Acute Myeloid Leukemia (AML)Phase 3NCT02927938
Leukemia Stem Cell Detection in Acute Myeloid Leukemia
A Phase 3 study testing Allogeneic HCT and Consolidation chemotherapy in people with Acute Myeloid Leukemia (AML), run by Wake Forest University Health Sciences at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- What is being tested?
- Allogeneic HCT and Consolidation chemotherapy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2016to 5 Oct 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Any debilitating medical or psychiatric illness that would preclude ability to follow study procedures.
- Indeterminate leukemia stem cell assay results at diagnosis.
Trial locations and contacts
1 site in 1 country.
United States1 site
Levine Cancer Institute
Charlotte, North Carolina, 28204
About this study
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.
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- Allogeneic HCT, Consolidation chemotherapy
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic HCTProcedure or surgery
Also called transplant, allo HCT, PBSCT, SCT, BMT
Allogeneic HCT
Consolidation chemotherapyDrug
Also called cytarabine, HiDAc, high dose cytarabine
Cytarabine-based consolidation chemotherapy
| Study group | Role | What they receive |
|---|---|---|
| Evaluable Cohort - Transplant Arm | Other | 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:
1. Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria)
2. Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0)
3. 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 Receives: Procedure: Allogeneic HCT |
| Evaluable Cohort - Consolidation Chemo Arm | Other | 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:
1. Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria)
2. Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0)
3. Cytarabine-based induction subjects: Are candidates for (as determined by the investigator) and receive consolidation therapy (cytarabine-based chemotherapy or HCT) Receives: Drug: Consolidation chemotherapy |
| Observational Cohort 1 | No intervention | 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. |
| Observational Cohort 2 | No intervention | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2016 (estimated)
- Study startSep 2016
- Primary completion5 Oct 2018
- Study completion5 Oct 2018
Why it stopped: Study terminated 7/23/2019 due to limited participation and testing challenges.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael Grunwald, M.D., Principal investigator, Wake Forest University Health Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02927938
- Sponsor's protocol ID
- LCI-HEM-AML-SCD-001
- Status last verified
- Mar 2020
- First submitted
- 29 Sep 2016
- Last updated
- 21 Apr 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Leukemia Stem Cell Detection in Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT02927938
- Acronym / sponsor ID
- LCI-HEM-AML-SCD-001
- Conditions
- Acute Myeloid Leukemia (AML)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated 7/23/2019 due to limited participation and testing challenges.
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Study start
- Sep 2016
- Primary completion
- 5 Oct 2018 (actual)
- Study completion
- 5 Oct 2018 (actual)
- First posted
- 7 Oct 2016 (estimated)
- Results
- Posted 23 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Evaluable Cohort - Transplant Arm | Other | Procedure: Allogeneic HCTOther - 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: 1. Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria) 2. Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0) 3. 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 |
| Evaluable Cohort - Consolidation Chemo Arm | Other | Drug: Consolidation chemotherapyOther - 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: 1. Complete remission (CR1) from standard cytarabine or HMA-based induction therapy per standard clinical criteria (Cheson Criteria) 2. Have confirmed presence of CD34+CD38-ALDHint population by flow cytometry at the diagnostic LSC assay (LSC0) 3. Cytarabine-based induction subjects: Are candidates for (as determined by the investigator) and receive consolidation therapy (cytarabine-based chemotherapy or HCT) |
| Observational Cohort 1 | No intervention | –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. |
| Observational Cohort 2 | No intervention | –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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- Any debilitating medical or psychiatric illness that would preclude ability to follow study procedures.
- Indeterminate leukemia stem cell assay results at diagnosis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic HCTAlso: transplant, allo HCT, PBSCT, SCT, BMTAllogeneic HCT | Procedure or surgery | Evaluable Cohort - Transplant Arm |
| Consolidation chemotherapyAlso: cytarabine, HiDAc, high dose cytarabineCytarabine-based consolidation chemotherapy | Drug | Evaluable Cohort - Consolidation Chemo Arm |
Primary outcome
2 Year Relapse Free Survival (RFS)
Time frame: 2 years
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Grunwald, M.D. principal investigator
Wake Forest University Health Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Allogeneic HCT and Consolidation chemotherapy in people with Acute Myeloid Leukemia (AML), run by Wake Forest University Health Sciences at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 21 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2 Year Relapse Free Survival (RFS) (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Apr 2022.