A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease

  • STATUS
    Recruiting
  • End date
    Mar 3, 2023
  • participants needed
    20
  • sponsor
    EpiDestiny, Inc.
Updated on 14 April 2022
hydroxyurea
narcotics
electrophoresis
acute pain
acute chest syndrome
chest syndrome

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).

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)

Details
Condition Sickle Cell Disease
Treatment Nicotinamide
Clinical Study IdentifierNCT04055818
SponsorEpiDestiny, Inc.
Last Modified on14 April 2022

Eligibility

Yes No Not Sure

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
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

0/250

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note