Stem Cell Therapy for Age-Related Macular Degeneration
Trial Summary
What is the purpose of this trial?
Age-related macular degeneration (AMD) is an eye disease which causes people to lose their sharp central vision over time. Aging damages the macula, which is in the middle of the retina - the light-sensitive part at the back of the eye. There are 2 types of AMD - wet AMD and dry AMD. The advanced stage of dry AMD causes vision loss. This is known as geographic atrophy. AMD makes everyday tasks like reading or driving difficult. ASP7317 is a potential new treatment for people with AMD. ASP7317 are human stem cells which have changed into cells found in the retina. ASP7317 is injected under the macula. It is hoped that ASP7317 will replace some of the damaged cells in the macula and improve vision for people with dry AMD. Before ASP7317 is available as a treatment, the researchers need to check its safety and how well it is tolerated. They will also check for signs of improved vision. People taking part in this study will be older people who have geographic atrophy caused by dry AMD. This is an open-label study. This means that people in this study and clinic staff will know that people will receive ASP7317. There will be 3 doses of ASP7317. These are low, medium and high numbers of cells. ASP7317 will be injected under the macula after the person is given either a local or a general anesthetic. To prevent the body from rejecting the cells, people will take tablets of tacrolimus a few days before receiving ASP7317 for up to a few weeks afterwards. Other medicines will be taken during this time to stop infections. There will be 2 groups in the study. Group 1 will be people with severe vision loss and Group 2 will be people with moderate vision loss. There will be different small groups of people within Group 1 and Group 2, with each small group receiving 1 of the 3 doses of ASP7317. Different small groups of people within Group 1 and Group 2 will receive lower to higher doses of ASP7317. Each small group will only receive 1 dose. Group 1 will start treatment first. At each dose, a medical expert panel will check the results of the first person in the group to decide if the rest of the group will receive the same dose. Then, the panel will decide if more people may receive the same dose or if the next group may receive the next highest dose. The panel will use the results from the lower dose of Group 1 to decide when Group 2 starts treatment (also at the lower dose). The panel will also use the results of the middle and higher doses in Group 1 to decide when and how many people in Group 2 can receive these doses. During the study, people will visit the clinic several times for up to 12 months (1 year). During all visits, the study doctors will check for any medical problems after receiving ASP7317. Vital signs will be checked a few days before treatment with ASP7317 and up to about a month afterwards. Vital signs include blood pressure, pulse, and temperature. At some visits, the study doctors will also take blood samples for blood tests. At most visits, people will have eye tests and have different images, scans, and measurements taken. This could be for the affected eye or both eyes, depending on the test. People can visit the clinic extra times, if needed.
Will I have to stop taking my current medications?
The trial requires participants to stop taking any medications that strongly interact with tacrolimus, a drug used in the study. If you are on such medications, you will need to discontinue them. However, if you are taking an antidepressant, you must continue it at a stable and effective dosage.
What data supports the effectiveness of the treatment for age-related macular degeneration?
Research shows that transplanting human embryonic stem cell-derived retinal pigment epithelial cells can improve vision in patients with macular degeneration by replacing damaged cells in the eye. Studies have demonstrated that these cells can integrate into the retina and lead to functional improvements, although challenges remain in optimizing their use.12345
Is stem cell therapy for age-related macular degeneration safe?
Research shows that stem cell therapy using human embryonic stem cell-derived retinal pigment epithelial cells has been generally safe in early trials for age-related macular degeneration. No major safety issues like tumor growth or severe inflammation were reported, although one case of infection occurred after surgery.23467
How is the treatment ASP7317 unique for age-related macular degeneration?
ASP7317 is unique because it uses human embryonic stem cell-derived retinal pigment epithelial cells to replace damaged cells in the eye, potentially improving vision in patients with age-related macular degeneration. This approach is novel as it aims to restore the function of the retina by directly transplanting healthy cells, unlike other treatments that mainly focus on slowing disease progression.23489
Research Team
Medical Director
Principal Investigator
Astellas Pharma Global Development, Inc.
Eligibility Criteria
Adults over 50 with dry age-related macular degeneration (dry AMD) and geographic atrophy, resulting in central vision loss. Participants must be willing to discontinue certain supplements and medications, agree to use contraception if of childbearing potential, and not have a history of certain eye treatments or conditions that could affect the study's outcome.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ASP7317 injections under the macula with varying doses, along with tacrolimus and other medications to prevent infection
Follow-up
Participants are monitored for safety and effectiveness after treatment, including eye tests and imaging
Open-label extension (optional)
Participants may opt into continuation of treatment long-term
Treatment Details
Interventions
- ASP7317
- Tacrolimus
Find a Clinic Near You
Who Is Running the Clinical Trial?
Astellas Institute for Regenerative Medicine
Lead Sponsor