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

Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01576185
• Other study ID #: AAML12B8
• Secondary ID: NCI-2012-00724CO
• Status: Recruiting
• Phase: N/A
• First received: April 11, 2012
• Last updated: November 5, 2015
• Start date: April 2012

Study information

• Verified date: November 2015
• Source: Children's Oncology Group
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. primary completion date: January 2100
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Cryopreserved human AML samples

• FLT3-ITD samples with high allelic ratios

Study Design

Observational Model: Case-Only, Time Perspective: Prospective

Related Conditions & MeSH terms

• Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies
• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute

Intervention

Other:
• laboratory biomarker analysis
Correlative studies

Drug:
• quizartinib
Via gavage
• sorafenib tosylate
Via gavage

Locations

Country	Name	City	State
United States	Children's Oncology Group	Monrovia	California

Sponsors (2)

Lead Sponsor	Collaborator
Children's Oncology Group	National Cancer Institute (NCI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Engraftment ratio of human AML cells to murine cells	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.	Up to 9 months	No
Primary	Efficacy of sorafenib or quizartinib to inhibit AML proliferation in vivo		Up to 9 months	No