This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Nilotinib Versus Standard Imatinib (400/600 mg Every Day (QD)) Comparing the Kinetics of Complete Molecular Response for Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) Pts With Evidence of Persistent Leukemia by Real-time Quantitative Polymerase Chain Reaction (RQ-PCR) (NCT00760877), a randomized phase 3 study. The primary outcome is Rate of Confirmed Best Cumulative Complete Molecular Response (CMR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00760877 |
| Phase | Phase 3 |
| Condition | CHRONIC MYELOGENOUS LEUKEMIA |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 207 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Participants with chronic-phase chronic myelogenous leukemia and evidence of persistent leukemia by real-time quantitative polymerase chain reaction. The primary analysis set included all randomized participants.
Intervention
Nilotinib 400 mg orally twice daily for 48 months.
Comparator
Imatinib 400 mg or 600 mg once daily for 48 months, based on the participant’s dose before randomization.
Primary question
What was the rate of confirmed best cumulative complete molecular response during the first 12 months of treatment after randomization?
The study compared two treatment strategies in participants with chronic-phase disease who had evidence of persistent leukemia on molecular testing. Its primary question concerned whether participants achieved a confirmed complete molecular response in the first 12 months after randomization. This is a response-rate question: each participant was classified as a responder or non-responder under the endpoint definition.
The registry describes a broader interest in the kinetics of complete molecular response and in the relationship between response and patient outcomes, including progression-free survival, event-free survival, and overall survival. It also lists crossover and survival outcomes as secondary outcomes. Those aims do not change the primary endpoint’s defined time window or responder classification. The registry record does not report numerical results for the primary endpoint, so it does not permit a quantitative comparison of the response rates from the information recorded.
The study was randomized and parallel-group, but it was not masked. Randomization supports comparison of assigned groups, while open-label conduct means participants and study personnel knew the assigned treatment. Whether that knowledge affected testing, follow-up, or outcome assessment is not reported in the registry record. The endpoint relied on laboratory results and confirmation on a subsequent sample, but the record does not describe blinding of those assessing the tests.
3. Trial Design
This was a phase 3, randomized, open-label, parallel-group study of nilotinib versus standard imatinib in chronic-phase chronic myelogenous leukemia. The registry lists an enrollment of 207. The study is recorded as completed, with a start date of 2009-06 and primary completion in 2015-07. The registry record does not report the date of final study completion.
Participants assigned to nilotinib received 400 mg orally twice daily for 48 months. Those assigned to imatinib received 400 mg or 600 mg once daily for 48 months, with the dose determined by the participant’s dose before randomization. Because the assigned regimens differed in both drug and dosing schedule, the randomized comparison concerns these treatment strategies as recorded, rather than an isolated comparison of dosing frequency.
The primary outcome was assessed over the first 12 months after randomization, despite the planned treatment period being 48 months. The study description also identifies crossover and longer-term outcomes as interests. The secondary outcomes listed include confirmed best cumulative complete molecular response, the number of crossover participants with complete molecular response, progression-free survival, event-free survival, and overall survival. The registry record does not explain the timing or rules governing crossover.
The primary analysis population was the intent-to-treat set, defined in the record as all randomized participants. This means that the stated analysis population was based on random assignment, not on completing treatment. The record does not report the number randomized to each primary comparison group, the allocation ratio, or a detailed account of participants’ treatment exposure. It also does not report stratification factors, site structure, or procedures used to maintain allocation concealment.
The study’s stated primary goal was to determine the rate of confirmed best cumulative complete molecular response within the first year of study therapy. It also explored the impact of achieved response on patient outcomes and characterized response kinetics, including after crossover. These aims identify what the study set out to examine; they do not establish that a particular response or outcome was observed.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: Participants were randomized 1:1. Participants in the imatinib arm were permitted to cross-over to nilotinib after 2 years on study if complete molecular response (CMR) was not achieved, or at any time during the study if participants experienced treatment failure, had confirmed loss of major molecular response (MMR) or had confirmed loss of CMR.
