This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo. Participants who are on HU treatment before joining the study will get NDec, NDec and placebo, or continue on HU. Which treatment participants get is decided by chance. Participants getting NDec and/or Placebo will get capsules to take twice weekly. The study will last for about a year.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
96
Participants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight
Participants will get capsules daily (oral administration) according to local labelling
Participants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight
University Of South Alabama
Mobile, Alabama, United States
Keck Medical Center of USC - Outpatient Clinic
Los Angeles, California, United States
UCSF Oakland Benioff ChildHosp
Oakland, California, United States
Center for Inherited Blood Dis
Orange, California, United States
Harbor-UCLA Medical Center
Torrance, California, United States
Change in total haemoglobin
measured in g/dL
Time frame: From baseline (week 0) to week 24
Cmax for decitabine from pharmacokinetic assessment
measured in ng/mL
Time frame: At week 24
Cmax for tetrahydrouridine from pharmacokinetic assessment
measured in ng/mL
Time frame: At week 24
Change in DNA methyltransferase 1 (DNMT1) activity
measured in MFI units
Time frame: From baseline (week 0) to week 24
Change in cytidine deaminase (CDA) activity
µmol/L/min
Time frame: From baseline (week 0) to week 24
Change in foetal haemoglobin (g/dL)
measured in g/dL
Time frame: From baseline (week 0) to week 24
Change in foetal haemoglobin as a proportion of total haemoglobin (%HbF)
measured in %
Time frame: From baseline (week 0) to week 24
Change in F-cell level as a proportion of total red blood cell (RBC) (%F-cells)
measured in %
Time frame: From baseline (week 0) to week 24
Change in haemolysis measure: absolute reticulocyte count
measured in cells × 10\^9/L
Time frame: From baseline (week 0) to week 24
Change in haemolysis measure: indirect bilirubin
measured in mg/dL
Time frame: From baseline (week 0) to week 24
Change in haemolysis measure: lactate dehydrogenase
measured in U/L
Time frame: From baseline (week 0) to week 24
Number of vaso-occlusive crises
number of events
Time frame: From baseline (week 0) to week 48
Number of acute chest syndrome
number of events
Time frame: From baseline (week 0) to week 48
Number of RBC units transfused
measured in Units
Time frame: From baseline (week 0) to week 48
Number of adverse events of grade 3 or higher
number of events
Time frame: From baseline (week 0) to week 52
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Clinical and Transl Res Center
Aurora, Colorado, United States
Carole&Ray Neag Comp S.C. Ctr
Farmington, Connecticut, United States
Tova Community Health Inc
Wilmington, Delaware, United States
Howard University Hospital Department Med
Washington D.C., District of Columbia, United States
Howard University
Washington D.C., District of Columbia, United States
...and 108 more locations