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New treatment option for Acute Myeloid Leukemia Post Cytotoxic Therapy
Official title A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia
ClinicalTrials.gov ID: NCT07012044
What this study is testing
What is Decitabine?
Decitabine is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for acute myeloid leukemia post cytotoxic therapy.
Also referred to as 5-Aza-2'-deoxycytidine, 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
- This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that does not respond to treatment (refractory) who have undergone allogenic hematopoietic stem cell transplantation. ASTX727 is a combination of cedazuridine and decitabine.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 21
You may be able to join if
- STEP 0: Patient must be ≤ 21 years of age
- PLEASE NOTE: Eligibility criteria to enroll onto Step 1 for the treatment trial is ≤ 21 years of age at the time of Step 1 enrollment. Please plan...
- STEP 0: Patients with newly diagnosed high risk\ de novo AML, newly diagnosed therapy-related AML, relapsed, or refractory AML. in complete remission...
- Based on risk criteria adopted by the AAML1831 study
- STEP 0: Patient must plan to have bone marrow sample submitted to Hematologics within 14 days prior to the start of conditioning regimen for HCT.
You likely can't join if
- STEP 0: Patients with known inherited marrow failure syndromes, including, but not limited to Fanconi Anemia, Dyskeratosis congenita, and...
- STEP 0: Known or suspected hypersensitivity to filgrastim, decitabine or cedazuridine (ASTX727)
- STEP 0: Patients who have received a prior solid organ transplantation are not eligible
- STEP 1: ASTX727 can cause fetal harm when administered to pregnant women. Pregnancy tests must be obtained in girls who are post-menarchal. Males or...
- STEP 1: Patients who are currently receiving another investigational drug are not eligible
- STEP 1: Patients who are currently receiving other anti-cancer agents are not eligible. Patients receiving intrathecal chemotherapy in the prior 14...
See the full eligibility criteria
Who can join
- STEP 0: Patient must be ≤ 21 years of age
- PLEASE NOTE: Eligibility criteria to enroll onto Step 1 for the treatment trial is ≤ 21 years of age at the time of Step 1 enrollment. Please plan accordingly to ensure that patients who are screened with Step 0 will be...
- STEP 0: Patients with newly diagnosed high risk\ de novo AML, newly diagnosed therapy-related AML, relapsed, or refractory AML. in complete remission at the time of transplant. Patients with a history of isolated or...
- Based on risk criteria adopted by the AAML1831 study
- STEP 0: Patient must plan to have bone marrow sample submitted to Hematologics within 14 days prior to the start of conditioning regimen for HCT.
- Note: In order to be eligible to enroll onto Step 1 to receive treatment on this study, pre-HCT disease status must be assessed prior to receiving HCT. AML must be in complete remission (Children's Oncology Group...
- STEP 0: Human immunodeficiency virus (HIV)-infected patients are eligible for this trial if the following criteria are met:
- No history of HIV complications with the exception of CD4 count \< 200cells/mm\^3
- No antiretroviral therapy with overlapping toxicity such as myelosuppression
- CD4 count \> 500 cells/mm\^3 prior to the diagnosis of newly diagnosed, relapsed, refractory AML.
- HIV viral loads below the limit of detection within 6 months, as long as the patient is NOT receiving anti-retroviral agents that may interact with ASTX727.
- No history of highly active antiretroviral therapy (HAART)-resistant HIV
- STEP 0: Patients must be receiving an allogeneic (related, unrelated, and mismatched related, including haploidentical) marrow, peripheral blood, or cord blood transplant for the first time
- STEP 0: HCT conditioning regimen must be planned to begin within 14 days after bone marrow assessment to determine disease status
- STEP 0: Conditioning regimen must be myeloablative and include high dose busulfan, or treosulfan, or total body irradiation
- STEP 0: Patients must not have received prior exposure to ASTX727. Note that patients may have had prior exposure to decitabine
- STEP 1: Patient must be ≤ 21 years of age at the time of study enrollment to step 0 and step 1
- STEP 1: Patients must have a body surface area ≥ 1m\^2 at enrollment to step 1
- STEP 1 (PRE-HCT): AML must be in complete remission (COG-CR, COG-CRp, COG-Cri), with or without detectable MRD; bone marrow CR must be assessed by central flow cytometry performed at Hematologics. Disease assessment...
