Acute Myeloid LeukemiaNCT02527447
Biomarkers for Personalized Early Assessment of Response During Salvage Chemotherapy in People With Relapsed or Refractory Acute Myeloid Leukemia (PEARL15)
An observational study of Acute Myeloid Leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Background: -Acute myeloid leukemia (AML) is a cancer of the white blood cells. It can be fatal. Standard treatment involves intensive chemotherapy. Not all treatment works. AML that has not responded to treatment (refractory) or that has returned after treatment (relapsed) is high-risk even with treatment. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Acute Myeloid Leukemia
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Aug 2015to 10 May 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
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 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Unequivocal diagnosis of relapsed or refractory acute myeloid leukemia (AML) according to WHO criteria confirmed by bone marrow evaluation within 30 days prior to study enrollment
- Age 18-70 years inclusive
- ECOG performance status of 0 to 2
- Must have received at least one prior AML therapy before study enrollment
- Ability to comprehend the investigational nature of the study and provide informed consent
- Availability of a physician willing to assume clinical care after completion of this research study.
- Subject agreement to use a medically-approved method of contraception to avoid pregnancy throughout the study if a woman of childbearing potential or a male subject with partner of childbearing potential.
Who cannot join
- Diagnosis of acute promyelocytic leukemia
- Decreased oxygen saturation at rest (e.g. pulse oximeter less than 88% or PaO2 less than or equal to 55 millimeters of mercury)
- Clinically significant active infection not responding adequately to therapy
- Known positive or history of HIV; active Hepatitis B or C infection. If unknown, screening will be done but results are not needed before proceeding with treatment
- Prior allogeneic hematopoietic stem cell transplant
- Additional malignancy requiring concurrent treatment
Show all 11Show fewer
- Uncontrolled hepatic, renal, cardiac, gastrointestinal, pulmonary, neurologic, infectious, metabolic or other disease of such severity, which in the opinion of the PI, would preclude ability to tolerate protocol.
- Severe psychiatric illness or complex social situations that would limit the patient s ability to tolerate and/or comply with study requirements.
- Active central nervous system (CNS) leukemic infiltration
- Current pregnancy or breastfeeding
- Prolonged QTc interval prior to therapy (>470ms)
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: A Pilot Study of Biomarkers for Personalized Early Assessment of Response During Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia
Background:
-Acute myeloid leukemia (AML) is a cancer of the white blood cells. It can be fatal. Standard treatment involves intensive chemotherapy. Not all treatment works. AML that has not responded to treatment (refractory) or that has returned after treatment (relapsed) is high-risk even with treatment. Success of therapy is normally determined after 28 to 56 days. This study will see if a blood test on day 4 of therapy can help identify earlier those who will not respond.
Read more
Objectives:
-To see if a blood test on day 4 of therapy can help identify those who will not respond to treatment for AML.
Eligibility:
-People ages 18-70 who have refractory or relapsed AML and have had at least one previous therapy for it.
Design:
- Participants will be screened with medical history, physical exam, and blood tests.
- Participants will have:
- Several blood tests.
- Bone marrow exams: a needle is inserted into the hip to take cells from the bone marrow.
- Echocardiogram: a small probe is held to the chest to take pictures of the heart.
- ECG: soft electrodes are stuck to the skin. A machine records the heart s signals.
- CT scans: they will lie in a machine that takes pictures of the body.
- Standard chemotherapy.
- Possible transfusions of blood products such as red blood cells or platelets.
- Participants will be expected to stay in the study typically for 2 3 months. This will include inpatient treatment. Inpatient stay normally will be 1 or 2 months.
Read the detailed description
Effective treatment of patients with relapsed and refractory acute myeloid leukemia (RR-AML) remains a significant unmet need. Current response criteria were originally proposed in 1956 and do not provide a sensitive assessment of AML disease burden, as evidenced by the disconnect between the apparent success of current induction therapy in achieving complete remission in most patients and the stark reality of median overall survival times of less than two years. While sensitive minimal/measurable residual disease assays assessing AML disease burden have been developed, the role for these tests in the treatment algorithm for patients with RR-AML has yet to be defined. We propose evaluating if these high sensitivity assays could function as early predictors of salvage therapy failure.
This protocol is designed as a feasibility study to evaluate if use of high sensitivity peripheral blood-based molecular assays on day 4 of induction salvage chemotherapy can predict failure to ultimately achieve a CR or CRi in patients with RR-AML. Eligible patients will receive salvage induction therapy using FDA approved antineoplastic agents given either alone or in combinations as defined in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for AML.
Additional secondary objectives include investigating the relationship between kinetics of gene expression changes and response to chemotherapy, determining the proportion of patients for whom results of day 4 peripheral blood gene expression testing are available before day 8 of therapy, determining the proportion of patients for whom results from pre-treatment molecular and genetic testing are available before day 8 of therapy, assessing patient and disease specific factors associated with failure to achieve a CR, determining the feasibility of measuring AML residual disease burden using alternative technologies and alternative tissue sources, determining the proportion of patients who receive allogeneic hematopoietic stem cell transplant after completion of therapy, determining the incidence of infectious complications with the use of modern prophylactic antimicrobial agents compared with historical comparisons and determining the feasibility of recruiting patients with RR-AML to the NIH Clinical Center.
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Measurable Residual Disease, Relapsed/Refractory AML, Salvage Chemotherapy
- Medical subject headings
- Leukemia, Myeloid, Acute, Neoplasm, Residual
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome of this protocol are the outcomes of the blood test performed on day 4 during chemotherapy, and the standardresponse criteria including morphological bone marrow examination following chemotherapy.
