Stem Cell Monitoring for CML Patients Undergoing Nilotinib Therapy
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|ClinicalTrials.gov Identifier: NCT02353728|
Recruitment Status : Completed
First Posted : February 3, 2015
Results First Posted : March 25, 2022
Last Update Posted : March 25, 2022
|Condition or disease||Intervention/treatment||Phase|
|Chronic Myeloid Leukemia||Drug: Nilotinib||Phase 2|
Patients with newly diagnosed Ph+ CML in chronic phase will be eligible for enrollment in this trial. Prior treatment with nilotinib for less than 2 weeks and hydroxyurea is allowed.
Before therapy and during therapy, peripheral blood and bone marrow samples will be obtained for cytogenetic and molecular evaluations. During study, blood will be collected at approximately month 1, month 3, and every 3 months thereafter; aspirate samples will be collected at approximately month 1, month 3, and month 12. These samples will be collected to analyze the quantitative and qualitative changes in the leukemic stem cell population before and during therapy with nilotinib. The study is intended as a hypothesis finding analysis in order to establish whether in response to nilotinib therapy, defined differences in the baseline or therapy-induced changes in the characteristics of the stem cell population will be predictive of the ability to successfully discontinue therapy in subjects with CML.
In order to determine the effect of nilotinib in stem and progenitor populations we will evaluate 40 newly diagnosed CML subjects undergoing treatment with nilotinib at different time points. We will evaluate the levels of expression of Breakpoint Cluster Region-Abelson protooncogene (BCR-ABL) in purified stem cell populations during the course of treatment. In addition, we will compare the stem and progenitor populations present during the course of treatment in peripheral blood and bone marrow. We will perform transcriptional profiling of such populations to determine changes in signaling pathways driving survival, self-renewal or proliferation. Whole exome sequencing will be also performed in all diagnostic samples to determine whether there are novel cooperating mutations.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||16 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Basic Science|
|Official Title:||Detection, Monitoring, and Molecular Characterization of Leukemic Stem Cells From Patients With Chronic Myeloid Leukemia (CML) Undergoing Therapy With Nilotinib|
|Actual Study Start Date :||January 2015|
|Actual Primary Completion Date :||February 2021|
|Actual Study Completion Date :||February 2021|
Experimental: All Patients
Nilotinib at a dose of 300 mg P.O. twice a day (BID) daily
- Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population [ Time Frame: 1 month, 3 months, 12 months ]The data obtained from these bone marrow samples, from these patients, may identify stem cell variables that can more accurately predict the success of discontinuation of tyrosine kinase inhibitor (TKI) therapy.
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): NCT02353728
|United States, New York|
|Weill Cornell Medical College|
|New York, New York, United States, 10065|
|Principal Investigator:||Ellen K Ritchie, MD||Associate Professor of Clinical Medicine|