- Patients with CNS or extramedullary disease within 14 days prior to the start of the HCT condition regimen are not eligible
- STEP 1 (POST-HCT): AML must be in complete remission (COG-CR, COG-CRp, COG-CRi). Bone marrow evaluation must show CR with no detectable minimal residual disease (MRD negative by central flow cytometry at Hematologics)
- Note: Disease assessment is required to be performed within 14 days prior to enrollment onto Step 1
- Patients with CNS or extramedullary disease post-HCT are not eligible
- STEP 1: Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients ≤ 16 years of age...
- STEP 1: Patients must have fully recovered from the acute toxicities related to the conditioning regimen and the transplant. If, after 42-100 days post-transplant, the eligibility criteria are met, the patient is...
- STEP 1: Platelet count ≥ 50,000 µL (without requirement for platelet transfusion within the last 7 days)
- STEP 1: Hemoglobin ≥ 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions)
- STEP 1: Absolute neutrophil count ≥ 1,000 µL with no myeloid growth factor support within the last 3 days
- Estimated glomerular filtration rate (GFR) (eGFR) ≥ 60 mL/min/1.73 m\^2 "Bedside" Schwartz formula (2009) OR
- OR- A 24 hour urine creatinine clearance ≥ 60 mL/min/1.73 m\^2
- A GFR ≥ 60 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
- Note: Estimated GFR (eGFR) from cystatin C or other estimates not listed above are not acceptable for determining eligibility
- STEP 1: Bilirubin (total or sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age
- STEP 1: Alanine aminotransferase (ALT) ≤ 3 x ULN
- STEP 1: Aspartate aminotransferase (AST) ≤ 3 x ULN
- STEP 1: Albumin ≥ 2 g/dL
What rules you out
- STEP 0: Patients with known inherited marrow failure syndromes, including, but not limited to Fanconi Anemia, Dyskeratosis congenita, and Shwachman-Diamond syndrome
- STEP 0: Known or suspected hypersensitivity to filgrastim, decitabine or cedazuridine (ASTX727)
- STEP 0: Patients who have received a prior solid organ transplantation are not eligible
- STEP 1: ASTX727 can cause fetal harm when administered to pregnant women. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have...
- STEP 1: Patients who are currently receiving another investigational drug are not eligible
- STEP 1: Patients who are currently receiving other anti-cancer agents are not eligible. Patients receiving intrathecal chemotherapy in the prior 14 days are eligible
- STEP 1: Patients receiving or for whom there is a plan to administer anticancer non-protocol therapy, radiation therapy or immunotherapy during the study period are not eligible (note that prophylactic use of...
- STEP 1: Drugs known to be metabolized by cytidine deaminase (CDA) should not be given (such drugs include cytarabine, gemcitabine, azacitidine, vidarabine, zalcitabine, zidovudine, telbivudine, didanosine, stavudine...
- STEP 1: Patients is not able to swallow intact tablets. Nasogastric or G tube administration is not allowed
- STEP 1: Patient is not able to start ASTX727 and filgrastim (rh-GCSF) between day 42 and day 100 following completion of allo-HCT. If, after enrollment, protocol therapy is started more than 100 days following allo-HCT...
- STEP 1: Patients with graft loss are not eligible
- STEP 1: Patients with steroid refractory or dependent acute GVHD are not eligible. Patients must be on \< 1 mg/kg/day of methylprednisolone (or equivalent prednisone/prednisolone dose) that is being tapered. Patients...
- STEP 1: Patients who have an uncontrolled viral, bacterial, fungal, or protozoal infection are not eligible
- STEP 1: Patients with active transplant associated thrombotic microangiopathy with ongoing hemolysis and need treatment (e.g. eculizumab) are not eligible
- STEP 1: Patients with sinusoidal obstruction syndrome with ongoing need for treatment (e.g. defibrotide, diuresis, supplemental oxygen) are not eligible
- STEP 1: Idiopathic pneumonitis syndrome or other non-infectious lung injury requiring ongoing treatment (corticosteroids, tumor necrosis factor [TNF] inhibitors, or other biologics) or supplemental oxygen are not...
- STEP 1: Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Indianapolis, Indiana, United States
- Ann Arbor, Michigan, United States
- Minneapolis, Minnesota, United States
- New York, New York, United States
- Philadelphia, Pennsylvania, United States
- Pittsburgh, Pennsylvania, United States
- Memphis, Tennessee, 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, up to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Indianapolis, Indiana, United States; Ann Arbor, Michigan, United States; Minneapolis, Minnesota, United States; New York, New York, United States; Philadelphia, Pennsylvania, United States and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
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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.