Testing new medications to help people with severe sickle cell disease

This trial is testing whether a combination of nicotinamide and oral tetrahydrouridine (THU) and decitabine can help people with sickle cell disease who continue to have symptoms. You would take these medications by mouth over several months to see if they reduce pain episodes, improve blood counts, or decrease other complications of the disease. The study compares how well each medication works alone and then together.

Many people with sickle cell disease continue to suffer even after trying standard treatments like hydroxyurea. Researchers believe this medication might work through a different mechanism to reduce symptoms and improve quality of life for patients who need better options.