Actively Recruiting
Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat High Risk Sickle Cell Disease
Led by EpiDestiny, Inc. · Updated on 2025-05-25
20
Participants Needed
1
Research Sites
26 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
Researchers are conducting a phase 1 randomized controlled trial to evaluate treatments for high risk sickle cell disease. The study compares oral THU-decitabine to nicotinamide alone and then assesses the combination of nicotinamide with THU-decitabine. This trial aims to better understand the effects of these treatments on patients who continue to experience symptoms despite prior therapy. Participants receive either oral nicotinamide 1000 mg twice daily or oral THU 250 mg with decitabine 5 mg once per week for 12 weeks. After this period, all participants receive the combination treatment for another 12 weeks. Those who wish can join an extension phase of combination therapy lasting an additional 24 weeks, making a total treatment period of up to 48 weeks. Throughout the study, participants undergo assessments including blood tests to measure hemoglobin levels at 12 weeks. Researchers monitor the effects of the treatments on sickle cell disease symptoms and safety. The study involves regular visits to track treatment response and side effects, with a focus on the participants' overall well-being during and after the treatment periods.
CONDITIONS
Brief 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
- Written informed consent provided before study entry
- Confirmed sickle cell disease by hemoglobin electrophoresis or liquid chromatography
- Participant is in steady state with no acute sickle cell complications in past 14 days
- Weight at least 40 kg
- Regular compliance with comprehensive care and previous therapy
- Symptomatic sickle cell disease despite at least 6 months of hydroxyurea therapy or refusal to take hydroxyurea, defined by fetal hemoglobin <0.5 g/dL, or 3 or more pain episodes per year needing narcotics, or 1 or more acute chest syndrome episodes, or hemoglobin <9 g/dL and reticulocyte count <250,000/mm3
You will not qualify if you...
- Inability to give informed consent
- Severe sepsis or septic shock within previous 12 weeks
- Hydroxyurea dose taken within past 4 weeks
- Currently pregnant or breastfeeding
- Alanine Aminotransferase (ALT) ≥ 3 times upper limit of normal, or albumin <2.0 mg/dL, or direct bilirubin ≥ 1.5 mg/dL
- Serum creatinine >2.9 mg/dL or creatinine clearance <30 mL/min
- Platelet count >800 x 10^9/L
- Absolute neutrophil count <1.5 x 10^9/L
- Female of childbearing potential unwilling to use specified birth control methods
- Sexually active male unwilling to use condom during study and 4 weeks after last dose
- Altered mental status or recurrent seizures requiring anti-seizure medications
- Moribund or severe concurrent disease likely to cause death within 24 weeks
- Concurrent malignancy including myelodysplastic syndrome, leukemia, or abnormal karyotype
- New York Heart Association class III/IV status
- Eastern Cooperative Oncology Group performance status ≥3
- On chronic transfusion therapy
- History of illicit drug or alcohol abuse within past 12 months
- Use of other experimental drug therapies within past 28 days
- Use of l-glutamine within past 28 days
- Positive for HIV infection
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 24 weeks
Participants receive oral nicotinamide or oral THU-decitabine alone for 12 weeks, followed by a combination of nicotinamide with THU-decitabine for an additional 12 weeks.
Duration - 24 weeks
Participants may choose to continue combination treatment with nicotinamide and THU-decitabine for an additional 24 weeks.
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
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here