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New treatment option for Acute Myeloid Leukemia
Official 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
ClinicalTrials.gov ID: NCT05796570
What this study is testing
What is Decitabine?
Decitabine is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for acute myeloid leukemia.
Also referred to as Dacogen.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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)
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 39
You may be able to join if
- Disease Criteria: Participants must have a histologically confirmed diagnosis of one of the following hematologic malignancies for eligibility, as...
- 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
You likely can't join 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...
- 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...
See the full eligibility criteria
- 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...
- 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 Table 2: Overview Inherited Bone Marrow Failure syndromes (iBMF)
- 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)...
- 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...
- 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...
- 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...
- 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...
- 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...
- Ability to understand and the willingness to sign a written informed consent document.
- 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...
- Participants with a concurrent active malignancy are not eligible for this trial.
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 39 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.