Menin Inhibitors in Pediatric Relapsed or Refractory Acute Leukemia

Condition(s) studied

Study summary

Acute leukemia is the most common childhood cancer. Despite advances in targeted therapies and immunotherapies, treatment remains challenging for children with relapsed or refractory acute leukemia, particularly those with KMT2A rearrangements, NPM1 mutations, or NUP98 rearrangements. Menin inhibitors have shown promising antileukemic activity in these molecular subgroups.

This French national, multicenter, retrospective observational study will describe the pediatric population treated with menin inhibitors for relapsed or refractory acute leukemia. Clinical and biological data will be collected from the SACHA database for patients treated between February 1, 2020, and July 31, 2026. Approximately 10 patients are expected to be included. The study will describe patient and disease characteristics, treatment administration, safety, and efficacy, including remission status, minimal residual disease, relapse, and overall survival.

Eligibility

Primary outcome measure(s)

• Clinical and biological characteristics of pediatric patients treated with menin inhibitors for relapsed or refractory acute leukemia — From February 1, 2020, to July 31, 2026.
  Descriptive assessment of the patient population, including clinical symptoms and biological characteristics at diagnosis, time to refractory disease or relapse, previous treatment regimens, and minimal residual disease monitoring during leukemia management.

Trial sites (1)

More University Hospital, Strasbourg, France trials in France

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