Overall Study
| Stage | Nilotinib | Imatinib |
|---|---|---|
| STARTED | 104 | 103 |
| Cross-over to Nilotinib | 0 | 46 |
| Safety Set | 101 | 103 |
| COMPLETED | 59 | 77 |
| NOT COMPLETED | 45 | 26 |
Reasons for not completing this period:
| Reason | Nilotinib | Imatinib |
|---|---|---|
| Protocol Violation | 1 | 0 |
| Non-compliance with protocol treatment | 2 | 1 |
| Pregnancy | 1 | 4 |
| Death | 2 | 1 |
| Withdrawal by Subject | 7 | 3 |
| Adverse Event | 19 | 12 |
| Participant diagnosed with AML | 0 | 1 |
| Participant not eligible to cross-over | 0 | 1 |
| Physician Decision | 1 | 2 |
| Participants transferred to AMN107A2408 | 11 | 1 |
| Treatment failure | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: The intent-to-treat analysis set, which included all randomized participants, was analyzed.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Nilotinib | Imatinib | Total |
|---|---|---|---|---|
| Participants analysed | 104 | 103 | 207 | |
| Age, Continuous | Years | 48.3 (SD 13.26) | 49.9 (SD 13.07) | 49.1 (SD 13.16) |
| Sex: Female, Male: Female | Participants | 33 | 38 | 71 |
| Sex: Female, Male: Male | Participants | 71 | 65 | 136 |
6. Primary Endpoint
The registry lists the primary outcome as Rate of Confirmed Best Cumulative Complete Molecular Response (CMR), measured over 12 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the rate of confirmed best cumulative complete molecular response during the first 12 months of treatment after randomization. Participants with a confirmed response in that interval were classified as responders. Participants who dropped out early or did not provide sufficient data for any reason were classified as non-responders. The analysis set included all randomized participants.
Complete molecular response was defined using real-time quantitative polymerase chain reaction testing for BCR-ABL. The stated threshold was an undetectable result with a BCR-ABL ratio of ≤ < 0.001 on the international scale. The test also had to have sensitivity of at least 4.5 logs below the standardized baseline. A negative result had to be confirmed on the next RQ-PCR sample, usually about 3 months later, and the response date was assigned to the first of the two negative results with sensitivity greater than 4.5 logs.
Using a binary response endpoint gives a clear classification for comparing the proportions of participants who met a defined molecular criterion within a specified period. Requiring confirmation reduces reliance on a single negative test. The trade-off is that response classification depends on availability and timing of repeated measurements; the endpoint’s rule treats early dropout or insufficient data as non-response.
General statistical context: binary endpoints are commonly summarized by the number of participants meeting the definition and the corresponding proportion in each group. A confidence interval, when reported, would describe the precision of an estimated response rate or between-group contrast. The registry record does not report primary endpoint counts, response proportions, a treatment-effect estimate, or a confidence interval.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a randomized comparison and defines the primary response endpoint, but it does not report the primary response counts or a numerical comparison between nilotinib and imatinib. Accordingly, the record does not establish the size or direction of any difference in confirmed complete molecular response. No response percentage, effect estimate, confidence interval, or p-value for the primary endpoint is reported in the registry record.
The outcome definition is important to interpretation. A participant counted as a responder had to meet the molecular threshold and have a subsequent confirmatory negative test within the relevant response process. A single negative test was not sufficient. Conversely, early dropout or insufficient data resulted in a non-responder classification, even if the reason for missing data was not related to molecular response. The response rate therefore represents the endpoint’s combined biological and data-availability rule, not simply the proportion with an isolated negative test.
The randomized design is relevant because, in general, randomization is intended to make treatment groups comparable at the start of a trial with respect to measured and unmeasured characteristics. That rationale does not remove the need to inspect the reported analysis, follow-up, and outcome data. Those details are not reported for the primary comparison. The study was open-label, and the registry record does not describe whether laboratory assessment or response confirmation was performed without knowledge of assigned treatment.
The endpoint’s 12-month window answers a specific early-response question. It does not by itself describe response kinetics across the full 48-month treatment period, nor does it establish later progression-free, event-free, or overall survival. Those outcomes appear among the study’s secondary outcomes, but their numerical results are not reported in the registry record. The study description’s interest in the relationship between response and later outcomes should not be read as evidence that such a relationship was demonstrated.
