Badanie skuteczności i bezpieczeństwa stosowania decytabiny i tetrahydrourydyny u pacjentów z niedokrwistością sierpowatokrwinkową, którzy nie kwalifikują się do leczenia hydroksykarbamidem

Sickle cell disease is a condition that affects the blood, causing red blood cells to become misshapen and break down. This study is looking at how well a new treatment works for people with this disease. The treatment being tested is a combination of two substances: decitabine and tetrahydrouridine, taken in the form of a capsule. The study will compare this treatment to a placebo, which looks like the treatment but does not contain the active substances.

The main goal of the study is to see if the new treatment can improve hemoglobin levels in patients who cannot take another medication called hydroxycarbamide. Hemoglobin is a protein in red blood cells that carries oxygen throughout the body. Participants in the study will receive either the new treatment or a placebo and will be monitored over a period of time to see how their hemoglobin levels change.

The study will last for about 48 weeks, during which participants will have regular check-ups to monitor their health and the effects of the treatment. The researchers will also look at other factors, such as the number of pain episodes related to sickle cell disease and any side effects experienced by the participants. This research aims to find a new way to help people with sickle cell disease manage their condition more effectively.