Study of Nilotinib in Ph+ CML-CP Patients With Low Imatinib Trough Plasma Concentrations (MACS1148)
Primary Purpose
CML, Philadelphia Chromosome Positive (Ph+), Chronic Myelogenous Leukemia Chronic Phase(CML-CP) Patients With Low Imatinib Trough Levels
Status
Terminated
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
nilotinib
Sponsored by
About this trial
This is an interventional treatment trial for CML focused on measuring Philadelphia chromosome positive, Ph+, chronic myelogenous leukemia chronic phase, CML-CP, low imatinib trough levels
Eligibility Criteria
Inclusion Criteria:
- Cytogenetically confirmed Ph+ CML-CP Any prior dose of Imatinib
- Imatinib 400 mg daily for ≥7 consecutive days prior to imatinib trough collection
- Imatinib trough plasma concentration <850 ng/mL
Exclusion Criteria:
- Prior documented failure events as defined by ELN guidelines:
- Loss of CHR, CCyR, or clonal progression/Ph+
- Less than CHR at 3 months after diagnosis
- No CyR at 6 months after diagnosis
- Less than PCyR at 12 months after diagnosis
- Less than CCyR at 18 months after diagnosis
- Prior accelerated phase or blast phase CML
- Previously documented T315I mutation
- Previous treatment for CML with any other tyrosine kinase inhibitor except for imatinib
- Patients who had any other treatment for CML (transplant) except interferon +/- ara- C, imatinib, hydroxyurea and/or anagrelide
- Impaired cardiac function
- Patients receiving therapy with strong inhibitors of CYP3A4 or medications that prolong the QT interval and cannot be either discontinued or switched to a different medication prior to starting study drug.
- Any other malignancy that is clinically significant or requires active intervention.
- Major surgery within 4 weeks prior to Day 1 of study or who have not recovered from prior surgery
- Treatment with other investigational agents within 30 days of Day 1
- Women who are pregnant, breast feeding, or of childbearing potential without a negative serum test at baseline. Post-menopausal women must be amenorrhoeic for at least 12 months to be considered of non-childbearing potential. Women of childbearing potential must have a negative serum pregnancy test within 7 days of the first dose of nilotinib
- Sexually active male and female patients taking nilotinib unwilling to use adequate contraception throughout the trial and 3 months following discontinuation of study drug
Sites / Locations
- Comprehensive Cancer Centers of Nevada CCC of Nevada (1)
- Cancer Center of the High Plains
- Baylor Health Care System/Sammons Cancer Center Dept. of Sammons Cancer (2)
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Nilotinib
Arm Description
Outcomes
Primary Outcome Measures
Number of Treatment Failure Events up to 2 Years
Treatment failure events from time of study entry in Complete molecular response-Chronic phase (CML-CP) participants with low imatinib trough concentrations less than 850 nanogram per milliliter (<850 ng/mL) treated with nilotinib as defined in European LeukemiaNet (ELN)-guideline.
Secondary Outcome Measures
European LeukemiaNet (ELN)-Defined Optimal Responses
Loss of Complete Cytogenetic Response (CCyR), Major Molecular Response (MMR) and Complete Molecular Response (CMR) on Nilotinib
Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as CCyR at 12 cycles if the patient met the CCyR criteria at the Cycle 12 Visit.
Major molecular response is defined as values equal or below 0.1% on the International Scale.
Complete Molecular Response is defined as a Bcr-Abl (a fusion of gene of Bcr and ABl genes) ratio ≤0.0032% on the International Scale Bcr = breakpoint cluster gene Abl = abelson proto-oncogene.
Duration of Complete Cytogenetic Response (CCyR), Major Molecular Response (MMR) and Complete Molecular Response (CMR)Achieved on Nilotinib
Durations of major/complete cytogenetic response is defined as the time from the first documentation of the major/ complete response to the first documentation of the disease progression.
Event-free Survival (EFS), Progression-free Survival (PFS) and Overall Survival (OS) up to 2 Years
Event-free survival was defined as the time from the date of randomization to the date of first occurrence of any of the following events on study treatment: loss of complete hematological response, confirmed loss of complete cytogenetic response (CCyR), confirmed loss of major molecular response (MMR), death from any cause during treatment, progression to the accelerated phase or blast crisis of chronic myelogenous leukemia (CML) per European Leukemia Network (ELN) criteria, whichever was earliest.
Progressions free survival is defined as time between Day 1 cycle 1 and time to first documented disease progression or death. Disease progression will be determined as per response criteria.
Overall survival time is defined as the time from the treatment start to the date of death due to any reason.
