Beat AML Core Study

• ClinicalTrials.gov Identifier: NCT02927106
• Recruitment Status: Completed
• First Posted: October 6, 2016
• Last Update Posted: August 5, 2020
• Sponsor: University of Florida
• Collaborators: Oregon Health and Science University; Cellworks Group Inc.; The Leukemia and Lymphoma Society
• Information provided by (Responsible Party): University of Florida

Study Description

Brief Summary:

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).

Condition or disease
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 Design

• Study Type: Observational
• Actual Enrollment: 22 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Beat AML: Personalized Medicine for Acute Myeloid Leukemia Based on Functional Genomics
• Actual Study Start Date: February 15, 2017
• Actual Primary Completion Date: July 29, 2020
• Actual Study Completion Date: July 29, 2020

Groups and Cohorts

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.

Outcome Measures

Primary Outcome Measures :

1. the genomic abnormality spectrum [ Time Frame: 5 years ]
   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.
2. drug sensitivity [ Time Frame: 5 years ]
   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.

Biospecimen Retention:   Samples With DNA

Perform molecular and cellular studies on blood, skin biopsy, and bone marrow samples done at the time of diagnosis, after treatments, disease progression, and relapse.

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 80 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• 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.

Criteria

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

Contacts and Locations

Locations

United States, Florida

UF Health Cancer Center

Gainesville, Florida, United States, 32608

Sponsors and Collaborators

University of Florida

Oregon Health and Science University

Cellworks Group Inc.

The Leukemia and Lymphoma Society

Investigators

• Principal Investigator: Christopher R. Cogle, MD; University of Florida

More Information

• Responsible Party: University of Florida
• ClinicalTrials.gov Identifier: NCT02927106
• Other Study ID Numbers: IRB201601364; OCR13966 ( Other Identifier: University of Florida )
• First Posted: October 6, 2016
• Last Update Posted: August 5, 2020
• Last Verified: August 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes

• 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

Keywords provided by University of Florida:

leukemia; myeloid; genomics

Additional relevant MeSH terms:

Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Neoplasms by Histologic Type; Neoplasms