Clinical Trials Directory
We are doing this study to find out if an investigational drug called iptacopan (the study drug) is safe and has beneficial effects for people who have generalized Myasthenia Gravis (gMG).
This study will last about 35 weeks and has three parts: Screening, Dosing, and Follow-up. It is a double-blind study, which means neither you nor the doctors will know if you get the real medicine or a placebo. The medicine or placebo will be given as two shots under the skin. After you agree to join, treatment will start. There will be phone calls at weeks 3, 5, 7, 9, and 11 to check on your medicine and any side effects. You will have regular health checkups, physical exams, and tests like ECGs to check your heart. You will also answer short surveys about your symptoms, energy, mood, and work. You will come back at 3, 6, and 12 months for blood tests. Over the whole study, about one cup of blood will be taken. These tests will check your health, medicine levels, antibodies, and screen for diseases like HIV, hepatitis, and tuberculosis.
In this study, participants will have different medical tests such as MRI scans, PET scans, heart tests, and exams to check how the brain and body are working. They will stay in the research unit for at least two nights while doctors watch their health closely. The study drug will be given through a vein in the arm. Participants must also have a study partner who can come with them to visits and help during the study.
This study lasts about nine to twelve months and includes time to check if a person can join, time to receive treatment, and time for follow up visits. People will come to the clinic for regular visits. At these visits, they may receive medicine through a vein, have blood tests, physical exams, and heart checks, and answer questions about how they feel and what they can do each day. Everyone will first receive a medicine called efgartigimod, which is already approved for myasthenia gravis. If their symptoms only improve a little, they may then receive a second study medicine called empasiprubart along with the first medicine. This second medicine is being tested to help block parts of the immune system that may still be causing symptoms. After treatment, participants will keep having checkups to make sure the medicines are safe, even if they stop taking them.
We are doing this study to find out if an experimental drug called CABA-201 (the study drug) is a safe and effective option for people with systemic sclerosis.
In this study, people may be placed into one of two groups when they join. One group will receive chemotherapy that lowers the immune system before getting the study treatment, and the other group will not. Everyone in the study will receive special CAR T cells that are made to fight disease. After receiving the cells, participants will stay in the hospital for several days so doctors can watch their health closely. They must also stay near the study center for about 45 days and will have regular doctor visits during that time.
In this study, participants will first receive chemotherapy for one to three days to lower their immune system. After that, they will receive special CAR T cells in the hospital and stay there so doctors can watch them closely. For the first month after receiving the cells, participants will be monitored very carefully and must stay near the study center for about 30 days. After that, they will return to the clinic once a month for at least two years so doctors can continue to check their health.