Induction therapy options for children and adolescents with acute myeloid leukemia

A Multicenter Clinical Study of Molecular Subtyping Combined With MRD-driven Remission Induction Regimen in Children and Adolescents With AML: A Phase II Cohort Study (GMCAII)

This study is testing different treatment plans for kids and teens with acute myeloid leukemia to see which ones help them get better and stay healthy longer.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 500 (estimated)
• Ages: N/A to 18 Years
• Sex: All
• Sponsor: Children's Hospital of Soochow University (Academic / other)
• Drugs / interventions: chemotherapy, immunotherapy, avaptitinib, gilteritinib
• Locations: 13 sites (Hefei, Anhui and 12 other locations)
• Trial ID: NCT06221683 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the effectiveness of different induction therapy options based on molecular subtyping and measurable residual disease (MRD) in children and adolescents diagnosed with acute myeloid leukemia (AML). The study will compare the rates of complete remission and event-free survival among patients receiving tailored chemotherapy regimens, including low-dose and standard-dose treatments, alongside targeted therapies. The trial will also assess the impact of specific fusion genes on treatment response and adjust therapies accordingly. By utilizing precision medicine approaches, the study seeks to improve outcomes for pediatric AML patients.

Who should consider this trial

Good fit: Ideal candidates for this study are children and adolescents under 18 years old who have newly diagnosed, untreated acute myeloid leukemia.

Not a fit: Patients with acute megakaryocytic leukemia, acute promyelocytic leukemia, or those with treatment-related secondary AML may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to more effective and personalized treatment options for children and adolescents with AML, potentially improving remission rates and survival outcomes.

How similar studies have performed: Previous studies have shown promising results with tailored chemotherapy approaches in pediatric AML, indicating potential for success with this novel strategy.

Eligibility criteria

Inclusion Criteria:

* 1、Newly diagnosed, untreated AML；
* 2、Under 18 years old;
* 3、Patients who have used hydroxyurea or cytarabine before diagnosis, but the dosage of cytarabine does not exceed 5 days, and the total dose does not exceed 500 mg/m2 (50 mg/m2, q12h × 5d)；
* 4、 Liver function：Tbil≤2×ULN, ALT/AST≤3×ULN, creatinine clearance ≥50ml/min；Cardiac NYHA grading\<3；SaO2\>92%;
* 5、No active infection (symptoms resolved for more than 3 days if infected)
* 6、ECOG\<2;
* 7、Expected survival time greater than 12 weeks;
* 9、Obtain the consent of the child and/or guardian and sign the informed consent form.

Exclusion Criteria:

* 1、Acute megakaryocytic leukemia (AMKL)；
* 2、Acute promyelocytic leukemia (APL);
* 3、Treatment-related secondary AML and AML with definite MDS transformation；
* 4、Myeloproliferative neoplasm (such as Juvenile myelomonocytic leukemia, JMML);
* 5、AML secondary to congenital bone marrow failure (such as AML secondary to Fanconi anemia (FA)；
* 6、AML secondary to Down syndrome;
* 7、Only temporary chemotherapy, radiotherapy, or immunotherapy, but not systematic treatment according to the treatment plan;
* 8、 Temporary chemotherapy, radiotherapy, or immunotherapy only, not systemic therapy per protocol;
* 9、Having any significant abnormal concurrent disease or mental illness that impacts the life safety and compliance of the patient and impacts informed consent, study participation, follow-up, or interpretation of results. In this case, all the participating units are required to report directly to the responsible person for this project to jointly decide whether they meet the exclusion criteria；
* 10、Patients with very poor nutritional status, severe infection, cardiac insufficiency, and intolerance to chemotherapy;
* 11、Relapsed AML at any time；
* 12、The attending physician considers that the patient is not suitable for entering the study protocol based on the patient's physical condition, economic status, and other factors.

Where this trial is running

Hefei, Anhui and 12 other locations

• First Affiliated Hospital Of University of Science and Technology of China — Hefei, Anhui, China (Not_yet_recruiting)
• The Second Hospital of Anhui Medical University — Hefei, Anhui, China (Not_yet_recruiting)
• Guangzhou Women and Children Medical Center — Guangzhou, Guangdong, China (Not_yet_recruiting)
• The First Affiliated Hospital of Guangxi Medical University — Nanning, Guangxi, China (Not_yet_recruiting)
• Kaifeng Children's Hospital — Kaifeng, Henan, China (Not_yet_recruiting)
• The First Affiliated Hospital of Zhengzhou University — Zhengzhou, Henan, China (Not_yet_recruiting)
• Third Xiangya Hospital of Central South University — Changsha, Hunan, China (Not_yet_recruiting)
• XiangYa Hospital Central South University — Changsha, Hunan, China (Not_yet_recruiting)
• Children's Hospital of Soochow University — Suzhou, Jiangsu, China (Recruiting)
• Xuzhou Children's Hospital — Xuzhou, Jiangsu, China (Not_yet_recruiting)
• Qilu Hospital of Shandong University — Jinan, Shandong, China (Not_yet_recruiting)
• Children's Hospital Of Fudan University — Shanghai, Shanghai Municipality, China (Not_yet_recruiting)
• Beijing Institute of Genomics, Chinese Academy of Sciences — Beijing, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Shaoyan Hu, MD, PhD

Conditions AML, Childhood; Acute Myeloid Leukemia; AML; Childhood; Molecular subtyping; MRD