Phase 1 Study Comparing Oral Nicotinamide and THU-Decitabine for Treating High Risk Sickle Cell Disease

What this Trial Is About

Researchers are conducting a phase 1 randomized controlled trial to evaluate treatments for high risk sickle cell disease. The study compares oral THU-decitabine to nicotinamide alone and then assesses the combination of nicotinamide with THU-decitabine. This trial aims to better understand the effects of these treatments on patients who continue to experience symptoms despite prior therapy. Participants receive either oral nicotinamide 1000 mg twice daily or oral THU 250 mg with decitabine 5 mg once per week for 12 weeks. After this period, all participants receive the combination treatment for another 12 weeks. Those who wish can join an extension phase of combination therapy lasting an additional 24 weeks, making a total treatment period of up to 48 weeks. Throughout the study, participants undergo assessments including blood tests to measure hemoglobin levels at 12 weeks. Researchers monitor the effects of the treatments on sickle cell disease symptoms and safety. The study involves regular visits to track treatment response and side effects, with a focus on the participants overall well-being during and after the treatment periods.

A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease