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

Conditions

• Acute Myeloid Leukemia
• Aml
• Inherited Bone Marrow Failure Syndrome
• MDS
• Myelodysplastic Syndromes
• Myeloid Malignancies
• Myeloid Neoplasm

Eligibility Criteria

Sex

ALL

Age

1 Year - 39 Years

Healthy Volunteers

Not accepted

Interventions

• Decitabine — DRUG
  Nucleoside metabolic inhibitor, via IV infusion.
• Filgrastim — DRUG
  Recombinant granulocyte colony-stimulating factor (G-CSF), via subcutaneous injection.

Study Details

The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with myelodysplastic syndrome, acute myeloid leukemia and related myeloid disorders, and if the treatment is effective in preventing relapse after HCT. The names of the study drugs involved in this study are: * Decitabine (a nucleoside metabolic inhibitor) * Filgrastim (a recombinant granulocyte colony-stimulating factor (G-CSF)

Key Dates

Start date

Apr 19, 2023

Status verified

Mar 2026

Primary completion

Sep 1, 2026

Completion

Sep 1, 2029

Study Design

Enrollment

37 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort A: Standard Risk
  Participants with MDS, AML, AML/MDS, treatment related myeloid neoplasm (tAML/MDS) with either idiopathic disease or inherited bone marrow failure syndrome (iBMF) with standard risk for treatment related toxicities will be enrolled and will undergo study procedures as outlined: * Cycles 1: Study treatment start must occur 40 - 120 days post allogenic HCT. * Day 2 - 6 of 28-day cycle: Predetermined dose of Decitabine. * Day 1 - 6 of 28-day cycle: Predetermined dose of Filgrastim. * Cycles 2 - 6: * Day 2 - 6 of 28-day cycle: Predetermined dose of Decitabine. * Day 1 - 6 of 28-day cycle: Predetermined dose of Filgrastim. * Follow up visit every 6 months for 24 months post allogenic HCT.
• Experimental: Cohort B: inherited bone marrow failure (iBMF) with Increased Risk for treatment related toxicities
  Participants with MDS, AML, AML/MDS, tAML/MDS with iBMF with increased risk for treatment related toxicities will be enrolled and will undergo study procedures as outlined: * Cycles 1: Study treatment must occur 40 - 120 days post allogenic HCT. * Day 2 - 6 of 28-day cycle: Predetermined dose of Decitabine. * Day 1 - 6 of 28-day cycle: Predetermined dose of Filgrastim. * Cycles 2 - 6: * Day 2 - 6 of 28-day cycle: Predetermined dose of Decitabine. * Day 1 - 6 of 28-day cycle: Predetermined dose of Filgrastim. * Follow up visit every 6 months for 24 months post allogenic HCT.

Primary Outcome Measure

Feasibility Failure Rate (FFR) [ Time Frame: Treatment duration up to 6 cycles (28 days/cycle) or 168 days ]

Central Contacts

• Franziska Wachter, MD
  617-632-4583
  [email protected]

Locations (2)

Find similar trials in Boston, MA

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