Trial Outcomes & Findings for Study of Nilotinib in Ph+ CML-CP Patients With Low Imatinib Trough Plasma Concentrations (NCT NCT01131325)
NCT ID: NCT01131325
Last Updated: 2021-05-20
Results Overview
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.
TERMINATED
PHASE4
3 participants
up to 2 years
2021-05-20
Participant Flow
The study was conducted at 3 centers in the United States.
A total of 3 participants were enrolled in the study, of which 1 discontinued the study due to Adverse Event (AE) and 2 participants discontinued as the study got terminated.
Participant milestones
| Measure |
Nilotinib
Participants received nilotinib 300 milligram (mg) twice daily (bid) through the mouth (po), every 12 hours in a continuous 28-day cycle up to 2 years.
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Nilotinib
Participants received nilotinib 300 milligram (mg) twice daily (bid) through the mouth (po), every 12 hours in a continuous 28-day cycle up to 2 years.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
The participants discontinued the study as the study got terminated
|
2
|
Baseline Characteristics
Study of Nilotinib in Ph+ CML-CP Patients With Low Imatinib Trough Plasma Concentrations
Baseline characteristics by cohort
| Measure |
Nilotinib
n=3 Participants
Participants received nilotinib 300 mg, BID po, every 12 hours in continuous 28-day cycle up to 2 years.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to 2 yearsPopulation: The overall number of participants considered for analysis was "0" due to the premature termination of the study and low enrollment; The efficacy and quality of life assessments were not collected and reported in the study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 2 yearsPopulation: The overall number of participants considered for analysis was "0" due to the premature termination of the study and low enrollment; The efficacy and quality of life assessments were not collected and reported in the study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 2 yearsPopulation: The overall number of participants considered for analysis was "0" due to the premature termination of the study and low enrollment; The efficacy and quality of life assessments were not collected and reported in the study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 2 yearsPopulation: The overall number of participants considered for analysis was "0" due to the premature termination of the study and low enrollment; The efficacy and quality of life assessments were not collected and reported in the study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 2 yearsPopulation: The overall number of participants considered for analysis was "0" due to the premature termination of the study and low enrollment; The efficacy and quality of life assessments were not collected and reported in the study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 2 yearsPopulation: The overall number of participants considered for analysis was "0" due to the premature termination of the study and low enrollment; The efficacy and quality of life assessments were not collected and reported in the study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 2 yearsAn 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.
Outcome measures
| Measure |
Nilotinib
n=3 Participants
Participants received nilotinib 300 mg, BID po, every 12 hours in continuous 28-day cycle up to 2 years.
|
|---|---|
|
Number of Participants Reported Adverse Events
|
2 Participants
|
Adverse Events
Nilotinib
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Nilotinib
n=3 participants at risk
Participants received nilotinib 300 mg, BID po, every 12 hours in continuous 28-day cycle up to 2 years.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Muscle cramps - legs
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Blood and lymphatic system disorders
Neutropenia
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Gastrointestinal disorders
Nausea
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
General disorders
Fatigue
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Skin and subcutaneous tissue disorders
Pruritus Genital
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
Infections and infestations
Sore Throat
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
|
General disorders
Mild sweats
|
33.3%
1/3 • up to 2 years
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical examination or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place