Last reviewed · How we verify
UPdated Disease Monitoring And Treatment for Enhanced Outcomes for Pediatric AML: A Pilot Trial
This research study investigates the tolerability of substituting two cycles of chemotherapy into the standard pediatric acute myeloid leukemia (AML) chemotherapy treatment regimen for patients with newly diagnosed AML at intermediate-risk (IR) and high-risk (HR) of relapse. The goal is to achieve similar or better survival with chemotherapy cycles that are intensive but less likely to cause long-term complications. Patients will enroll on this trial at the end of their first induction cycle. The two cycles to be substituted are: * "Ida-FLA" (idarubicin+fludarabine/cytarabine) as Induction 2 * "VIA" (venetoclax+idarubicin+cytarabine) as Intensification 1 of the HR treatment regimen, and Intensification 2 of the IR treatment backbone. Researchers will evaluate side effects and outcomes for up to three years after enrollment. Participants will also have the opportunity to participate in optional research studies including patient surveys and blood and bone marrow sample testing.
Details
| Lead sponsor | Joanna Yi |
|---|---|
| Phase | EARLY_PHASE1 |
| Status | RECRUITING |
| Enrolment | 36 |
| Start date | 2025-10-22 |
| Completion | 2031-08 |
Conditions
- Acute Myeloid Leukemia
- Pediatric AML
Interventions
- Idarubicin Hydrochloride
- Fludarabine
- Cytarabine (Ara-C)
- Venetoclax
- Etoposide
- Asparaginase Erwinia Chrysanthemi (recombinant)
- Intrathecal triple
- SOC
Primary outcomes
- Tolerability rate of Ida-FLA — From Day 1 of Ida-FLA through up to 50 days after completion of Ida-FLA
Tolerability rate is the proportion of participants in the tolerability dataset who did not experience an intolerable event during Induction 2 for IR and HR patients. Number of intolerable participants will be used. Intolerability is defined by events outlined in Section 6.6 of the protocol as follow: Non-Hematological Toxicity: Any Grade 5 toxicity, Grade 4 cardiac disorder, Grade 4 infection, or other Grade 4+ non-hematologic toxicity except: * Grade 4 nausea/vomiting (\<3 days) * Grade 4 ALT/AST/GGT elevation (returns to ≤Grade 1 before next cycle) * Grade 4 electrolyte abnormalities (corrected by supplementation) * Cycle delays \>50 days indicate intolerability. Hematologic Toxicity: Intolerability includes failure to recover ANC \>500/mL and platelets \>20,000/mL (without transfusion in past 7 days) by day 50 or cycle start delay \>50 days. A cycle of Ida-FLA is 29-56 days. - Tolerability rate of VIA for IR patients — From Day 1 of VIA through up to 50 days after completion of VIA
Tolerability rate is the proportion of participants in the tolerability dataset who did not experience an intolerable event during intensification 2 for IR patients as the 4th cycle. Number of intolerable participants will be used. Intolerability is defined by events outlined in Section 6.6 of the protocol as follow: Non-Hematological Toxicity: Any Grade 5 toxicity, Grade 4 cardiac disorder, Grade 4 infection, or other Grade 4+ non-hematologic toxicity except: * Grade 4 nausea/vomiting (\<3 days) * Grade 4 ALT/AST/GGT elevation (returns to ≤Grade 1 before next cycle) * Grade 4 electrolyte abnormalities (corrected by supplementation) * Cycle delays \>50 days indicate intolerability. Hematologic Toxicity: Intolerability includes failure to recover ANC \>500/mL and platelets \>20,000/mL (without transfusion in past 7 days) by day 50 or cycle start delay \>50 days. A cycle of VIA is 29-56 days. - Tolerability rate of VIA for HR patients — From Day 1 of VIA through up to 50 days after completion of VIA
Tolerability rate is the proportion of participants in the tolerability dataset who did not experience an intolerable event during intensification 2 for HR patients as the 3rd cycle. Number of intolerable participants will be used. Intolerability is defined by events outlined in Section 6.6 of the protocol as follow: Non-Hematological Toxicity: Any Grade 5 toxicity, Grade 4 cardiac disorder, Grade 4 infection, or other Grade 4+ non-hematologic toxicity except: * Grade 4 nausea/vomiting (\<3 days) * Grade 4 ALT/AST/GGT elevation (returns to ≤Grade 1 before next cycle) * Grade 4 electrolyte abnormalities (corrected by supplementation) * Cycle delays \>50 days indicate intolerability. Hematologic Toxicity: Intolerability includes failure to recover ANC \>500/mL and platelets \>20,000/mL (without transfusion in past 7 days) by day 50 or cycle start delay \>50 days. A cycle of VIA is 29-56 days.
Countries
United States