The registry also reports safety counts for serious and other adverse events, including a crossover subgroup. These are separate from the primary molecular-response endpoint. They do not substitute for response results and should not be combined across groups or interpreted as a randomized comparison of treatment effects without details about the populations, follow-up, and analysis. The listed adverse-event counts describe participants with events as recorded; the registry record does not provide exposure-adjusted rates or a full account of event timing.
General statistical context: an estimate without a confidence interval can convey the observed result but not its precision. A p-value, if available, would address compatibility with a specified statistical null model under the analysis assumptions; it would not quantify clinical importance. Neither such inferential quantity nor the numerical primary effect estimate is reported. The safest reading is therefore limited to the study design, endpoint definition, analysis population, and recorded safety counts—not a conclusion about comparative efficacy.
9. Statistical Methods Explained
The registry records a randomized, parallel-group design with no masking. Randomization assigns participants to treatment groups using a chance-based process. In general, this helps limit systematic differences between groups at baseline, although the quality of a comparison also depends on allocation procedures, follow-up, outcome measurement, and analysis. The registry record does not report the randomization sequence method, allocation concealment, or any stratification factors.
What does the primary endpoint count?
The endpoint is binary at the participant level: a participant either met the confirmed complete molecular response definition during the first 12 months after randomization or did not. The endpoint specifies both a molecular threshold and confirmation on the next RQ-PCR sample. The registry defines the relevant response as undetectable BCR-ABL with a ratio of ≤ < 0.001 on the international scale, a test sensitivity of at least 4.5 logs below the standardized baseline, and confirmation of RQ-PCR negativity on the next sample, usually about 3 months later. The date of confirmed response is the date of the first of two qualifying negative results, with sensitivity greater than 4.5 logs.
For a binary outcome, common summaries include a count and proportion in each group, followed by a between-group contrast such as a difference in proportions or a ratio. These approaches answer related but distinct questions. A count shows how many participants met the definition; a proportion accounts for the group’s analysis denominator; a contrast quantifies the relative or absolute separation between groups. The registry record does not report the primary endpoint counts, denominators by randomized group, or a selected contrast.
What does the intent-to-treat population mean?
The record states that the intent-to-treat analysis set included all randomized participants. In general, an intent-to-treat approach preserves the randomized assignment as the basis for comparison, even if participants stop treatment or do not follow the assigned regimen. This can help avoid bias that may arise if analysis is restricted to participants who remain on treatment or have complete follow-up. Here, however, the endpoint specifies that early dropout or insufficient data is classified as non-response. The registry record does not provide further details on how other missing or unconfirmed measurements were handled.
That endpoint rule is not the same as an analysis that excludes every participant with missing information. Instead, the rule explicitly places early dropouts and participants lacking sufficient data in the non-responder category. General statistical context: this kind of classification can be conservative in some settings, but its impact depends on why data are missing and how missingness differs between groups. The registry record does not report the amount, timing, or reasons for missing endpoint data, so their influence on the comparison cannot be assessed.
How should precision and significance be understood?
General statistical context: an estimate describes the magnitude of an observed result, while a confidence interval describes the uncertainty around that estimate under a statistical model. A wider interval indicates less precision than a narrower interval. A p-value summarizes how incompatible the data are with a specified null model, subject to the analysis assumptions; it is not the probability that a treatment is effective or ineffective. No primary endpoint estimate, confidence interval, or p-value is reported in the registry record.
The study is described as phase 3 and randomized, but the registry record does not report the primary endpoint’s statistical test, model, significance threshold, covariate adjustment, or a prespecified hypothesis. It also does not report whether interim analyses were conducted or whether any statistical monitoring boundaries were used. These methods cannot be inferred from the study phase or from the outcome definition.
What do the planning assumptions represent?
General statistical context: sample-size calculations are planning tools. They explore how many participants may be needed under stated assumptions about an effect, allocation, significance level, and statistical approximation. A calculation based on an observed effect is an illustration rather than proof of the trial’s original design assumptions. The recorded planning illustration uses a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation; it considers effects defined as a stated fraction of the observed effect. Those assumptions do not establish the trial’s actual sample-size justification or original planning basis. The registry record does not report that justification.
Such planning calculations are not observed power. Observed power is not a substitute for interpreting an effect estimate and its uncertainty after a study. A planning illustration also depends on the effect chosen as its reference and on assumptions about the test and allocation. Since no numerical primary effect is reported in the registry record, the value and precision of any resulting planning scenario cannot be evaluated from the record.
The registry lists six outcome measures and one analysis, but does not give a detailed statistical analysis plan. General statistical context: when several outcomes or analyses are tested, the chance of at least one false-positive finding can increase if each test is interpreted without regard to multiplicity. Methods for addressing multiplicity depend on which outcomes are confirmatory, the testing sequence, and the intended error control. None of those details is reported for this trial’s outcomes.
Finally, the 12-month response endpoint and the survival outcomes have different statistical structures. A binary response classification records whether a defined event occurred within a window. Time-to-event outcomes use information about event timing and censoring. General statistical context: time-to-event methods can account for participants whose event has not been observed by the end of follow-up, subject to assumptions about censoring. The registry record lists progression-free survival, event-free survival, and overall survival, but does not report their estimates, analysis methods, censoring rules, or follow-up distributions.
10. Multiplicity, Missing Data and Analysis Populations
The registry identifies a primary endpoint and lists several secondary outcomes, including confirmed best cumulative complete molecular response, crossover participants with complete molecular response, progression-free survival, event-free survival, and overall survival. It records six outcome measures and one analysis. The registry record does not report a multiplicity strategy, a hierarchy or testing sequence, or which outcomes were intended to support confirmatory inference. Without those details, the degree to which the reported analyses account for multiple outcomes cannot be assessed.
General statistical context: testing multiple outcomes can increase the probability of at least one apparently notable result under a collection of null hypotheses. Adjustment or a prespecified hierarchy can manage that issue, but the appropriate approach depends on the trial’s objectives and analysis plan. No specific correction or family-wise error procedure is reported in the registry record. It would be inappropriate to assume either that multiplicity was controlled or that every outcome was tested independently.
For the primary endpoint, early dropout and insufficient data for any reason were classified as non-response. This rule is explicitly part of the endpoint definition and should be considered when interpreting a response rate. The record does not report how many participants fell into these categories, why their data were insufficient, or whether missingness differed by randomized group. It also does not describe sensitivity analyses using alternative assumptions about missing outcomes.
The primary analysis population was the intent-to-treat set, defined as all randomized participants. The registry record does not provide detailed participant flow, exclusions, or the endpoint denominator in each randomized group. The safety data separately include an imatinib subgroup that crossed over to nilotinib; that subgroup should not be treated as a separate randomized arm for the primary comparison. The record does not describe crossover timing or how crossover was handled in analyses of later outcomes.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Nilotinib | 21 | 101 | 20.8% |
| Imatinib | 16 | 103 | 15.5% |
| Imatinib Subset That Crossed Over to Nilotinib | 8 | 46 | 17.4% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Nilotinib | 99 of 101 |
| Imatinib | 79 of 103 |
| Imatinib Subset That Crossed Over to Nilotinib | 39 of 46 |
Most frequent serious adverse event terms
| Event term | Organ system | Nilotinib | Imatinib | Imatinib Subset That Crossed Over to Nilotinib |
|---|---|---|---|---|
| ATRIAL FIBRILLATION | Cardiac disorders | 1 of 101 | 2 of 103 | 0 of 46 |
| ACUTE CORONARY SYNDROME | Cardiac disorders | 1 of 101 | 0 of 103 | 1 of 46 |
| CHOLELITHIASIS | Hepatobiliary disorders | 1 of 101 | 1 of 103 | 0 of 46 |
| CELLULITIS | Infections and infestations | 0 of 101 | 2 of 103 | 0 of 46 |
| PNEUMONIA | Infections and infestations | 0 of 101 | 1 of 103 | 1 of 46 |
| PROSTATE CANCER | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 101 | 2 of 103 | 0 of 46 |
