What is decitabine studying for children and young adults with AML and related myeloid malignancies?

Decitabine is being studied to see if it is feasible and effective when given with filgrastim after stem cell transplant to prevent cancer relapse. Researchers want to learn if this combination can help maintain remission in young patients with acute myeloid leukemia, myelodysplastic syndrome, and related conditions. The study is led by Dr. Franziska Wachter.

Who may be eligible for this AML and MDS clinical trial?

Children and young adults aged 1 to 39 years with confirmed AML, MDS, or related myeloid malignancies who have had an allogeneic stem cell transplant may be eligible. Participants must be in remission with no minimal residual disease before treatment and able to attend follow-up visits. Both males and females can join. Please review eligibility criteria for more details.

Is there any cost to participate in this AML and MDS clinical trial?

Study-related care, including the study drugs decitabine and filgrastim and all trial visits, is provided at no cost. Personal travel expenses and other costs may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 2

Age: 1Year - 39Years

All Genders

ID05796570

Pilot Study of Decitabine and Filgrastim Treatment After Stem Cell Transplant for Children and Young Adults with AML, MDS, and Related Myeloid Cancers

Led by Franziska Wachter · Updated on 2026-03-19

Participants Needed

Research Sites

156 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating whether giving decitabine and filgrastim after allogeneic hematopoietic stem cell transplant HCT is feasible and effective in preventing relapse in children and young adults with acute myeloid leukemia AML, myelodysplastic syndrome MDS, and related myeloid malignancies. This is a phase 2 single-arm pilot study focusing on patients with idiopathic or inherited bone marrow failure syndromes who may have different risks of treatment toxicity. The study is supported by Dana-Farber Cancer Institute philanthropy and institutional grants. Participants will receive cycles of decitabine a nucleoside metabolic inhibitor given by IV infusion and filgrastim a granulocyte colony-stimulating factor given by subcutaneous injection starting between 40 and 120 days after HCT. Treatment cycles last 28 days, with decitabine given on days 2 to 6 and filgrastim on days 1 to 6. Up to six cycles will be given if tolerated. There are two cohorts one with standard risk and one with increased risk for treatment-related toxicities based on inherited bone marrow failure syndromes. After treatment, participants will have follow-up visits every 6 months for 24 months post-HCT. During the study, participants will undergo screening, treatment, and regular follow-up including blood tests and bone marrow biopsies as standard care. Researchers will monitor treatment feasibility by measuring how many complete the treatment cycles, as well as event-free and overall survival up to 24 months. Treatment tolerability will also be assessed. The total participation includes treatment for 6 months plus 24 months of follow-up after transplant to evaluate outcomes and safety.

CONDITIONS

Brief Title

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

Who Can Participate

Age: 1Year - 39Years

All Genders

Eligibility Criteria

You may qualify if you...

Participants must be aged 1 to 39 years.
Must have a confirmed diagnosis of AML, MDS, treatment-related myeloid neoplasm, myeloid sarcoma, or acute undifferentiated leukemia.
Must have received an allogeneic hematopoietic stem cell transplant with any donor type or conditioning regimen.
Must be in minimal residual disease negative remission before starting treatment.
ECOG performance status of 2 or better (Lansky or Karnofsky score ≥60%).
Adequate organ function for transplant as per institutional standards.
For HIV, HBV, and HCV infections, must have undetectable viral loads with effective treatment.
Must agree to use contraception if of child-bearing potential during and after the study.
Ability to understand and sign informed consent.

You will not qualify if you...

Participants with unresolved severe side effects (greater than Grade 2) from previous cancer treatments, except bone marrow suppression.
Participants enrolled in other studies that prevent starting maintenance therapy.
History of allergic reactions to decitabine, filgrastim, or similar compounds.
Uncontrolled illnesses.
Unable to attend clinic visits for at least 7 months after treatment starts.
Presence of FLT3/ITD mutations unless co-occurring with NUP28 mutation.
Having an active concurrent malignancy.

Research Team

Franziska Wachter, MD

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Pilot Study of Decitabine and Filgrastim Treatment After Stem Cell Transplant for Children and Young Adults with AML, MDS, and Related Myeloid Cancers

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria