What is 5-azacitidine and decitabine studying for myelodysplastic syndromes?

This study is evaluating a combined low-dose treatment of 5-azacitidine and decitabine for myelodysplastic syndromes and related blood disorders. Researchers aim to understand how this alternating schedule affects disease response and to overcome resistance seen with each drug alone. The study is sponsored by Benjamin Tomlinson and focuses on improving treatment options for these conditions.

Who may be eligible for this myelodysplastic syndromes clinical trial?

Adults 18 years or older with diagnosed myelodysplastic syndrome or related overlap disorders may be eligible. Participants should not have had prior treatment with azacitidine or decitabine and must have adequate organ function and performance status. Both males and females can join. Please review eligibility criteria for more details.

Is there any cost to participate in this myelodysplastic syndromes clinical trial?

Study-related care, including the study drugs and all trial visits, is provided at no cost. However, personal travel and other expenses may vary and are not guaranteed to be covered. Participants should plan accordingly when considering participation.

Actively Recruiting

Early Phase 1

Age: 18Years +

All Genders

ID04187703

Proof-Of-Concept Study of Metabolically Optimized, Non-Cytotoxic 5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Led by Benjamin Tomlinson · Updated on 2026-05-18

Participants Needed

Research Sites

56 weeks

Total Duration

AI-Summary

What this Trial Is About

Myelodysplastic syndrome MDS, also called bone marrow failure, is a condition where the bone marrow produces fewer blood cells due to abnormal cell development. This study evaluates a new approach to treating MDS by using an alternating low-dose schedule of two chemotherapy drugs, 5-azacitidine 5AZA and decitabine DEC, to overcome resistance that can occur when either drug is given alone. The study is an early phase 1 pilot trial focusing on this combined treatment for myeloid malignancies including MDS and related disorders. Participants will receive 5AZA and DEC in a weekly alternating schedule 5AZA at 50 mgm on Day 1 and DEC at 5 mgm on Day 4 each week. The first 8 weeks serve as an induction phase, followed by a long-term treatment phase starting from week 9. Treatment will continue for at least 24 weeks unless the disease progresses. Those who respond to therapy may continue treatment until relapse or disease progression not responsive to dose escalation. During the trial, participants will be regularly monitored for response using criteria including complete or partial response and hematologic improvement. Safety will be assessed by tracking adverse events. The study also explores biological markers related to treatment response. Participants may remain in the study for up to 6 months after treatment to assess overall response, with some outcomes followed for up to 2 years. Careful evaluation of blood counts, disease status, and side effects will guide treatment continuation and study assessments.

CONDITIONS

Brief Title

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Who Can Participate

Age: 18Years +

All Genders

Eligibility Criteria

You may qualify if you...

Participants must have MDS or MDS/myeloproliferative overlap disorder with potential sensitivity to hypomethylating agent therapy, supported by prior evidence of response
Diagnoses include various WHO-classified subtypes of MDS and MDS/MPN crossover syndromes with limited extramedullary hematopoiesis and mild reticulin fibrosis
Indication for hypomethylating agent therapy includes symptomatic anemia, low platelet counts, transfusion dependence, or low neutrophil counts
Lower risk MDS participants must have failed or have contraindications to available effective therapies like lenalidomide or epoetin
Performance status of 60% or greater by Karnofsky Performance Status
Adequate liver function with AST and ALT less than 3 times upper limit of normal and bilirubin within 1.5 times upper limit unless due to certain conditions
Ability and willingness to provide informed consent and complete study procedures

You will not qualify if you...

Participants with high or very high risk MDS by IPSS-R or intermediate-2 or higher risk
Prior treatment with azacitidine, decitabine, or investigational hypomethylating agents with similar mechanisms
Use of other disease-directed therapy except hydroxyurea within 14 days before study
Unresolved toxicity grade 2 or higher from prior therapies
Currently pregnant or breastfeeding; females of childbearing potential must have a negative pregnancy test
Uncontrolled illnesses limiting life expectancy or study completion, including active infections without controlled treatment, uncontrolled malignancy, severe heart failure, unstable angina, unstable arrhythmias, decompensated liver cirrhosis
Psychiatric or social issues limiting compliance
Unwillingness to use dual contraception for females and males of reproductive potential
Known active HIV infection unless stable on treatment with undetectable viral load
Allergy or hypersensitivity to azacitidine or decitabine components

Research Team

Benjamin Tomlinson, MD

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