| CEREBROVASCULAR ACCIDENT | Nervous system disorders | 1 of 101 | 0 of 103 | 1 of 46 |
| NEPHROLITHIASIS | Renal and urinary disorders | 0 of 101 | 2 of 103 | 0 of 46 |
| PLEURAL EFFUSION | Respiratory, thoracic and mediastinal disorders | 0 of 101 | 2 of 103 | 0 of 46 |
| ACUTE MYOCARDIAL INFARCTION | Cardiac disorders | 1 of 101 | 0 of 103 | 0 of 46 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Nilotinib | Imatinib | Imatinib Subset That Crossed Over to Nilotinib |
|---|---|---|---|---|
| HEADACHE | Nervous system disorders | 43 of 101 | 13 of 103 | 8 of 46 |
| RASH | Skin and subcutaneous tissue disorders | 30 of 101 | 4 of 103 | 9 of 46 |
| NAUSEA | Gastrointestinal disorders | 22 of 101 | 16 of 103 | 4 of 46 |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | 20 of 101 | 14 of 103 | 7 of 46 |
| LIPASE INCREASED | Investigations | 19 of 101 | 13 of 103 | 7 of 46 |
| DIARRHOEA | Gastrointestinal disorders | 14 of 101 | 19 of 103 | 4 of 46 |
| MUSCLE SPASMS | Musculoskeletal and connective tissue disorders | 15 of 101 | 18 of 103 | 1 of 46 |
| ALANINE AMINOTRANSFERASE INCREASED | Investigations | 19 of 101 | 5 of 103 | 8 of 46 |
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | 16 of 101 | 10 of 103 | 6 of 46 |
| PRURITUS | Skin and subcutaneous tissue disorders | 30 of 101 | 0 of 103 | 2 of 46 |
Serious events with the highest arm-level rate: acute coronary syndrome (2.2%), atrial flutter (2.2%), anal fissure (2.2%), pyrexia (2.2%), cholecystitis (2.2%).
The registry reports serious adverse events and other adverse events as counts of participants with an event, alongside the number at risk for each listed group. For nilotinib, the serious-event figures are 21 of 101 participants, and other-event figures are 99 of 101. For imatinib, the corresponding figures are 16 of 103 and 79 of 103. The separate imatinib subgroup that crossed over to nilotinib has figures of 8 of 46 for serious events and 39 of 46 for other events.
These are participant counts, not event totals: the registry’s outcome description identifies the measure as participants with an event. A participant may have experienced more than one event, but the counts do not indicate how many events occurred or when they occurred. The crossover subgroup is a subset described separately in the safety record; it should not be combined with the imatinib comparison group or regarded as an independently randomized arm.
The registry lists acute coronary syndrome, atrial flutter, anal fissure, pyrexia, cholecystitis, and pneumonia among the top serious adverse-event terms, each reported as 2.2%. The record does not give further detail about event duration, severity grading, attribution, or timing. Counts alone do not establish that treatment caused an event or support a complete safety comparison. Exposure-adjusted rates and detailed follow-up information are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a randomized, open-label study comparing nilotinib with imatinib in chronic-phase chronic myelogenous leukemia, and it defines a confirmed molecular-response endpoint within the first 12 months. It also specifies that all randomized participants were included in the primary analysis set and that early dropout or insufficient data counted as non-response. These details clarify what the intended comparison meant.
They do not establish the comparative result. The registry record does not report how many participants in either randomized group achieved confirmed complete molecular response, nor does it report response proportions, an effect estimate, a confidence interval, or a p-value. The direction, magnitude, and precision of the primary comparison therefore cannot be determined from the recorded results.
Even if a response proportion were available, it would answer a defined, time-limited question: whether the participant met the molecular criterion and confirmation rule during the first 12 months. It would not, by itself, establish a difference in response kinetics across the full treatment period or an effect on progression-free, event-free, or overall survival. Those outcomes are listed as secondary outcomes, but numerical results and analysis details are not reported in the registry record.
General statistical context: statistical significance and clinical importance are different considerations. An estimate’s magnitude and uncertainty are both relevant, and interpretation also depends on the outcome’s meaning, follow-up, and the trial’s analysis plan. Here, the numerical result and its precision are not available in the registry record, so neither statistical significance nor clinical importance can be judged from the primary endpoint description alone.
