Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat High Risk Sickle Cell Disease
EpiDestiny, Inc.
Summary
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
Description
A randomized control trial comparing oral THU-decitabine to nicotinamide (1:1 randomization), and then comparing the effects of the combination of nicotinamide with THU-decitabine vs either treatment alone. Treatment with each agent alone is for 12 weeks followed by the combination for a further 12 weeks. Patients have the option to enter an extension phase of combination treatment for an additional 24 weeks (total of 48 weeks)
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Age 18 years or older. * Written informed consent provided by the subject before study entry. * Confirmed sickle cell disease (SCD) as determined by hemoglobin electrophoresis or liquid chromatography. * Subject is in his/her steady state and not having any acute complication due to SCD (i.e., hospitalization, acute pain, or acute chest syndrome in the past 14 days). * Weight at least 40kg * Regular compliance with comprehensive care and previous therapy. * Symptomatic SCD is defined as having one of following, despite at least 6 months of hydroxyurea therapy, or refuse…
Interventions
- DrugNicotinamide
Oral nicotinamide (Vitamin B3) alone compared to THU Decitabine combination
Location
- University of Illinois at Chicago College of MedicineChicago, Illinois