Study of Revumenib with Chemotherapy for Children with Relapsed or Hard-to-Treat Leukemia

Program Overview

This study is exploring whether a new drug, Revumenib, combined with chemotherapy, can help treat leukemia in infants and young children whose cancer has come back or is not responding to treatment. Leukemia is a type of cancer that affects white blood cells, and in some children, it involves a specific genetic change called KMT2A rearrangement. This change can make the cancer more likely to return after treatment. Revumenib targets this genetic change and has shown promise in lab tests. The study aims to find out if this combination treatment is safe and effective for these young patients.

Description

Primary Goals:

1. Determine the best dose of Revumenib with chemotherapy for children with relapsed or hard-to-treat leukemia.
2. Measure how many children achieve remission, meaning no detectable cancer, after treatment.

Secondary Goals:

1. Study how Revumenib is processed in the body when given with chemotherapy.
2. Evaluate how long children remain free of cancer and their overall survival after 18 months.
3. Assess how well children tolerate Revumenib when given alone.

Exploratory Goals:

1. Investigate the biological effects of Revumenib with chemotherapy.
2. Measure remission rates in older children and those with different types of leukemia.
3. Study the effects of another drug, calaspargase pegol-mknl, and its side effects.
4. Review other cancer treatments received before and after Revumenib.

Treatment Plan:

• Regimen A:

  • Cycle 1: Revumenib is given orally or through a feeding tube every 12 hours, along with chemotherapy drugs like vincristine, prednisone, and others. If the disease progresses early, patients may move to Cycle 2 sooner.
  • Cycle 2: Continues with Revumenib and different chemotherapy drugs. Patients with early disease progression may proceed to Cycle 3.
  • Cycle 3: Similar to Cycle 2, with additional chemotherapy.
  • Monotherapy: Revumenib is given alone, with additional chemotherapy as needed.

• Regimen B:

  • Cycles 1-2: Revumenib is given with chemotherapy, repeating every 28 days for 2 cycles.
  • Monotherapy: Revumenib is given alone, repeating every 28 days for up to 12 cycles, with additional chemotherapy as needed.

All patients will undergo various tests, including heart scans, blood and spinal fluid tests, and bone marrow exams throughout the study.

Eligibility Criteria