Actively Recruiting
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Led by EpiDestiny, Inc. · Updated on 2025-05-25
20
Participants Needed
1
Research Sites
379 weeks
Total Duration
On this page
Sponsors
E
EpiDestiny, Inc.
Lead Sponsor
N
National Institutes of Health (NIH)
Collaborating Sponsor
AI-Summary
What this Trial Is About
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
CONDITIONS
Official Title
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Provided written informed consent before joining the study
- Confirmed diagnosis of sickle cell disease by hemoglobin electrophoresis or liquid chromatography
- In steady state without acute sickle cell complications such as hospitalization, acute pain, or acute chest syndrome within the past 14 days
- Weighs at least 40 kg
- Regularly complies with comprehensive care and previous therapy
- Has symptomatic sickle cell disease defined by any of these despite at least 6 months of hydroxyurea therapy or refusal to take hydroxyurea: fetal hemoglobin below 0.5 g/dL, or 3 or more pain episodes per year requiring narcotics, or 1 or more acute chest syndrome episodes, or hemoglobin below 9 g/dL with low reticulocyte count
You will not qualify if you...
- Unable to give informed consent
- Severe sepsis or septic shock within the last 12 weeks
- Took hydroxyurea within the past 4 weeks
- Currently pregnant or breastfeeding
- Alanine aminotransferase (ALT) 3 or more times above normal, or albumin less than 2.0 mg/dL, or direct bilirubin 1.5 mg/dL or higher
- Serum creatinine above 2.9 mg/dL or creatinine clearance under 30 mL/min
- Platelet count above 800 x 10^9/L
- Absolute neutrophil count below 1.5 x 10^9/L
- Female of childbearing potential unwilling to use specified birth control methods
- Sexually active males unwilling to use condoms during sexual contact with females of childbearing potential until 4 weeks after last dose
- Altered mental status or recurrent seizures requiring medication
- Severely ill with expected death within 24 weeks due to other diseases
- Concurrent diagnosis of malignancy including myelodysplastic syndrome, leukemia, or abnormal karyotype
- New York Heart Association class III/IV heart failure
- Eastern Cooperative Oncology Group performance status 3 or higher
- On chronic transfusion therapy
- History of illicit drug or alcohol abuse within past 12 months
- Received other experimental drugs in past 28 days
- Taken l-glutamine within last 28 days
- Positive for HIV infection
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University of Illinois at Chicago College of Medicine
Chicago, Illinois, United States, 60612
Actively Recruiting
Research Team
L
Lani Krauz
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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