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Looking back at menin inhibitor treatment in children with relapsed or refractory acute leukemia

Reviews past records of children with acute leukemia treated with menin inhibitors.

Plain-language title generated from the registry record
Registry title: Menin Inhibitors in Pediatric Relapsed or Refractory Acute Leukemia

Key facts

Where
Strasbourg, FranceSee the site

Could this be for you?

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 0 and 17 years old?

Source: NCT07898436, who can take part

Where in France

  • Service de Pédiatrie 3 - Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg - FranceStrasbourg, FranceTaking people

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?
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Trial details

Who runs it
University Hospital, Strasbourg, France
Trial code
NCT07898436
Record updated
27 October 2026
Original description
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.Read full original descriptionShow less

Management of Relapsed or Refractory Acute Leukemia With Menin Inhibitors in Children: A French National Retrospective Study

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.

Inclusion: Patients with relapsed or refractory acute leukemia eligible for menin inhibitor treatment because of a KMT2A or NUP98 rearrangement or an NPM1 mutation; Patients who received menin inhibitor treatment under a Temporary Authorization for Use (ATU); Patients younger than 18 years at the time of acute leukemia diagnosis; Patients who have not objected to the reuse of their data for scientific research; Patients whose holders of parental authority have already provided authorization for the reuse of the child's data for scientific research.

Exclusion: Patients who received a Temporary Authorization for Use (ATU) but did not receive the treatment.

ClinicalTrials.gov NCT07898436