RecruitingPhase 2NCT06316960

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

A Prospective, Multicenter Clinical Study on The Safety and Efficacy of Avapritinib in The Treatment of Relapsed/Refractory Pediatric Core Binding Factor Acute Myeloid Leukemia (CBF-AML) With KIT Mutation


Sponsor

Children's Hospital of Soochow University

Enrollment

50 participants

Start Date

Mar 1, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

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

Max Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study tests a targeted drug called avapritinib in children with a type of leukemia (blood cancer) called AML that has come back or stopped responding to treatment. AML is a cancer of the blood and bone marrow, and this trial focuses on a specific type that has certain genetic changes (CBF-AML with a KIT mutation). **You may be eligible if...** - You are under 18 years old - You have been diagnosed with acute myeloid leukemia (AML) with specific genetic features (t(8;21) or inv(16) and a KIT mutation) - Your AML did not respond to standard treatment, or it came back after remission - You have no active infections - Your liver and kidney function are in an acceptable range - You are generally well enough to participate (ECOG score under 2) - Your expected survival is more than 12 weeks **You may NOT be eligible if...** - You have already received avapritinib - You are currently taking other targeted leukemia drugs (e.g., dasatinib, sorafenib, venetoclax) - You have an uncontrolled active infection - You have serious heart, liver, or kidney disease - You have another cancer requiring treatment - You are already enrolled in another interventional clinical trial Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGAvapritinib

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

DRUGAzacitidine Injection

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.

DRUGDecitabine Injection

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.

DRUGIdarubicin Hydrochloride

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.

DRUGCytarabine

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

DRUGGranulocyte Colony-Stimulating Factor

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


Locations(12)

First Affiliated Hospital Of University of Science and Technology of China

Hefei, Anhui, China

The Second Hospital of Anhui Medical University

Hefei, Anhui, China

Guangzhou Women and Children Medical Center

Guangzhou, Guangdong, China

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Kaifeng Children's Hospital

Kaifeng, Henan, China

The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, China

Third Xiangya Hospital of Central South University

Changsha, Hunan, China

XiangYa Hospital Central South University

Changsha, Hunan, China

Children's Hospital of Soochow University

Suzhou, Jiangsu, China

Xuzhou Children's Hospital

Xuzhou, Jiangsu, China

Qilu Hospital of Shandong University

Jinan, Shandong, China

Children's Hospital Of Fudan University

Shanghai, Shanghai Municipality, China

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06316960


Related Trials