The primary outcome of this protocol are the outcomes of the blood test performed on day 4 during chemotherapy, and the standard response criteria at day 30 including morphological bone marrow examination following chemotherapy.
Measured: 4 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2015 (estimated)
- Study start18 Aug 2015
- Primary completion10 May 2018
- Study completion10 May 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Christopher S Hourigan, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02527447
- Sponsor's protocol ID
- 150176
- Other IDs
- 15-H-0176
- Status last verified
- 28 Oct 2019
- First submitted
- 18 Aug 2015
- Last updated
- 30 Oct 2019
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Biomarkers for Personalized Early Assessment of Response During Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT02527447
- Acronym / sponsor ID
- 150176
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Measurable Residual Disease, Relapsed/Refractory AML, Salvage Chemotherapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 18 Aug 2015
- Primary completion
- 10 May 2018 (actual)
- Study completion
- 10 May 2018 (actual)
- First posted
- 19 Aug 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
-Acute myeloid leukemia (AML) is a cancer of the white blood cells. It can be fatal. Standard treatment involves intensive chemotherapy. Not all treatment works. AML that has not responded to treatment (refractory) or that has returned after treatment (relapsed) is high-risk even with treatment. Success of therapy is normally determined after 28 to 56 days. This study will see if a blood test on day 4 of therapy can help identify earlier those who will not respond.
Objectives:
-To see if a blood test on day 4 of therapy can help identify those who will not respond to treatment for AML.
Eligibility:
-People ages 18-70 who have refractory or relapsed AML and have had at least one previous therapy for it.
Design:
- Participants will be screened with medical history, physical exam, and blood tests.
- Participants will have:
- Several blood tests.
- Bone marrow exams: a needle is inserted into the hip to take cells from the bone marrow.
- Echocardiogram: a small probe is held to the chest to take pictures of the heart.
- ECG: soft electrodes are stuck to the skin. A machine records the heart s signals.
- CT scans: they will lie in a machine that takes pictures of the body.
- Standard chemotherapy.
- Possible transfusions of blood products such as red blood cells or platelets.
- Participants will be expected to stay in the study typically for 2 3 months. This will include inpatient treatment. Inpatient stay normally will be 1 or 2 months.
Detailed description
Effective treatment of patients with relapsed and refractory acute myeloid leukemia (RR-AML) remains a significant unmet need. Current response criteria were originally proposed in 1956 and do not provide a sensitive assessment of AML disease burden, as evidenced by the disconnect between the apparent success of current induction therapy in achieving complete remission in most patients and the stark reality of median overall survival times of less than two years. While sensitive minimal/measurable residual disease assays assessing AML disease burden have been developed, the role for these tests in the treatment algorithm for patients with RR-AML has yet to be defined. We propose evaluating if these high sensitivity assays could function as early predictors of salvage therapy failure.
This protocol is designed as a feasibility study to evaluate if use of high sensitivity peripheral blood-based molecular assays on day 4 of induction salvage chemotherapy can predict failure to ultimately achieve a CR or CRi in patients with RR-AML. Eligible patients will receive salvage induction therapy using FDA approved antineoplastic agents given either alone or in combinations as defined in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for AML.
Additional secondary objectives include investigating the relationship between kinetics of gene expression changes and response to chemotherapy, determining the proportion of patients for whom results of day 4 peripheral blood gene expression testing are available before day 8 of therapy, determining the proportion of patients for whom results from pre-treatment molecular and genetic testing are available before day 8 of therapy, assessing patient and disease specific factors associated with failure to achieve a CR, determining the feasibility of measuring AML residual disease burden using alternative technologies and alternative tissue sources, determining the proportion of patients who receive allogeneic hematopoietic stem cell transplant after completion of therapy, determining the incidence of infectious complications with the use of modern prophylactic antimicrobial agents compared with historical comparisons and determining the feasibility of recruiting patients with RR-AML to the NIH Clinical Center.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Unequivocal diagnosis of relapsed or refractory acute myeloid leukemia (AML) according to WHO criteria confirmed by bone marrow evaluation within 30 days prior to study enrollment
- Age 18-70 years inclusive
- ECOG performance status of 0 to 2
- Must have received at least one prior AML therapy before study enrollment
- Ability to comprehend the investigational nature of the study and provide informed consent
Key exclusion criteria (11 listed)
- Diagnosis of acute promyelocytic leukemia
- Decreased oxygen saturation at rest (e.g. pulse oximeter less than 88% or PaO2 less than or equal to 55 millimeters of mercury)
- Clinically significant active infection not responding adequately to therapy
- Known positive or history of HIV; active Hepatitis B or C infection. If unknown, screening will be done but results are not needed before proceeding with treatment
- Prior allogeneic hematopoietic stem cell transplant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The primary outcome of this protocol are the outcomes of the blood test performed on day 4 during chemotherapy, and the standardresponse criteria including morphological bone marrow examination following chemotherapy.
Time frame: 4 days
The primary outcome of this protocol are the outcomes of the blood test performed on day 4 during chemotherapy, and the standard response criteria at day 30 including morphological bone marrow examination following chemotherapy.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2019.
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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.
Christopher S Hourigan, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Myeloid Leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) 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 7 inclusion and 11 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: finished, as of the sponsor's last update on 30 Oct 2019. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The primary outcome of this protocol are the outcomes of the blood test performed on day 4 during chemotherapy, and the standardresponse criteria including morphological bone marrow examination following chemotherapy. (time frame: 4 days). 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 30 Oct 2019.