Actively Recruiting
A Pilot Study to Evaluate Feasibility of Decitabine Combined With Filgrastim After Stem Cell Transplant for Children and Young Adults With AML, MDS, and Related Myeloid Malignancies
Led by Franziska Wachter · Updated on 2026-03-19
37
Participants Needed
2
Research Sites
156 weeks
Total Duration
On this page
Sponsors
F
Franziska Wachter
Lead Sponsor
H
Harvard Clinical and Translational Science Center (Harvard Catalyst)
Collaborating Sponsor
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
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.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 6 cycles of 28 days each (approximately 6 months)
Participants receive up to 6 cycles of maintenance therapy with decitabine and filgrastim after their stem cell transplant. Each cycle lasts 28 days, with decitabine given by IV infusion on days 2 to 6 and filgrastim given by subcutaneous injection on days 1 to 6 of each cycle.
6 treatment cycles with visits corresponding to each cycle
Duration - 24 months
Participants are followed for 24 months after their stem cell transplant to monitor their health and the effectiveness of the treatment.
Follow-up visits every 6 months for 24 months
Trial Site Locations
Total: 2 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02215
Actively Recruiting
2
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
Research Team
F
Franziska Wachter, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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