Clinical trials

Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye. In people with macular dystrophies, some of the cells in the macula gradually stop working and may die over time. This leads to loss of central vision, which can make it harder to read, recognize faces or see fine details. What's seen out of the corner of the eye (peripheral vision) is mostly unaffected. Stargardt disease (STGD) is a type of macular dystrophy which is caused by 1 faulty gene (ABCA4). Vision loss most typically begins in childhood or teenage years but may also develop in adulthood. As well as STGD, there are other macular dystrophies that look very similar to STGD and are called STGD-like macular dystrophies. These are caused by many other different genes. Together, STGD and STGD-like conditions can be called STGD-type macular dystrophies. This is an early development study of ASP2020 in adults, teenagers, and children with STGD-type macular dystrophies. ASP2020 are human stem cells which have been changed into cells found in the macula. In this study ASP2020 will be given to people for the first time. The main aim of the study is to check the safety of ASP2020 and how well people tolerate it. Other aims are to learn if people have an immune reaction to ASP2020, and if there are signs that the stem cells replace damaged cells in the retina, and vision improves for people with STGD-type macular dystrophies. ASP2020 will be given as a single injection into the eye, under the retina. This requires a surgical procedure where the person is put to sleep by a general anesthetic. At the end of surgery, a steroid will be injected into the eye to reduce any swelling. The study has 2 parts. In Part 1, different small groups will receive a lower to higher dose of ASP2020. This is done to find a suitable dose to use in Part 2. The adults will receive the lower dose and higher dose before the teenagers. There will be a 6-month gap between the last adult receiving the lower dose of ASP2020 and the first teenager receiving the same lower dose. This will also happen for the last adult receiving the higher dose of ASP2020 and the first teenager receiving the higher dose of ASP2020. Any medical problems will be recorded for each dose in each group. Children will not receive ASP2020 in Part 1. In Part 2, different groups of adults, teenagers and children will receive the most suitable dose of ASP2020 worked out from Part 1. People will be in the study for about 1 year and they will visit the clinic several times. In both parts of the study, safety checks will be done at each visit, and the study doctors will continue to check for any medical problems throughout the study. Various eye tests and eye imaging will be done throughout the study. Blood tests will also be done at some of the visits during the study.

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.