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Chronic Myeloid LeukemiaPhase 2NCT02353728

Stem Cell Monitoring for CML Patients Undergoing Nilotinib Therapy

A Phase 2 study testing Nilotinib in people with Chronic Myeloid Leukemia, run by Weill Medical College of Cornell University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The study is an open-label phase 2 clinical and translational trial designed to evaluate the effects of nilotinib on the leukemic stem cell population in subjects with newly diagnosed chronic phase chronic myeloid leukemia (Ph+ CML in CP). Nilotinib is FDA-approved to treat subjects with Ph+ CML in CP. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Chronic Myeloid Leukemia
What is being tested?
Nilotinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteUnited States
Study dates
Jan 2015to Feb 2021
Phase
Phase 2
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

  • Patients 18 years or older
  • Eastern Cooperative Oncology Group (ECOG) Performance status 0,1, or 2
  • Documented diagnosis of Ph+ Chronic phase CML:
  • Chronic phase: None of the criteria for accelerated or blastic phase
  • Accelerated phase
  • Blasts ≥ 15% in blood or BM
Show all 26Show fewer
  • Blasts plus progranulocytes ≥ 30% in blood or bone marrow (BM)
  • Basophilia ≥ 20% in blood or BM
  • Platelets < 100 × 109/L unrelated to therapy
  • Cytogenetic clonal evolution
  • Blast phase
  • ≥ 30% blasts in blood or BM
  • Extramedullary disease with localized immature blasts
  • Adequate end organ function, defined as the following:
  • Creatinine < 1.5 x upper limit of normal (ULN)
  • Absolute neutrophil count (ANC) > 1.5 x 109/L
  • Platelets > 100 x 109/L
  • Total bilirubin < 1.5 x ULN (Does not apply to patients with isolated hyperbilirubinemia [e.g., Gilbert's disease] grade <3)
  • Aspartate aminotransferase (AST) (SGOT) and Alanine aminostransferase (ALT) (SGPT) < 3 x ULN
  • Serum amylase and lipase ≤ 2 x ULN
  • Alkaline phosphatase ≤ 2.5 x ULN
  • Patients must have the following laboratory values (WNL = within normal limits at the local institution lab) or corrected to within normal limits with supplements prior to the first dose of study medication:
  • Potassium (WNL)
  • Magnesium (WNL)
  • Phosphorus (WNL)
  • Calcium (WNL)

Who cannot join

  • Previous treatment with any other tyrosine kinase inhibitor except for up to 2 weeks of nilotinib
  • Impaired cardiac function including any one of the following:
  • Inability to monitor the QT interval on ECG
  • Congenital long QT syndrome or a known family history of long QT syndrome.
  • Clinically significant resting brachycardia (<50 beats per minute)
  • Q-T Corrected (corrected Q-T interval) (QTc) > 450 msec on baseline ECG. If QTc >450 msec and electrolytes are not within normal ranges, electrolytes should be corrected and then the patient re-screened for QTc
Show all 22Show fewer
  • Myocardial infarction within 12 months prior to starting study Other clinically significant uncontrolled heart disease (e.g. unstable angina, congestive heart failure or uncontrolled hypertension)
  • History of or presence of clinically significant ventricular or atrial tachyarrhythmias
  • Complete left bundle branch block
  • Right bundle branch block plus left anterior/posterior hemiblock
  • Use of ventricular-paced pacemaker
  • History of unstable angina within 1 year of study entry
  • Patients currently receiving treatment with strong CYP3A4 inhibitors and treatment cannot be either discontinued or switched to a different medication prior to starting study drug. (http://medicine.iupui.edu/clinpharm/ddis/) ).
  • Patients currently receiving treatment with any medications that have the potential to prolong the QT interval and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug (http://crediblemeds.org/)
  • Impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection or gastric bypass surgery).
  • History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
  • Severe and/or uncontrolled concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, uncontrolled infection)
  • History of another active malignancy within 5 years prior to study entry with the exception of previous or concomitant basal cell skin cancer and previous carcinoma in situ treated curatively
  • Known presence of significant congenital or acquired bleeding disorder unrelated to cancer
  • Major surgery within 4 weeks prior to Day 1 of the study or who have not recovered from prior surgery
  • Treatment with other investigational agents within 30 days of Day 1.
  • Pregnant or nursing (lactating) women, where pregnancy is defined as the state of female after conception and until the termination of gestation, confirmed by a positive Human chorionic gonadotropin (hCG) laboratory test. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during the study and for 14 days after the final dose of nilotinib.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Weill Cornell Medical College

    New York, New York, 10065

Other trials in United States

About this study

Official title: Detection, Monitoring, and Molecular Characterization of Leukemic Stem Cells From Patients With Chronic Myeloid Leukemia (CML) Undergoing Therapy With Nilotinib

The study is an open-label phase 2 clinical and translational trial designed to evaluate the effects of nilotinib on the leukemic stem cell population in subjects with newly diagnosed chronic phase chronic myeloid leukemia (Ph+ CML in CP). Nilotinib is FDA-approved to treat subjects with Ph+ CML in CP. Subjects on study will be monitored according to accepted National Cancer Comprehensive Network [NCCN] clinical guidelines for 24 months. After 24 months, if continued therapy is needed subjects will be transitioned to commercial supply of study drug.

Read the detailed description

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.

Being tested
Nilotinib
Medical subject headings
Leukemia, Myelogenous, Chronic, BCR-ABL Positive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NilotinibDrug

    Also called Tasigna, AMN107

Study groups in NCT02353728
Study groupRoleWhat they receive
All PatientsExperimentalNilotinib at a dose of 300 mg P.O. twice a day (BID) daily
Receives: Drug: Nilotinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population

    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.

    Measured: 1 month, 3 months, 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Feb 2015 (estimated)
  2. Study startJan 2015
  3. Primary completionFeb 2021
  4. Study completionFeb 2021

Resources

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 2
Primary purpose
Basic science
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02353728
Sponsor's protocol ID
1403014950
Status last verified
Mar 2022
First submitted
13 Jan 2015
Last updated
25 Mar 2022
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Nilotinib in people with Chronic Myeloid Leukemia, run by Weill Medical College of Cornell University 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 26 inclusion and 22 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 25 Mar 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 25 Mar 2022). 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?

Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population (time frame: 1 month, 3 months, 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02353728.

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 25 Mar 2022.

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