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

Recruiting now · Phase 2

Conditions studied: Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloid Malignancies, MDS, Inherited Bone Marrow Failure Syndrome, Myeloid Neoplasm, Aml

In brief

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 facts

Study ID

NCT05796570

Run by

Franziska Wachter

People needed

37

Starts

2023-04-19

Expected to finish

2029-09-01

Last updated by the study team

2026-03-19

Who can join

Age: 1 and older, up to 39. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Disease Criteria: Participants must have a histologically confirmed diagnosis of one of the following hematologic malignancies for eligibility, as defined by the criteria below:
• AML (relapsed, de-novo or secondary) based on WHO classification
• MDS (relapsed, de-novo or secondary) based on WHO classification
• Treatment myeloid neoplasm (tMDS/AML; relapsed disease included)
• Myeloid Sarcoma
• Acute Undifferentiated Leukemia (MPAL and acute leukemia of ambiguous lineage/NOS not eligible)
• Note: MDS, AML, MDS/AML, or tMDS/AML as defined above may be idiopathic/de novo or derived from a germline predisposition to myeloid malignancy. For patients with an underlying germline disorder, those conditions that are not associated with increased risk for toxicity to treatment, including patients with known germline ANKRD26, DDX41, ELANE and other congenital neutropenia disorders, ETV6, GATA-2, Li-Fraumeni, RUNX1, SAMD9/SAMD9L, or Shwachman-Diamond Syndrome, will be analyzed within the general treatment cohort (Cohort A, see Table 1) along with patients with idiopathic disease (Cohort B, see Table 2).
• MDS, AML, MDS/AML, or tMDS/AML derived from the following germline disorders will be enrolled in a separate cohort (B) and adverse events monitored closely for higher rates compared to cohort A:
• Dyskeratosis Congenita or associated telomeropathies as defined by telomere length <1st percentile on 3 out of 4 lymphocyte subsets and/or corresponding pathogenic genetic mutation.
• Fanconi Anemia as defined by positive chromosomal breakage test to DEB/MMC and/or corresponding pathogenic genetic mutation.
• Nijmegen Breakage Syndrome as defined by positive chromosomal breakage test to DEB/MMC and/or corresponding pathogenic genetic mutation
• ERCC6L2 by genomic testing.
• Table 2: Overview Inherited Bone Marrow Failure syndromes (iBMF)
• iBMF with Standard risk for Treatment Related toxicities:
• germline mutations in ANKRD26
• germline mutations in DDX41
• ELANE and other Congenital Neutropenia Disorders
• germline mutations in ETV6
• germline mutations in GATA-2
• Li-Fraumeni
• germline mutations in RUNX1
• SAMD9/SAMD9L
• Shwachman-Diamond Syndrome
• Familial MDS with thrombocytopenia
• Diamond-Blackfan Anemia

You may not qualify if…

• Participants who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities > Grade 2) except for bone marrow suppression.
• Participants should not be enrolled on another study that prohibits initiation of maintenance therapy.
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to decitabine or filgrastim.
• Participants with uncontrolled intercurrent illness.
• Participant who are not able to present for clinic visits for at least 7 months after study treatment initiation.
• Participant with FLT3/ITD mutations are excluded as maintenance therapy with tyrosine kinase therapy should be considered in this context. However, if a participant has a co-occurring NUP28 mutation, they will be considered eligible.
• Participants with a concurrent active malignancy are not eligible for this trial.

Where it is running

• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.