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 An Extension Study to Determine the Safety and Anti-Leukemic Effects of Imatinib Mesylate in Adult Participants With Ph+ Leukemia (NCT00171249), a non-randomized phase 2 study. The primary outcome is Percentage of Participants With Hematologic Response in Accelerated Phase Chronic Myeloid/Myelogenous Leukemia.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00171249 |
| Phase | Phase 2 |
| Condition | Philadelphia Positive Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 293 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Adults with Philadelphia chromosome-positive leukemia, including people with accelerated phase chronic myeloid leukemia, lymphoid blast crisis, acute lymphoblastic leukemia, and acute myeloid leukemia. The primary endpoint was specific to participants with accelerated phase chronic myeloid/myelogenous leukemia receiving STI571 400 mg or 600 mg.
Intervention
STI571, also called imatinib mesylate in the study title, given orally once daily at 400 mg or 600 mg. Treatment was described as continuing until death, intolerable toxicity, or the investigator judged that continued treatment was no longer in the participant’s best interest.
Comparator
The record describes dose and disease groups in a non-randomized, parallel study. It does not describe a randomized control group or a like-for-like control comparison. The dose groups should not be read as randomized treatment arms.
Primary question
Among participants with accelerated phase chronic myeloid/myelogenous leukemia, what percentage had a hematologic response? Response was defined as complete hematologic remission, no evidence of leukemia, or return to chronic phase. The registry record does not report the percentage or its confidence interval.
The study title and conditions cover several forms of Philadelphia chromosome-positive leukemia, but the primary endpoint was narrower: it concerned the accelerated phase chronic myeloid/myelogenous leukemia group. The record describes the first part of the study as evaluating hematologic response and additional clinical outcomes. The extension’s stated purpose was to let participants continue study treatment and to reduce data collection to overall survival and serious adverse events.
The primary endpoint was assessed up to 3 years after treatment started. The primary analysis population is described as all enrolled participants, while data for this endpoint were collected and analyzed only for the specified accelerated phase chronic myeloid/myelogenous leukemia dose groups. The record does not report the number of participants contributing endpoint data, the response count, or the response percentage. Consequently, the clinical question can be described from the endpoint definition, but its answer cannot be read from the registry record.
3. Trial Design
This was a Phase 2, non-randomized, unmasked, parallel study sponsored by Novartis Pharmaceuticals. The registry lists enrollment of 293 participants and a completed status. The recorded start date is 1999-08-09, and the primary completion date is 2013-09-23. These dates describe the registry record and do not establish how long any individual participant received treatment.
The study included disease groups with Philadelphia chromosome-positive leukemia and described STI571 at doses of 400 mg and 600 mg. Listed groups included accelerated phase chronic myeloid/myelogenous leukemia, lymphoid blast crisis, acute lymphoblastic leukemia, and acute myeloid/myelogenous leukemia. The record does not describe random assignment; the doses and disease groups therefore do not constitute a randomized comparison. Differences between groups could reflect differences in disease group, treatment selection, or other factors not reported in the registry record.
For the accelerated phase chronic myeloid/myelogenous leukemia groups, treatment was oral and once daily. The arm descriptions state that treatment continued until death, intolerable toxicity, or the investigator’s judgment that continuing was no longer in the participant’s best interest. The extension was intended to permit access to study drug and continued treatment while narrowing data collection to overall survival and serious adverse events.
The first part’s objectives included hematologic response lasting at least 4 weeks and evaluation of response duration, overall survival, cytogenetic response, time to blast crisis, symptomatic parameters, tolerability, and safety. The registry lists secondary measures for cytogenetic response, time to response, duration of response, time to progression to blast crisis, and overall survival by disease and by dose in the specified disease group. The detailed protocol, treatment assignment process, and statistical analysis plan are not reported in the registry record.
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: On July 31, 2002, the core study was completed and 76 participants on study medication were enrolled to the extension protocol #1 (E1), and as of July 31, 2004, 42 participants were still on treatment and were enrolled in a further extension protocol #2 (E2).
Only the first 6 of 7 groups are shown.
