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Study of Nicotinamide with Oral Tetrahydrouridine and Decitabine for Sickle Cell Disease

This study is testing different treatments for high-risk sickle cell disease. Researchers are comparing oral nicotinamide (a form of Vitamin B3) alone, a combination of oral tetrahydrouridine (THU) and decitabine, and a combination of all three (nicotinamide, THU, and decitabine). The main goal is to see how these treatments affect your blood hemoglobin (the protein in red blood cells that carries oxygen) after 12 weeks. You may be able to join if you are 18 or older, have confirmed sickle cell disease, and are not currently experiencing an acute complication. The study aims to enroll 20 participants, but its current recruitment status is unclear.

Study design
This is a randomized control trial involving 20 participants. It compares nicotinamide alone, THU-decitabine alone, and the combination of all three treatments.
What's involved
You would receive treatment with one agent alone for 12 weeks, followed by the combination for another 12 weeks. There's an option to continue combination treatment for an additional 24 weeks.
Compensation
Not stated in the trial record.
Follow-up
Blood hemoglobin is measured at 12 weeks. Patients have the option to enter an extension phase for an additional 24 weeks of combination treatment (total of 48 weeks).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04055818

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

Recruiting
PHASE1Ages 18+InterventionalTreatment
EpiDestiny, Inc.
~20 participants
Updated 2025-05-25 on ClinicalTrials.gov
What's tested:Nicotinamide

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Blood Hemoglobin
Measured over 12 weeks

Conditions

Sickle Cell Disease

Explore related trials

Sickle Cell Disease

Where it's being run

1 sites across 1 states
Illinois1

Study leadership

  • Robert Molokie · PRINCIPAL_INVESTIGATOR · University of Illinois at Chicago College of Medicine

Who to contact

Lani Krauz
[email protected]
312-413-0242
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

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

Inability to give informed consent.
Experienced severe sepsis or septic shock within the previous 12 weeks.
Last HU dose was ingested within the previous 4 weeks.
Currently pregnant or breast-feeding.
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.
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.
Altered mental status or recurrent seizures requiring anti-seizure medications.
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.
New York Heart Association (NYHA) class III/IV status.
Eastern Co-operative Oncology Group (ECOG) performance status ≥3.
Participant is on chronic transfusion therapy
Known history of illicit drug or alcohol abuse within the past 12 months.
Other experimental or investigational drug therapy in the past 28 days.
Taking l-glutamine within the last 28 days
Being positive for HIV infection

What this trial measures

  • Blood Hemoglobin12 weeks

    Measure hemoglobin function