NCT04055818 A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
| NCT ID | NCT04055818 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | EpiDestiny, Inc. |
| Condition | Sickle Cell Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2020-01-24 |
| Primary Completion | 2028-10-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 20 participants in total. It began in 2020-01-24 with a primary completion date of 2028-10-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
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
Contact & Investigator
Robert Molokie
PRINCIPAL INVESTIGATOR
University of Illinois at Chicago College of Medicine
Frequently Asked Questions
Who can join the NCT04055818 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04055818 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04055818 currently recruiting?
Yes, NCT04055818 is actively recruiting participants. Contact the research team at LIgnacio@UIC.EDU for enrollment information.
Where is the NCT04055818 trial being conducted?
This trial is being conducted at Chicago, United States.
Who is sponsoring the NCT04055818 clinical trial?
NCT04055818 is sponsored by EpiDestiny, Inc.. The principal investigator is Robert Molokie at University of Illinois at Chicago College of Medicine. The trial plans to enroll 20 participants.
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