This trial is active, not recruiting.

Condition age-related macular degeneration
Treatment ma09-hrpe
Sponsor Astellas Institute for Regenerative Medicine
Start date July 2012
End date August 2029
Trial size 11 participants
Trial identifier NCT02463344, ACT MA09-hRPE AMD -001 LTFU


This is the long term follow-up of 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 Age-Related Macular Degeneration(AMD).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Experimental: Subretinal injection of MA09-hRPE Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells
Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells

Primary Outcomes

Number of patients experiencing a grade 2 or greater adverse event related to the cell product or showed any evidence that the cells were contaminated with an infectious agent or showed any evidence that the cells have tumorigenic potential
time frame: 15 years

Secondary Outcomes

Exploratory evaluations for potential efficacy endpoints.
time frame: 15 years
Exploratory evaluation for successful engraftment.
time frame: 15 years

Eligibility Criteria

Male or female participants at least 55 years old.

Inclusion Criteria: - Must have met the eligibility of the core protocol. - Able to understand and willing to sign the informed consent to participate in the follow up. - Treated with hESC-RPE cell transplant in the core protocol. - Previous participation in study Exclusion Criteria: - Unwilling or unable to participate in the study.

Additional Information

Official title Long Term Follow up to A Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-RPE) Cells in Patients With Advanced Dry Age Related Macular Degeneration (AMD)
Description This study is a long term, non interventional follow up to the phase I/II, open-label, non randomized, sequential, multi-center trial (referred to as the core protocol) in which 13 AMD patients were treated. Ten poor vision patients (20/400) were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted (three subjects), 100,000 hESC-RPE cells transplanted (three subjects), 150,000 hESC-RPE cells transplanted (three subjects) and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted(one subject). Three better vision patients (20/100) were transplanted with a dose of 100,000 hESC-RPE cells. Eligible patients will be those who sign a consent form for the extension study, and who fulfill all inclusion/exclusion criteria. 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. During the first 2 years after completion of the core protocol, i.e. from Year 1 to Year 2 and from Year 2 to Year 3 post transplantation, the patients will be evaluated at approximately 3 month intervals; during years three thru five patients will be evaluated at approximately six month intervals. During years 6-15, patients will either be required to return to the study center or provide the name of an ophthalmologist in their locality who would provide an evaluation of the patient's vision and any abnormal findings and report these results to the study site annually. Follow up will include obtaining information about visual acuity, ophthalmological findings and adverse events (AEs), specifically including cancer, neurological, autoimmune or hematological disorders that developed or worsened since the last visit.
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by Astellas Pharma Inc.