European LeukemiaNet (ELN)-Defined Suboptimal Events
Number of Participants Reported Adverse Events
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. An SAE is defined as any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect, or is an event of possible drug-induced liver injury.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01131325
Brief Title
Study of Nilotinib in Ph+ CML-CP Patients With Low Imatinib Trough Plasma Concentrations
Acronym
MACS1148
Official Title
A Multi-center, Single Arm Study of Nilotinib in Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) Patients With Low Imatinib Trough Plasma Concentrations
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Terminated
Study Start Date
October 21, 2010 (Actual)
Primary Completion Date
May 12, 2011 (Actual)
Study Completion Date
May 12, 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study is to determine the number of European Leukemia Network (ELN)guideline defined treatment failure events from time of study entry in CML-CP patients with low imatinib trough concentrations treated with nilotinib.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
CML, Philadelphia Chromosome Positive (Ph+), Chronic Myelogenous Leukemia Chronic Phase(CML-CP) Patients With Low Imatinib Trough Levels
Keywords
Philadelphia chromosome positive, Ph+, chronic myelogenous leukemia chronic phase, CML-CP, low imatinib trough levels
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Nilotinib
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
nilotinib
Other Intervention Name(s)
AMN107, Tasigna
Intervention Description
All patients will receive nilotinib 300mg bid po daily. Nilotinib dose is taken every 12 hours
Primary Outcome Measure Information:
Title
Number of Treatment Failure Events up to 2 Years
Description
Treatment failure events from time of study entry in Complete molecular response-Chronic phase (CML-CP) participants with low imatinib trough concentrations less than 850 nanogram per milliliter (<850 ng/mL) treated with nilotinib as defined in European LeukemiaNet (ELN)-guideline.
Time Frame
up to 2 years
Secondary Outcome Measure Information:
Title
European LeukemiaNet (ELN)-Defined Optimal Responses
Time Frame
up to 2 years
Title
Loss of Complete Cytogenetic Response (CCyR), Major Molecular Response (MMR) and Complete Molecular Response (CMR) on Nilotinib
Description
Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as CCyR at 12 cycles if the patient met the CCyR criteria at the Cycle 12 Visit.
Major molecular response is defined as values equal or below 0.1% on the International Scale.
Complete Molecular Response is defined as a Bcr-Abl (a fusion of gene of Bcr and ABl genes) ratio ≤0.0032% on the International Scale Bcr = breakpoint cluster gene Abl = abelson proto-oncogene.
Time Frame
up to 2 years
Title
Duration of Complete Cytogenetic Response (CCyR), Major Molecular Response (MMR) and Complete Molecular Response (CMR)Achieved on Nilotinib
Description
Durations of major/complete cytogenetic response is defined as the time from the first documentation of the major/ complete response to the first documentation of the disease progression.
Time Frame
up to 2 years
Title
Event-free Survival (EFS), Progression-free Survival (PFS) and Overall Survival (OS) up to 2 Years
Description
Event-free survival was defined as the time from the date of randomization to the date of first occurrence of any of the following events on study treatment: loss of complete hematological response, confirmed loss of complete cytogenetic response (CCyR), confirmed loss of major molecular response (MMR), death from any cause during treatment, progression to the accelerated phase or blast crisis of chronic myelogenous leukemia (CML) per European Leukemia Network (ELN) criteria, whichever was earliest.
Progressions free survival is defined as time between Day 1 cycle 1 and time to first documented disease progression or death. Disease progression will be determined as per response criteria.
Overall survival time is defined as the time from the treatment start to the date of death due to any reason.
Time Frame
up to 2 years
Title
European LeukemiaNet (ELN)-Defined Suboptimal Events
Time Frame
up to 2 years
Title
Number of Participants Reported Adverse Events
Description
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. An SAE is defined as any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect, or is an event of possible drug-induced liver injury.
Time Frame
Up to 2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Cytogenetically confirmed Ph+ CML-CP Any prior dose of Imatinib
Imatinib 400 mg daily for ≥7 consecutive days prior to imatinib trough collection
Imatinib trough plasma concentration <850 ng/mL
Exclusion Criteria:
Prior documented failure events as defined by ELN guidelines:
Loss of CHR, CCyR, or clonal progression/Ph+
Less than CHR at 3 months after diagnosis
No CyR at 6 months after diagnosis
Less than PCyR at 12 months after diagnosis
Less than CCyR at 18 months after diagnosis
Prior accelerated phase or blast phase CML
Previously documented T315I mutation
Previous treatment for CML with any other tyrosine kinase inhibitor except for imatinib
Patients who had any other treatment for CML (transplant) except interferon +/- ara- C, imatinib, hydroxyurea and/or anagrelide
Impaired cardiac function
Patients receiving therapy with strong inhibitors of CYP3A4 or medications that prolong the QT interval and cannot be either discontinued or switched to a different medication prior to starting study drug.
Any other malignancy that is clinically significant or requires active intervention.
Major surgery within 4 weeks prior to Day 1 of study or who have not recovered from prior surgery
Treatment with other investigational agents within 30 days of Day 1
Women who are pregnant, breast feeding, or of childbearing potential without a negative serum test at baseline. Post-menopausal women must be amenorrhoeic for at least 12 months to be considered of non-childbearing potential. Women of childbearing potential must have a negative serum pregnancy test within 7 days of the first dose of nilotinib
Sexually active male and female patients taking nilotinib unwilling to use adequate contraception throughout the trial and 3 months following discontinuation of study drug
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Comprehensive Cancer Centers of Nevada CCC of Nevada (1)
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89109
Country
United States
Facility Name
Cancer Center of the High Plains
City
Amarillo
State/Province
Texas
ZIP/Postal Code
79106
Country
United States
Facility Name
Baylor Health Care System/Sammons Cancer Center Dept. of Sammons Cancer (2)
City
Dallas
State/Province
Texas
ZIP/Postal Code
75246
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Study of Nilotinib in Ph+ CML-CP Patients With Low Imatinib Trough Plasma Concentrations
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