Treatment for high-risk sickle cell disease using nicotinamide and other medications

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial is a randomized control study involving 20 subjects with sickle cell disease. It compares the effects of oral tetrahydrouridine (THU) combined with decitabine to nicotinamide, as well as the combination of all three treatments. Each treatment will be administered for 12 weeks, followed by an additional 12 weeks of combination therapy, with an option for participants to extend the combination treatment for another 24 weeks. The aim is to evaluate the efficacy and safety of these treatments in managing high-risk sickle cell disease.

Why it matters

Who can join: the full rules

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 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 Criteria:

* 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

Where it's running

• University of Illinois at Chicago College of Medicine
  Chicago, Illinois, United States Recruiting

Study contacts

• Principal investigator: Robert Molokie — University of Illinois at Chicago College of Medicine
• Study coordinator: Lani Krauz
• Email: LIgnacio@UIC.EDU
• Phone: 312-413-0242

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.