Developing and Treating a Mouse Model of Acute Myeloid Leukemia Using Tissue Samples From Younger Patients With Acute Myeloid Leukemia

Development of Pediatric Acute Myeloid Leukemia Xenograft Models for the Testing of Targeted Therapeutic Agents

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01576185

Enrollment

Registered

2012-04-12

Start date

2012-05-31

Completion date

2016-05-31

Last updated

2016-05-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies

Brief summary

These laboratory trial studies the development and treatment of a mouse model for acute myeloid leukemia (AML) using samples from younger patients with AML. Studying tissue samples from patients with cancer in the laboratory may help doctors learn more about cancer and how well patients will respond to treatment.

Detailed description

PRIMARY OBJECTIVES: I. To determine the rate of engraftment of pediatric FMS-Like Tyrosine Kinase-3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) samples in NOD scid gamma (NSG) mice. II. To determine the efficacy of treatment of FLT3-ITD xenografts with tyrosine kinase inhibitors. OUTLINE: Human acute myeloid leukemia cells are injected into NSG mice. Mice are then treated with sorafenib or quizartinib via gavage once daily for 28 days. Peripheral blood and tissue samples are collected biweekly or weekly and analyzed for the presence of human CD45+ and CD33+ cells by quantitative flow cytometry.

Interventions

OTHERlaboratory biomarker analysis

Correlative studies

DRUGquizartinib

Via gavage

DRUGsorafenib tosylate

Via gavage

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

* Cryopreserved human AML samples * FLT3-ITD samples with high allelic ratios

Design outcomes

Primary

Measure	Time frame	Description
Engraftment ratio of human AML cells to murine cells	Up to 9 months	We will measure total leukemic burden from harvested femurs, tibias, and spleen by quantitative flow cytometry, estimate engraftment, and describe 95% confidence intervals. The total AML cell count of the control and treatment cohorts will be compared using an analysis of variance (ANOVA) test.
Efficacy of sorafenib or quizartinib to inhibit AML proliferation in vivo	Up to 9 months	—

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026