Sickle Cell DiseasePhase 1NCT04055818
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
A Phase 1 study testing Nicotinamide in people with Sickle Cell Disease, run by EpiDestiny, Inc. at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Nicotinamide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Jan 2020to 20 Jan 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Illinois at Chicago College of Medicine
Chicago, Illinois, 60612
Lani Krauz, 312-413-0242, Llgnacio@UIC.EDU
About this study
Official title: Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat 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).
Read the detailed description
A randomized control trial comparing oral THU-decitabine to nicotinamide (1:1 randomization), and then comparing the effects of the combination of nicotinamide with THU-decitabine vs either treatment alone. Treatment with each agent alone is for 12 weeks followed by the combination for a further 12 weeks. Patients have the option to enter an extension phase of combination treatment for an additional 24 weeks (total of 48 weeks)
- Conditions
- Sickle Cell Disease
- Being tested
- Nicotinamide
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NicotinamideDrug
Also called Decitabine, Tetrahydrouridine
Oral nicotinamide (Vitamin B3) alone compared to THU Decitabine combination
| Study group | Role | What they receive |
|---|---|---|
| Nicotinamide | Experimental | Oral Nicotinamide 1000 mg twice daily Receives: Drug: Nicotinamide |
| THU Decitabine | Experimental | Oral 250 mg THU and 5 mg decitabine Once per week Receives: Drug: Nicotinamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood Hemoglobin
Measure hemoglobin function
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2019
- Study start24 Jan 2020
- Primary completion30 Oct 2028 (estimated)
- Study completion20 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 1:1 Randomization
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- EpiDestiny, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Robert Molokie, Principal investigator, University of Illinois at Chicago College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04055818
- Sponsor's protocol ID
- 2019-0631
- Status last verified
- Sep 2026
- First submitted
- 12 Aug 2019
- Last updated
- 30 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat High Risk Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT04055818
- Acronym / sponsor ID
- 2019-0631
- Conditions
- Sickle Cell Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- EpiDestiny, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 24 Jan 2020 (actual)
- Primary completion
- 30 Oct 2028 (estimated)
- Study completion
- 20 Jan 2029 (estimated)
- First posted
- 14 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
Detailed description
A randomized control trial comparing oral THU-decitabine to nicotinamide (1:1 randomization), and then comparing the effects of the combination of nicotinamide with THU-decitabine vs either treatment alone. Treatment with each agent alone is for 12 weeks followed by the combination for a further 12 weeks. Patients have the option to enter an extension phase of combination treatment for an additional 24 weeks (total of 48 weeks)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 1:1 Randomization
| Arm | Type | Receives |
|---|---|---|
| Nicotinamide | Experimental | Drug: NicotinamideOral Nicotinamide 1000 mg twice daily |
| THU Decitabine | Experimental | Drug: NicotinamideOral 250 mg THU and 5 mg decitabine Once per week |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (20 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NicotinamideAlso: Decitabine, TetrahydrouridineOral nicotinamide (Vitamin B3) alone compared to THU Decitabine combination | Drug | Nicotinamide, THU Decitabine |
Primary outcome
Blood Hemoglobin
Time frame: 12 weeks
Measure hemoglobin function
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Robert Molokie principal investigator
University of Illinois at Chicago College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nicotinamide in people with Sickle Cell Disease, run by EpiDestiny, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lani Krauz (312-413-0242, LIgnacio@UIC.EDU), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Blood Hemoglobin (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2026.