Beat AML Core Study

Beat AML: Personalized Medicine for Acute Myeloid Leukemia Based on Functional Genomics

In this study, DNA sequencing, computational biology modeling, and ex vivo drug sensitivity assays will be utilized to define clinically relevant gene mutations and identify potential therapeutics for patients with acute myeloid leukemia (AML).

Study Overview

• Status: Completed
• Conditions: Acute Myeloid Leukemia

Detailed Description

As part of normal clinical care, patients will undergo a peripheral blood draw and bone marrow aspiration & biopsy. Blood draws and bone marrow aspirations are performed at the time of diagnosis, after treatments , disease progression, and relapse. Under normal clinical care, patient specimens are analyzed by cytogenetics (giemsa staining), fluorescence in situ hybridization (FISH), and gene mutation profiling. Clinically, treatment can begin before these molecular diagnostics are available.

As part of this repository study, subjects are asked to:

• Allow access to banked blood and bone marrow specimens in IRB approved protocol # 532-2012.
• Donate peripheral blood specimens whenever blood is already being drawn for clinical purposes such as at times of diagnosis, relapse, refractory disease or disease progression. Additional samples may be requested at other standard of care visits in the event that initial samples are not viable for DNA sequencing, phenotyping, or functional assays for patients with AML, if disease is present.
• Donate bone marrow aspirate specimens whenever bone marrow aspiration is already being done for clinical purposes such as at times of diagnosis, relapse, refractory disease or disease progression. If bone marrow aspirate is being collected for banking protocol #532-2012, then an aliquot of the banked specimen will be accessed rather than collect an additional bone marrow aspirate for this study.
• Undergo skin biopsy and donate the skin biopsy specimen for genomic profiling.
• Allow bone marrow, peripheral blood and skin biopsy specimens to be collected for genomic profiling and ex vivo drug sensitivity testing.

• Study Type: Observational
• Enrollment (Actual): 22

Contacts and Locations

Study Locations

• United States
  • Florida
    • Gainesville, Florida, United States, 32608
      • UF Health Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

A total of 50 subjects with newly diagnosed or relapsed/refractory acute myeloid leukemia (AML) will be enrolled in this study.

Description

Inclusion Criteria:

• Individuals with newly diagnosed or relapsed/refractory Acute Myeloid Leukemia (AML) as defined by World Health Organization 2016.
• ≥ 18 years of age
• Capable of providing informed consent

Exclusion Criteria:

• 17 years of age or less
• greater than 80 years of age

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Acute Myeloid Leukemia (AML); AML samples will be collected from individuals with newly diagnosed or relapsed/refractory Acute Myeloid Leukemia (AML) as defined by World Health Organization 2016.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
the genomic abnormality spectrum	AML cells in the peripheral blood and bone marrow samples will be examined by next generation sequencing using an Illumina DNA sequencer. DNA from the skin biopsy will be used as the constitutional reference DNA. Using skin DNA greatly improves the ability to accurately and precisely identify somatic mutations in the AML cells.	5 years
drug sensitivity	Ex vivo drug sensitivity testing will be performed on each subject's AML cells derived from peripheral blood and bone marrow. AML cell viability will be recorded for each treatment condition after 72 hours of treatment. A rank-ordered list of drugs will be created in order of drug toxicity.	5 years

Collaborators and Investigators

• Sponsor: University of Florida
• Collaborators: Oregon Health and Science University; The Leukemia and Lymphoma Society; Cellworks Group Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 15, 2017
• Primary Completion (Actual): July 29, 2020
• Study Completion (Actual): July 29, 2020

Study Registration Dates

• First Submitted: October 5, 2016
• First Submitted That Met QC Criteria: October 5, 2016
• First Posted (Estimate): October 6, 2016

Study Record Updates

• Last Update Posted (Actual): August 5, 2020
• Last Update Submitted That Met QC Criteria: August 3, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Keywords: genomics; leukemia; myeloid
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: IRB201601364; OCR13966 (Other Identifier: University of Florida)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes