Actively Recruiting

Early Phase 1
Age: 18Years +
All Genders
NCT04187703

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Led by Benjamin Tomlinson · Updated on 2025-04-09

20

Participants Needed

1

Research Sites

315 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Another term for myelodysplastic syndrome is bone marrow failure. The bone marrow is where components of blood such as red cells, platelets and white cells are made. In bone marrow failure, the ability for bone marrow to make these cells is decreased. In myelodysplastic syndrome, this decreased bone marrow function is believed to result from abnormalities that prevent the normal maturation process by which bone marrow cells develop into red blood cells, white blood cells and platelets. In myelodysplastic syndrome, these abnormal bone marrow cells occupy space in the bone marrow and prevent the function of remaining normal bone marrow cells. One approach to treating the abnormal growth of immature cells is to give chemotherapy which damages DNA within these cells and causes their death. Unfortunately, such therapy has side-effects, since even normal cells can be affected by the treatment. Both 5-azacitidine (5AZA) and decitabine (DEC) are FDA-approved to treat MDS. In this study, 5AZA and DEC will be administered using an alternating low doses schedule in an attempt to overcome the known mechanisms of resistance to the administration of 5AZA or DEC as single agents caused by automatic adaptive shifts in DNA metabolism.

CONDITIONS

Official Title

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants must have MDS or MDS/myeloproliferative overlap disorder with potential sensitivity to hypomethylating agent (HMA) therapy based on prior response evidence
  • MDS diagnoses include WHO-classified subtypes such as refractory anemia with ring sideroblasts and thrombocytosis, CMML, and others with limited extramedullary hematopoiesis and reticulin fibrosis grade 1 or less
  • Indications for HMA therapy include symptomatic anemia, low platelet count (<100 x 10^9/L), transfusion dependence, or low absolute neutrophil count (<1.0 x 10^9/L)
  • Lower risk MDS participants must have failed or be ineligible for other effective therapies like lenalidomide or epoetin
  • Performance status of 60% or greater by Karnofsky Performance Status (KPS)
  • Adequate liver function with AST and ALT less than 3 times upper limit of normal and bilirubin ≤1.5 times upper limit of normal (with exceptions for Gilbert's disease or hemolysis)
  • Ability to understand, sign informed consent, and complete study procedures
Not Eligible

You will not qualify if you...

  • MDS with IPSS-R high or very high risk, or IPSS intermediate-2 or high risk disease
  • Prior treatment with azacitidine, decitabine, or investigational HMA therapies with overlapping mechanisms
  • Use of other disease-directed or experimental therapies (except hydroxyurea) within 14 days before study entry
  • Unresolved toxicity grade 2 or higher from prior therapies
  • Pregnant or breastfeeding women; females of childbearing potential must have negative pregnancy test within 72 hours before treatment
  • Uncontrolled illnesses including active infection without evidence of control, concurrent malignancy, severe heart failure (NYHA class III/IV), unstable angina, new or unstable arrhythmia, decompensated liver cirrhosis, psychiatric or social conditions limiting compliance
  • Unwillingness to use dual contraception if of childbearing potential or sexually active male unwilling to use condoms
  • Known active HIV infection unless well-controlled on stable antiretroviral therapy
  • Known allergy or hypersensitivity to azacitidine or decitabine components

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 1 location

1

Cleveland Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center

Cleveland, Ohio, United States, 44106

Actively Recruiting

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Research Team

B

Benjamin Tomlinson, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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