Azacitidine Treatment to Prevent Leukemia in Infants with TAM (Transient Abnormal Myelopoiesis)

A Multicenter, Open-Label Study Evaluating Azacitidine for Prevention of Leukemia Development in Infants with Transient Abnormal Myelopoiesis - TAM-25

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCTs041250149

Enrollment

175

Registered

2025-12-19

Start date

2026-01-11

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transient abnormal myelopoiesis Leukemia, Transient, Down syndrome

Interventions

This is a multicenter, open-label, uncontrolled trial to evaluate whether up to four cycles of azacitidine can prevent progression to acute myeloid leukemia (AML) in infants diagnosed with transient a

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) The patient was diagnosed with transient abnormal myelopoiesis (TAM) with the presence of blasts in peripheral blood before 30 days of age. 2) Blasts remain in the peripheral blood at the time of enrollment, and a sample for FCM-MRD analysis can be submitted. 3) Written informed consent for participation in this study has been obtained from the legal representative.

Exclusion criteria

Exclusion criteria: Not particularly provided.

Design outcomes

Primary

Measure	Time frame
Cumulative incidence of ML-DS at two years after TAM diagnosis in patients with positive FCM-MRD at three months post-diagnosis.	—

Secondary

Measure	Time frame
Incidence of adverse events (AEs) associated with azacitidine therapy in patients with FCM-MRD positivity at 3 months post-diagnosis 1-year and 2-year overall survival in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients 1-year and 2-year leukemia-free survival in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients 1-year and 2-year disease-free survival in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients Cumulative incidence of leukemia at 2 years after TAM diagnosis in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients Cumulative incidence of non-leukemic death at 2 years after TAM diagnosis in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients Proportion of FCM-MRD-positive and NGS-MRD-positive patients among FCM-MRD-positive cases at: start of Cycle 1, start of Cycle 3, 4 weeks after Cycle 4, and at 12 and 24 months post-diagnosis Early mortality rate and 2-year cumulative incidence of leukemia after TAM diagnosis stratified by presence or absence of therapeutic interventions (low-dose cytarabine, exchange transfusion, corticosteroids)	—

Measure

Time frame

Incidence of adverse events (AEs) associated with azacitidine therapy in patients with FCM-MRD positivity at 3 months post-diagnosis 1-year and 2-year overall survival in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients 1-year and 2-year leukemia-free survival in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients 1-year and 2-year disease-free survival in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients Cumulative incidence of leukemia at 2 years after TAM diagnosis in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients Cumulative incidence of non-leukemic death at 2 years after TAM diagnosis in all patients, FCM-MRD-positive patients, and FCM-MRD-negative patients Proportion of FCM-MRD-positive and NGS-MRD-positive patients among FCM-MRD-positive cases at: start of Cycle 1, start of Cycle 3, 4 weeks after Cycle 4, and at 12 and 24 months post-diagnosis Early mortality rate and 2-year cumulative incidence of leukemia after TAM diagnosis stratified by presence or absence of therapeutic interventions (low-dose cytarabine, exchange transfusion, corticosteroids)

—

Contacts

Public ContactHideki Muramatsu

Nagoya University Hospital

hideki-muramatsu@med.nagoya-u.ac.jp+81-52-744-2294

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026