
Description This study is a long-term, follow- up of a Phase I/II, open-label, non-randomized, 4-cohort, dose escalation, multi-center clinical trial (referred to as the core trial or core protocol) in which a maximum of 12 SMD patients were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted. All patients who participate in the core protocol are eligible for participation in the follow-up protocol. The first visit of this extension protocol will correspond to the last visit of the core protocol, and will take place at 12 months post-cell implantation. Informed consent for this follow-up protocol will be obtained at the first visit which will occur at the 12-month visit of the core protocol. 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 Outcome. 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 life-long annual health survey, under a separate protocol, to further monitor long-term safety.
Clinical trials phase Long term follow up
Start date (estimated) 2013-01-16
End date (estimated) 2019-12-31
Clinical feature
Label Stargardt Disease
Link http://purl.obolibrary.org/obo/NCIT_C85078
Description An autosomal recessive and rarely autosomal dominant inherited disorder caused by mutations in the ABCA4 or ELOVL4 genes respectively. It is characterized by macular degeneration that begins in late childhood resulting in progressive loss of vision.

Administrative Information

NCT Number NCT02941991
EudraCT Number 2012-002827-14
Other study identifiers
Name 7316-CL-0006
Name ACT-hESC-RPE-SMD-01FU EU
Description (Other Identifier: Sponsor)
Regulatory body approval
Country
Public contact
Country
Sponsors Astellas Pharma Inc.

Cell Line

Stem cell lines obtained from

Recruitment

Recruitment Status Active, not recruiting
Estimated number of participants 11