Overall Study
| Stage | Accelerated Phase Chronic Myeloid/Myelogenous Leukemia 400 mg | Lymphoid Blast Crisis 400 mg | Acute Lymphoblastic Leukemia 400 mg | Accelerated Phase Chronic Myeloid/Myelogenous Leukemia 600 mg | Lymphoid Blast Crisis 600 mg | Acute Lymphoblastic Leukemia 600 mg |
|---|---|---|---|---|---|---|
| STARTED | 77 | 5 | 5 | 158 | 3 | 43 |
| Completed Part 1 | 50 | 0 | 1 | 125 | 2 | 6 |
| COMPLETED | 1 | 0 | 0 | 6 | 0 | 0 |
| NOT COMPLETED | 76 | 5 | 5 | 152 | 3 | 43 |
Reasons for not completing this period:
| Reason | Accelerated Phase Chronic Myeloid/Myelogenous Leukemia 400 mg | Lymphoid Blast Crisis 400 mg | Acute Lymphoblastic Leukemia 400 mg | Accelerated Phase Chronic Myeloid/Myelogenous Leukemia 600 mg | Lymphoid Blast Crisis 600 mg | Acute Lymphoblastic Leukemia 600 mg |
|---|---|---|---|---|---|---|
| Withdrawal by Subject | 2 | 0 | 0 | 6 | 0 | 0 |
| Unsatisfactory Therapeutic Effect | 44 | 3 | 5 | 70 | 3 | 31 |
| Death | 7 | 1 | 0 | 4 | 0 | 3 |
| Other | 10 | 0 | 0 | 43 | 0 | 2 |
| No Longer Requires Study Drug | 4 | 0 | 0 | 3 | 0 | 5 |
| Administrative Problems | 1 | 0 | 0 | 2 | 0 | 0 |
| Adverse Event | 3 | 1 | 0 | 15 | 0 | 2 |
| Lost to Follow-up | 5 | 0 | 0 | 9 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: ITT population consisted of all enrolled participants. The data for this outcome measure was collected and analyzed in participants who belonged to the disease group of Accelerated Phase Chronic Myeloid/Myelogenous Leukemia 400 mg or 600 mg only, as pre-specified in the protocol. The other three acute leukemia disease groups were exploratory only. Data is reported with data that is available. No additional data tables were found that could provide results with arms broken down any further.
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.
Population: Intention-to-treat (ITT) population consisted of all enrolled participants. Baseline characteristics were collected and reported together for each disease and dose groups (Accelerated Phase Chronic Myeloid/Myelogenous Leukemia 400 mg or 600 mg, Lymphoid Blast Crisis 400 mg or 600 mg, Acute Lymphoblastic Leukemia 400 mg or 600 mg, and Acute Myeloid/Myelogenous Leukemia 600 mg) of participants, as pre-specified in the protocol.
| Characteristic | Unit | STI571 |
|---|---|---|
| Participants analysed | 293 | |
| Age, Continuous | years | 54.3 (SD 12.84) |
| Sex: Female, Male: Female | Participants | 146 |
| Sex: Female, Male: Male | Participants | 147 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Hematologic Response in Accelerated Phase Chronic Myeloid/Myelogenous Leukemia, measured over Up to 3 years after start of treatment.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with a hematologic response in accelerated phase chronic myeloid/myelogenous leukemia. The response definition combines findings from peripheral blood and bone marrow measurements with assessments of extramedullary leukemic involvement at physical examination. A response could be complete hematologic remission, no evidence of leukemia, or return to chronic phase. The endpoint timeframe is up to 3 years after treatment started.
A response percentage is a binary-outcome summary: each participant is classified as having or not having a response under the stated definition, and the result describes the proportion meeting that definition in the analyzed group. In general, hematologic response endpoints can summarize whether blood and marrow disease measures meet prespecified criteria. They do not, by themselves, describe how long a response lasts or whether participants survive longer. The registry lists response duration and overall survival as separate outcomes.
The registry specifies a 95% confidence interval as the measure of uncertainty for the primary endpoint. In general, a confidence interval expresses the precision of an estimate under the statistical method used; a wider interval indicates less precision than a narrower interval. Neither the response percentage nor its interval is reported in the registry record. The participant-level response counts and the criteria for handling participants without an evaluable assessment are also not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a primary endpoint and its response definition, but it does not report the primary response estimate or its 95% confidence interval. There is therefore no posted numerical result from which to describe the observed response frequency or its precision. The endpoint description alone is not evidence about the size or direction of the outcome.
The study was non-randomized and included multiple disease groups and dose groups. Any comparison between 400 mg and 600 mg groups would not have the protection against confounding that random assignment can provide. The record does not describe a like-for-like control comparison, and its stated design does not support treating the listed groups as randomized arms. Comparisons across groups could be affected by differences in disease characteristics, eligibility, or treatment selection; how these factors were addressed is not reported in the registry record.
