Pilot Study of Decitabine and Filgrastim After Stem Cell Transplant for Blood Cancers
This study is looking at whether it's practical and effective to give two medicines, Decitabine and Filgrastim, to children and young adults (ages 1 to 39) after they've had a stem cell transplant. These patients have certain blood cancers like acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or related myeloid malignancies. Decitabine is a drug that works by interfering with cell growth, and Filgrastim helps the body make more white blood cells. The main goal is to see if giving these drugs after transplant can help prevent the cancer from coming back. About 37 people are expected to join this study. The current recruitment status is unclear.
- Study design
- This is a single-arm pilot study, meaning all participants will receive the same treatment. Approximately 37 participants are planned for this study.
- What's involved
- You would undergo screening for eligibility, receive study treatment for up to 6 months if tolerated, and have evaluations, follow-up visits, and blood tests. Bone marrow biopsies and aspirates will also be performed.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 24 months after their stem cell transplant.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05796570
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
Active, Not Recruiting
PHASE2Ages 1–39InterventionalTreatmentFranziska WachterInvestigator-initiated
~29 participants
Updated 2026-08-20 on ClinicalTrials.gov
What's tested:DecitabineFilgrastim
At a glance
Recruiting sites
0 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Feasibility Failure Rate (FFR)
Measured over Treatment duration up to 6 cycles (28 days/cycle) or 168 days
Conditions
Where it's being run
2 sites across 1 statesMassachusetts2
Study leadership
- Franziska Wachter, MD · PRINCIPAL_INVESTIGATOR · Dana-Farber Cancer Institute
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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).
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.
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
iBMF with Increased Risk for Treatment Related Toxicities:
Fanconi Anemia
Dyskeratosis Congenita and associated Telomere Disorders
Nijmegen Breakage Syndrome
ERCC6L2
Patients must be receiving an allogeneic hematopoietic stem cell transplant. All donor types and graft sources are permitted. All conditioning regimens are permitted. All GVHD prophylaxis regimens are permitted.
Timing of Enrollment: Registration can occur from day - 40 to day - 1 prior to stem cell infusion.
Disease Status: Study enrollment will occur pre HCT. Any disease status is acceptable at the time of enrollment; however, patients must be in a MRD negative remission (as defined by multidimensional flow cytometry (MDF) post HCT prior to protocol treatment start). Post HCT/ pretreatment disease status will be performed by Hematologics.
No limitations on prior therapy.
Age ≥1 year and ≤ 39 year of age.
ECOG performance status ≤2 (Lansky, Karnofsky ≥60%).
Participants must have adequate organ function to be eligible for allogenic HCT as per institutional standard.
Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial. Anti- retroviral therapy must not have a non-acceptable drug interaction with protocol treatment.
For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated. Suppressive therapy must not have a non-acceptable drug interaction with protocol treatment.
Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load. Ongoing antiviral therapy must not have a non-acceptable drug interaction with protocol treatment.
Participants with a malignancy in remission are eligible for this trial.
Participants with known history or current symptoms of cardiac disease should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, participants should be class 2B or better.
The effects of filgrastim on the developing human fetus are unknown. For this reason and because decitabine is known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of decitabine administration.
Ability to understand and the willingness to sign a written informed consent document.
Exclusion
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.
What this trial measures
- Feasibility Failure Rate (FFR)Treatment duration up to 6 cycles (28 days/cycle) or 168 days
Feasibility failure relates to decitabine exposure in the setting of post-hematopoietic stem cell transplant (HCT) maintenance and in combination with filgrastim; FFR is defined as the proportion of participants that do not initiate at least 5 of 6 planned cycles and/or receive fewer than 22/30 overall planned decitabine doses during maintenance phase.