RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

Official Title

Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat High Risk Sickle Cell Disease

Quick Facts

Study Start:2020-01-24

Study Completion:2024-04-05

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04055818

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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

Inclusion Criteria

Exclusion 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.

* 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

Contacts and Locations

Study Contact

Lani Krauz

CONTACT

312-413-0242

LIgnacio@UIC.EDU

Principal Investigator

Robert Molokie

PRINCIPAL_INVESTIGATOR

University of Illinois at Chicago College of Medicine

Study Locations (Sites)

University of Illinois at Chicago College of Medicine

Chicago, Illinois, 60612

United States

Collaborators and Investigators

Sponsor: EpiDestiny, Inc.

Robert Molokie, PRINCIPAL_INVESTIGATOR, University of Illinois at Chicago College of Medicine

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-01-24

Study Completion Date2024-04-05

Study Record Updates

Study Start Date2020-01-24

Study Completion Date2024-04-05

Terms related to this study

Additional Relevant MeSH Terms

Sickle Cell Disease