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Leukemia Stem Cell Detection in Acute Myeloid Leukemia

Primary Purpose

Acute Myeloid Leukemia (AML)

Status
Terminated
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Allogeneic HCT
Consolidation chemotherapy
Sponsored by
Wake Forest University Health Sciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia (AML)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Must have previously signed the specimen procurement protocol consent associated with the leukemia stem cell assay ("Step 1 informed consent") prior to starting AML therapy.
  2. Age 18 years and older
  3. New diagnosis of AML, other than APL, confirmed by bone marrow aspirate/biopsy and reviewed by an institutional hematopathologist
  4. Completion of induction therapy, as defined by the Investigator and post-induction bone marrow biopsy.

Exclusion Criteria:

  1. Any debilitating medical or psychiatric illness that would preclude ability to follow study procedures.
  2. Indeterminate leukemia stem cell assay results at diagnosis.

Sites / Locations

  • Levine Cancer Institute

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Other

Other

No Intervention

No Intervention

Arm Label

Evaluable Cohort - Transplant Arm

Evaluable Cohort - Consolidation Chemo Arm

Observational Cohort 1

Observational Cohort 2

Arm Description

Other - Standard of Care Consolidation (HCT) Enrolled subjects that will contribute to the population of subjects who are evaluable for the primary and secondary objectives. This will not include any subjects who end up in either observational cohort. To be included in the evaluable cohort, the subject must meet the following requirements: Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria) Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0) Cytarabine-based induction subjects: Are candidates for (as determined by the investigator) and receive consolidation therapy (cytarabine-based chemotherapy or HCT); HMA-based induction subjects: Are candidates for (as determined by the investigator) and receive HCT

Other - Standard of Care Consolidation (cytarabine-based chemo) Enrolled subjects that will contribute to the population of subjects who are evaluable for the primary and secondary objectives. This will not include any subjects who end up in either observational cohort. To be included in the evaluable cohort, the subject must meet the following requirements: Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria) Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0) Cytarabine-based induction subjects: Are candidates for (as determined by the investigator) and receive consolidation therapy (cytarabine-based chemotherapy or HCT)

Enrolled subjects who do not achieve a CR to induction therapy, regardless of diagnostic phenotype. Following completion of induction therapy and remission bone marrow aspirate, if a subject is determined to not have achieved a complete remission to induction therapy, he or she would be included in observational cohort 1.

Enrolled subjects who achieve a CR to induction therapy but meet one or more of the following criteria: Lack the immunophenotype of interest, Cytarabine based induction subjects: Are not candidates for [as determined by the investigator (e.g. unfit or refusal)] and do not receive consolidation therapy (cytarabine-based chemotherapy or HCT) HMA-based induction subjects: Are not candidates for [as determined by the investigator (e.g. unfit, lack of donor, refusal)] and do not receive HCT Final investigator determination of fit-ness can occur at any time until the start of consolidation therapy. HMA-based induction subjects will not receive consolidation cytarabine-based chemotherapy as part of the evaluable cohort if they do not receive HCT.

Outcomes

Primary Outcome Measures

2 Year Relapse Free Survival (RFS)
Comparison of 2 year RFS in patient with detectable LSCs in the marrow at the end of consolidation to the 2 year RFS of patients without detectable LSCs. IWG Criteria (Cheson 2003) was utilized to classify relapse, with relapse defined as ≥ 5% blasts in the marrow or peripheral blood, extramedullary disease, or disease presence determined by a physician upon clinical assessment.

Secondary Outcome Measures

Full Information

First Posted
September 29, 2016
Last Updated
April 19, 2022
Sponsor
Wake Forest University Health Sciences
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1. Study Identification

Unique Protocol Identification Number
NCT02927938
Brief Title
Leukemia Stem Cell Detection in Acute Myeloid Leukemia
Official Title
Leukemia Stem Cell Detection in Acute Myeloid Leukemia
Study Type
Interventional

2. Study Status

Record Verification Date
March 2020
Overall Recruitment Status
Terminated
Why Stopped
Study terminated 7/23/2019 due to limited participation and testing challenges.
Study Start Date
September 2016 (undefined)
Primary Completion Date
October 5, 2018 (Actual)
Study Completion Date
October 5, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Wake Forest University Health Sciences