The safety figures provide counts of participants with recorded events in the listed groups, including a crossover subgroup. They are not a substitute for the missing primary outcome results and do not, by themselves, establish causal effects or a complete comparative safety profile. The study’s design and endpoint definition describe how a comparison was intended to be made; they should not be mistaken for evidence of a particular treatment outcome.
13. Why This Trial Matters Statistically
This trial is statistically notable for the way it operationalizes a molecular response. The primary endpoint is not simply a single negative RQ-PCR result: it requires a stated molecular threshold, a specified test sensitivity, and confirmation on the next sample. That structure makes the responder definition more explicit, while also making response classification depend on repeat testing and adequate data.
The endpoint’s treatment of early dropout and insufficient data is also important. Those participants were classified as non-responders, and the stated primary analysis population was all randomized participants. This connects the outcome definition to the analysis population and makes transparent that the response rate was intended to include more than participants with a complete treatment course. The registry record does not report how often this rule affected classification or whether the issue differed between groups.
The trial’s timing illustrates the distinction between an early binary response and longer-term outcomes. The primary measure covers the first 12 months, while the assigned treatment regimens were described for 48 months. The registry also lists response, crossover, and survival outcomes. In general, these outcomes address different questions and can require different analysis methods; the primary response endpoint cannot stand in for a time-to-event outcome.
Finally, the registry contains enough design information to identify a randomized, open-label comparison and define the primary endpoint, but it does not report the numerical primary results or a detailed statistical analysis plan. This is a useful distinction for statistical reading: a clear outcome definition and design description do not alone supply the effect estimate or its precision. The record therefore supports discussion of endpoint construction and analysis considerations, but not a numerical conclusion about the treatment comparison.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation for interpreting the primary comparison is that the registry record does not report the response counts, group-specific rates, effect estimate, confidence interval, or p-value. Without these results, the direction, magnitude, and precision of the comparison are unknown. The listed enrollment does not replace the missing group denominators or outcome data.
The endpoint’s non-responder rule includes participants who dropped out early or lacked sufficient data for any reason. The registry record does not report the number of participants affected, the reasons for missing data, or whether missingness differed between groups. It also does not report sensitivity analyses that might show how alternative assumptions would affect the result.
The study was open-label, but the record does not describe whether laboratory testing or response confirmation was blinded to assigned treatment. It also does not report details of randomization procedures, allocation concealment, or the statistical model used for the primary comparison. The registry record does not provide the original sample-size justification, interim-analysis plan, or a detailed approach to multiplicity.
The trial description includes crossover and longer-term outcomes, but crossover timing and analytic handling are not reported. Numerical results and methods for the listed survival outcomes are also not reported in the registry record. These gaps limit evaluation of response kinetics and longer-term outcomes separately from the primary early response question.
The safety record gives participant counts with serious and other adverse events for the listed groups, including a crossover subgroup. It does not provide exposure-adjusted rates, event timing, detailed attribution, or sufficient context for a full comparative safety assessment. The crossover subgroup is not an independent randomized comparison group.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Nilotinib Versus Standard Imatinib (400/600 mg Every Day (QD)) Comparing the Kinetics of Complete Molecular Response for Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) Pts With Evidence of Persistent Leukemia by Real-time Quantitative Polymerase Chain Reaction (RQ-PCR) — NCT00760877.
- Registry record last updated 2016-11-08; status last verified 2016-10.
- Results first posted 2016-08-16.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT00760877 was a completed, phase 3, randomized, open-label, parallel-group study sponsored by Novartis Pharmaceuticals. It enrolled 207 participants with chronic-phase chronic myelogenous leukemia and evidence of persistent leukemia by RQ-PCR. The recorded study start was 2009-06, and primary completion was 2015-07.
Participants received nilotinib 400 mg twice daily or imatinib 400 mg or 600 mg once daily for 48 months; the imatinib dose was based on the participant’s dose before randomization. The primary endpoint was confirmed best cumulative complete molecular response during the first 12 months after randomization. The primary analysis population was all randomized participants, with early dropout or insufficient data classified as non-response.
The registry record lists crossover and survival outcomes as secondary measures and reports participant counts for serious and other adverse events. It does not report numerical primary response results, comparative effect estimates, or their precision. The design and endpoint definition can be described, but the primary treatment comparison cannot be quantified from the registry record.