Tests nicotinamide with decitabine and tetrahydrouridine in sickle cell disease
Tests nicotinamide, alone or combined with other drugs, in adults with sickle cell disease.
Plain-language title generated from the registry record
Registry title: A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Key facts
- Where
- Chicago, United StatesSee the site
- Phase
- Phase 1
Where in United States
- University of Illinois at Chicago College of MedicineChicago, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- EpiDestiny, Inc.
- Trial code
- NCT04055818
- Record updated
- 30 September 2026
- Original description
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).Read full original descriptionShow less
Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat High Risk Sickle Cell Disease
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
Inclusion: 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 to take hydroxyurea for personal reasons: fetal hemoglobin <0.5 g/dL, or 3 or more pain episodes per year requiring parenteral narcotics, or 1 or more acute chest syndrome episodes, or Hemoglobin <9 g/dL and absolute reticulocyte count <250,000/mm3.
Exclusion: Experienced severe sepsis or septic shock within the previous 12 weeks; Last HU dose was ingested within the previous 4 weeks; Moribund or any concurrent disease (e.g., hepatic, renal, cardiac, metabolic) of such severity that death within 24 weeks is likely; Concurrent diagnosis of malignancy including known Myelodysplastic syndrome, leukemia, or an abnormal karyotype; Participant is on chronic transfusion therapy; Other experimental or investigational drug therapy in the past 28 days; Altered mental status or recurrent seizures requiring anti-seizure medications; New York Heart Association (NYHA) class III/IV status; Known history of illicit drug or alcohol abuse within the past 12 months; Being positive for HIV infection; Alanine Aminotransferase (ALT) ≥ 3 times the upper limit of normal or albumin <2.0 mg/dL or direct (conjugated) bilirubin ≥ 1.5 mg/dl; Serum creatinine >2.9 mg/dL and calculated creatinine clearance <30 mL/min; Platelet count >800 x 109/L; Absolute neutrophil count <1.5 x 109/L; Inability to give informed consent; Currently pregnant or breast-feeding; Female of active childbearing potential who is unwilling to use at least one of the two following forms of birth control: (i) not having heterosexual sexual contact beginning at the screening visit and continuing until 4 weeks after the last dose of decitabine OR (ii) intrauterine device (IUD); Sexually active male who is unwilling to use a condom when engaging in any sexual contact with a female with child-bearing potential, beginning at the screening visit and continuing until 4 weeks after taking the last dose of THU and decitabine. This requirement applies also to males who have had a successful vasectomy; Eastern Co-operative Oncology Group (ECOG) performance status ≥3; Taking l-glutamine within the last 28 days.