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Most patients with acute myeloid leukemia (AML) achieve complete remission (CR) following induction chemotherapy. However, a large majority subsequently relapse and succumb to the disease. Currently, cytogenetics and molecular aberrations are the best prognostic indicators; however, these factors cannot prognosticate accurately for individual patients. Overall, the majority of patients with favorable or intermediate-risk AML will experience relapse. Prognosis after relapse is dismal with a five-year overall survival rate of less than 10%. A leukemia stem cell (LSC) paradigm may explain this failure of CR to reliably translate into cure. This study is undertaken to determine whether the presence of LSCs has prognostic value as well as to determine whether the presence of LSCs has predictive value. This study has an observational component, whereby we intent evaluate whether the presence or absence of LSCs is prognostic. This study also has an interventional component in which it uses LSC status to determine whether favorable and intermediate risk AML patients in CR receive consolidation with chemotherapy or allogeneic HCT.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia (AML)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Evaluable Cohort - Transplant Arm
Arm Type
Other
Arm Description
Other - Standard of Care Consolidation (HCT) Enrolled subjects that will contribute to the population of subjects who are evaluable for the primary and secondary objectives. This will not include any subjects who end up in either observational cohort. To be included in the evaluable cohort, the subject must meet the following requirements: Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria) Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0) Cytarabine-based induction subjects: Are candidates for (as determined by the investigator) and receive consolidation therapy (cytarabine-based chemotherapy or HCT); HMA-based induction subjects: Are candidates for (as determined by the investigator) and receive HCT
Arm Title
Evaluable Cohort - Consolidation Chemo Arm
Arm Type
Other
Arm Description
Other - Standard of Care Consolidation (cytarabine-based chemo) Enrolled subjects that will contribute to the population of subjects who are evaluable for the primary and secondary objectives. This will not include any subjects who end up in either observational cohort. To be included in the evaluable cohort, the subject must meet the following requirements: Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria) Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0) Cytarabine-based induction subjects: Are candidates for (as determined by the investigator) and receive consolidation therapy (cytarabine-based chemotherapy or HCT)
Arm Title
Observational Cohort 1
Arm Type
No Intervention
Arm Description
Enrolled subjects who do not achieve a CR to induction therapy, regardless of diagnostic phenotype. Following completion of induction therapy and remission bone marrow aspirate, if a subject is determined to not have achieved a complete remission to induction therapy, he or she would be included in observational cohort 1.
Arm Title
Observational Cohort 2
Arm Type
No Intervention
Arm Description
Enrolled subjects who achieve a CR to induction therapy but meet one or more of the following criteria: Lack the immunophenotype of interest, Cytarabine based induction subjects: Are not candidates for [as determined by the investigator (e.g. unfit or refusal)] and do not receive consolidation therapy (cytarabine-based chemotherapy or HCT) HMA-based induction subjects: Are not candidates for [as determined by the investigator (e.g. unfit, lack of donor, refusal)] and do not receive HCT Final investigator determination of fit-ness can occur at any time until the start of consolidation therapy. HMA-based induction subjects will not receive consolidation cytarabine-based chemotherapy as part of the evaluable cohort if they do not receive HCT.
Intervention Type
Procedure
Intervention Name(s)
Allogeneic HCT
Other Intervention Name(s)
transplant, allo HCT, PBSCT, SCT, BMT
Intervention Description
Allogeneic HCT
Intervention Type
Drug
Intervention Name(s)
Consolidation chemotherapy
Other Intervention Name(s)
cytarabine, HiDAc, high dose cytarabine
Intervention Description
Cytarabine-based consolidation chemotherapy
Primary Outcome Measure Information:
Title
2 Year Relapse Free Survival (RFS)
Description
Comparison of 2 year RFS in patient with detectable LSCs in the marrow at the end of consolidation to the 2 year RFS of patients without detectable LSCs. IWG Criteria (Cheson 2003) was utilized to classify relapse, with relapse defined as ≥ 5% blasts in the marrow or peripheral blood, extramedullary disease, or disease presence determined by a physician upon clinical assessment.
Time Frame
2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Must have previously signed the specimen procurement protocol consent associated with the leukemia stem cell assay ("Step 1 informed consent") prior to starting AML therapy. Age 18 years and older New diagnosis of AML, other than APL, confirmed by bone marrow aspirate/biopsy and reviewed by an institutional hematopathologist Completion of induction therapy, as defined by the Investigator and post-induction bone marrow biopsy. Exclusion Criteria: Any debilitating medical or psychiatric illness that would preclude ability to follow study procedures. Indeterminate leukemia stem cell assay results at diagnosis.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael Grunwald, M.D.
Organizational Affiliation
Wake Forest University Health Sciences
Official's Role
Principal Investigator
Facility Information:
Facility Name
Levine Cancer Institute
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28204
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Leukemia Stem Cell Detection in Acute Myeloid Leukemia

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