A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat High Risk Sickle Cell Disease
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Time periods mentioned: 12 weeks
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "Treatment with each agent alone is for 12 weeks followed by the combination for a further 12 weeks."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests nicotinamide, oral tetrahydrouridine (THU) plus decitabine, and all three drugs together. It is for 20 adults with 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).
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- You are at least 18 years old and weigh at least 40 kg (about 88 pounds).
- You sign a form agreeing to join the study before it starts.
- Blood tests confirm that you have sickle cell disease.
- Your disease is stable, with no hospital stay, sudden pain, or acute chest syndrome in the past 14 days. Acute chest syndrome is a serious lung problem linked to sickle cell disease.
- You regularly follow your care plan and past treatments.
- You still have one of the problems listed below after at least 6 months of hydroxyurea treatment, or you refuse hydroxyurea for personal reasons.
- You have at least one of these: fetal hemoglobin below 0.5 g/dL; at least 3 pain attacks a year needing strong pain medicine by injection; at least 1 episode of acute chest syndrome; or hemoglobin below 9 g/dL with a young red blood cell count below 250,000/mm³. Hemoglobin is the protein that carries oxygen in blood.
Who can't join
- You cannot give informed consent, have changes in your thinking or awareness, or have repeated seizures that need medicine.
- You had a severe infection called sepsis or septic shock in the past 12 weeks, test positive for HIV, or have a history of illegal drug or alcohol abuse in the past 12 months.
- You took hydroxyurea in the past 4 weeks, another study drug in the past 28 days, or L-glutamine in the past 28 days; or you receive regular blood transfusions.
- You are pregnant or breastfeeding. If you can become pregnant, you must use an IUD or avoid sex with men from screening until 4 weeks after the last decitabine dose. Sexually active men must use condoms with women who can become pregnant from screening until 4 weeks after the last THU and decitabine dose, even after a vasectomy.
- Your liver tests show ALT at least 3 times the normal upper limit, albumin below 2.0 mg/dL, or direct bilirubin at least 1.5 mg/dL. You also cannot join if creatinine is above 2.9 mg/dL AND creatinine clearance is below 30 mL/min; these tests measure kidney function.
- Your platelet count is above 800 × 10⁹/L, or your neutrophil count is below 1.5 × 10⁹/L. Platelets help blood clot, and neutrophils fight infection.
- You have cancer, including myelodysplastic syndrome or leukemia (blood or bone marrow cancers), or an abnormal chromosome test.
- You are near death or have another illness likely to cause death within 24 weeks, severe heart problems rated NYHA class III or IV, or major limits in daily activity rated ECOG 3 or higher.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
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
Study sites (1)
IL
- University of Illinois at Chicago College of Medicine · Chicago, IL 60612Lani Krauz · Study contact · 312-413-0242 · Llgnacio@UIC.EDUMore trials in Chicago