Phase 2 Trial of Revumenib Plus FLA Chemotherapy for Children With Relapsed or Refractory NUP98-rearranged AML."

Sponsor
Therapeutic Advances in Childhood Leukemia Consortium
Study ID
NCT07686107
Phase
PHASE2
Status
Not Yet Recruiting

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Conditions

  • Leukemia, Myeloid, Acute

Eligibility Criteria

Sex
ALL
Age
30 Days - 22 Years
Healthy Volunteers
Not accepted

Interventions

  • Revumenib — DRUG
    Patients under 40 kg, and those unable to swallow pills, must receive oral suspension (40 mg/mL): * \< 40 kg: 95 mg/m2 (max 160 mg/dose) orally twice daily * ≥ 40 kg (flat-dose): 160 mg orally twice daily * Round to the nearest markings on the syringe, within 10% of the calculated dose Patients ≥ 40 kg that are able to swallow pills: \- 160 mg orally twice daily
  • Fludarabine — DRUG
    * 5 mg for patients with BSA 0.25-0.29 * 6.25 mg for patients with BSA 0.3-0.34 * 8.75 mg for patients with BSA 0.35-0.39 * 10 mg for patients with BSA 0.4-0.44 * 12.5 mg for patients with BSA 0.45-0.49 * 15 mg for patients with BSA 0.5-0.54 * 16.25 mg for patients with BSA 0.55-0.59 * 30 mg/m2/dose for patients with BSA \> 0.6
  • Cytarabine (Ara-C) — DRUG
    * 300 mg for patients with BSA 0.25-0.29 * 420 mg for patients with BSA 0.3-0.34 * 560 mg for patients with BSA 0.35-0.39 * 680 mg for patients with BSA 0.4-0.44 * 820 mg for patients with BSA 0.45-0.49 * 960 mg for patients with BSA 0.5-0.54 * 1100 mg for patients with BSA 0.55-0.59 * 2000 mg/m2/dose for patients with BSA \> 0.6
  • Methotrexate (IT) — DRUG
    Given intrathecally * 6 mg for patients \> 30 days - \< 1 year * 8 mg for patients \> 1 - \< 2 years * 10 mg for patients \> 2 - \< 3 years * 12 mg for patients \> 3 years
  • Hydrocortisone (intrathecal) — DRUG
    Given intrathecally * 12 mg for patients \> 30 days - \< 1 year * 16 mg for patients \> 1 - \< 2 years * 20 mg for patients \> 2 - \< 3 years * 24 mg for patients \> 3 years
  • Cytarabine (intrathecal) — DRUG
    Given intrathecally * 18 mg for patients \> 30 days - \< 1 year * 24 mg for patients \> 1 - \< 2 years * 30 mg for patients \> 2 - \< 3 years * 36 mg for patients \> 3 years

Study Details

The goal of this clinical trial is to learn if Revumenib in combination with Fludarabine and Cytarabine can treat children with NUP98-rearranged relapsed or refractory Acute Myeloid Leukemia. The main question it aims to answer is: What is the Best Overall Response Rate by morphologic response criteria after up to two cycles of revumenib in combination with fludarabine and cytarabine (FLA) chemotherapy? Participants will receive up to two cycles of Revumenib in combination with fludarabine and cytarabine with intrathecal triple therapy. Participant may also receive a stem cell transplant followed by Revumenib monotherapy if it is necessary. Up to 27 children and young adults will be enrolled.

Key Dates

First listed
Jul 7, 2026
Start date
Aug 31, 2026
Status verified
Jul 2026
Primary completion
Dec 31, 2029
Completion
Dec 31, 2030

Study Design

Enrollment
24 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1
    Revumenib -Days 1-28 Fludarabine -Days 1-5 Cytarabine -Days 1-5 IT Therapy Triples (Methotrexate, Hydrocortisone, Cytarabine) -Day 0. May receive with marrow done at the end of the previous course. For CNS2 and CNS3 patients, in Cycle 1 continue weekly for 3 to 6 total doses or until 3 negative CSF samples, whichever comes first.

Primary Outcome Measure

Best Overall Response Rate (BOR) by morphologic response criteria [ Time Frame: At the end of Cycle 1 and Cycle 2 (each cycle is 28 days) ]

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