Biomarkers for Personalized Early Assessment of Response During Salvage Chemotherapy in People With Relapsed or Refractory Acute Myeloid Leukemia (PEARL15)
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|ClinicalTrials.gov Identifier: NCT02527447|
Recruitment Status : Completed
First Posted : August 19, 2015
Last Update Posted : October 30, 2019
-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.
-To see if a blood test on day 4 of therapy can help identify those who will not respond to treatment for AML.
-People ages 18-70 who have refractory or relapsed AML and have had at least one previous therapy for it.
- 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.
|Condition or disease|
|Acute Myeloid Leukemia|
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 Type :||Observational|
|Actual Enrollment :||15 participants|
|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|
|Study Start Date :||August 18, 2015|
|Actual Primary Completion Date :||May 10, 2018|
|Actual Study Completion Date :||May 10, 2018|
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02527447
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Christopher S Hourigan, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|