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Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation

ConditionsAML, ChildhoodRelapse/RecurrenceRefractory AMLCore Binding Factor Acute Myeloid LeukemiaC-KIT Mutation
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 18
SponsorChildren's Hospital of Soochow University

About this trial

The purpose of this study is to evaluate the efficacy and safety of avapritinib in relapsed or refractory pediatric core binding factor acute myeloid leukemia with KIT mutation.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Gender unlimited;

Under 18 years;

Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).

Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11;

Disqualifiers

Have received prior treatment with avapritinib;

Receiving other targeted therapies for AML at the same time, such as dasatinib, sorafenib, gilteritinib, venetoclax, etc;

Presence of active uncontrolled infection (including bacterial, fungal, or viral infection);

Present of significant underlying organ diseases: such as myocardial infarction, chronic heart failure, decompensated liver or kidney dysfunction；

Trial design

Design model

Single group

Treatments tested in this trial

  • Avapritinib

    Drug

    50mg/m2/day for weighing bodyweight \>10kg, 1.65mg/kg/day for weighing ≤ 10kg, po, qd, d1-28.

  • Azacitidine Injection

    Drug

    75mg/m2/d for weighing \>10kg, 2.5mg/kg/d for weighing ≤ 10kg, d1-7, ivgtt, qd, more than 3 hours. Azacitidine and decitabine cannot be used simultaneously.

  • Decitabine Injection

    Drug

    20mg/m2/d for weighing \>10kg, 0.67mg/kg/d for weighing ≤ 10kg, d1-5, ivgtt, qd, more than 3 hours. Azacitidine and decitabine cannot be used simultaneously.

  • Idarubicin Hydrochloride

    Drug

    5mg/m2/day for weighing \>10kg, 0.17mg/kg/day for weighing ≤ 10kg, d 6, 8, 10 (d 8, 10, 12 for azacitidine) ivgtt, qod, more than 1 hour at 10 am.

  • Cytarabine

    Drug

    10mg/m2/day for weighing \>10kg, 0.33mg/kg/day for weighing ≤ 10kg, d6-15 （d8-17 for azacitidine ）, s.c., q12h.

  • Granulocyte Colony-Stimulating Factor

    Drug

    300ug/day for weighing \>10kg, 10ug/kg/day for weighing ≤10kg, d0-5, s.c., qd.

Treatment groups

50 Participants
are divided into 1 treatment group
Group A: Relapsed/Refractory CBF-AML with KIT mutationExperimental treatment 6 interventions

Trial outcomes

Primary outcomes

1

Composite remission rate (CRc)

Composite remission rate (CRc), including the sum of the number of patients with complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete blood count recovery (CRi), and morphologically leukemia-free (MLFS) as a percentage of the total number of patients who participated in the efficacy analysis.

Time frame
The evaluation time point is day28-day35 from the start of regimen.

Secondary outcomes

1

Overall survival

Overall survival (OS) was defined as the date from enrollment to the date of death or last follow-up for surviving patients.

Time frame
From date of enrollment until the date of the occurrence of death or last follow-up, assessed up to 60 months.
2

Progression-free survival

Progression-free survival (PFS) was defined as the date from enrollment to the date of disease progression, confirmed relapse, or death, whichever occurred first.

Time frame
From date of enrollment until the date of disease progression, confirmed relapse, or death, whichever occurred first, assessed up to 60 months.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Hospital of Soochow University

Lead sponsor

Source ClinicalTrials.gov
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