The primary endpoint applies only to the accelerated phase chronic myeloid/myelogenous leukemia groups at 400 mg or 600 mg, although the stated ITT population consists of all enrolled participants. This distinction matters: the overall enrollment figure is not the endpoint denominator. The registry record does not report the number analyzed for the primary endpoint or the amount of missing endpoint information. Without those details and the response result, the endpoint cannot be independently characterized from the registry record.
The record describes a 95% confidence interval, but gives no interval limits. In general, an estimate and its interval are needed together to understand both the reported response level and its uncertainty. A confidence interval is not a guarantee that the true value lies within a particular realized interval, nor does it correct for non-random allocation, missing observations, or differences between disease groups.
The registry reports 293 participants at risk for serious adverse events and 20 affected participants. These are safety summaries, not a comparison with an untreated or randomized control group. The record’s serious-event terms include several conditions and the term “Death”; the listed percentages are registry entries for those terms, not a substitute for an overall survival analysis. Overall survival is separately named as an outcome, but its numerical result is not reported in the registry record.
The study objectives and outcome list indicate that response, response duration, progression, cytogenetic response, and survival were of interest. The registry lists seven outcome measures and zero analyses. The results and analysis details needed to assess multiplicity, adjustment, or the relation between individual outcomes are not reported in the registry record. No conclusion about statistical significance can be drawn from endpoint definitions or event listings alone.
Any planning illustrations based on an observed effect should be distinguished from the study’s own design and from observed trial results. In general, sample-size illustrations describe hypothetical planning conditions; they are not observed power. The primary effect estimate required to interpret such illustrations is not reported in the registry record.
9. Statistical Methods Explained
The statistical methods that were actually used for the primary endpoint are not reported in the registry record. The endpoint is described as a percentage with a 95% confidence interval, but the record does not state the interval method, the analysis denominator, or the rules for handling missing assessments. The explanations below are general descriptions of the statistical ideas relevant to the stated endpoint, not claims about the study’s unreported analysis plan.
What does a response percentage describe?
General: A binary outcome places each participant into one of two categories, such as response or no response. A response percentage expresses the share of the analyzed group meeting the response definition. Its interpretation depends on a clear endpoint definition, a defined analysis population, and a known denominator. Here, the registry defines the response categories but does not report the response count, percentage, or denominator.
The specified response definition uses complete hematologic remission, no evidence of leukemia, or return to chronic phase. It draws on peripheral blood and bone marrow measurements and physical examination for extramedullary leukemic involvement. Because these criteria determine who is classified as a responder, operational details such as assessment timing and the handling of incomplete measurements can affect the result. Those details are not reported in the registry record.
What does a confidence interval add?
General: A confidence interval accompanies an estimate to communicate its statistical precision. A point estimate alone does not show how much uncertainty remains. The width of an interval depends on factors including the number of participants with usable data and the distribution of outcomes, as well as the method used to construct it. The registry specifies a 95% confidence interval but reports neither the interval nor the method used to calculate it. It is therefore not possible to assess the precision of the primary response estimate from the registry record.
General: A confidence interval is not a measure of clinical importance and does not account automatically for bias. In a non-randomized study, a precise estimate of a response percentage would still describe the observed group under the endpoint definition; it would not by itself establish what would have happened under a different treatment or in a control group.
Why does the non-randomized design matter?
General: Random allocation is one way to make treatment groups comparable on average with respect to measured and unmeasured factors. Without randomization, treatment groups can differ at baseline, including because different participants or disease groups receive different doses. Statistical adjustment may address some measured differences when the required data and methods are available, but it cannot ensure removal of unmeasured confounding. The registry identifies the allocation as non-randomized and does not report an adjustment strategy.
The record describes parallel groups and several disease and dose categories. Those categories are not, by their existence, evidence of a randomized head-to-head dose comparison. Disease composition and treatment selection may influence outcome patterns. The registry’s stated skip note also cautions that the arms are not a like-for-like control comparison.
What is the role of analysis populations?
General: An analysis population specifies which participants contribute to a particular estimate. The registry describes the ITT population as all enrolled participants, but says that the primary endpoint data were collected and analyzed only in the accelerated phase chronic myeloid/myelogenous leukemia 400 mg or 600 mg groups. This means the overall enrollment count should not be assumed to equal the primary endpoint denominator. The analyzed counts and handling of participants without endpoint data are not reported in the registry record.
Different analysis populations can answer different questions. A population of all enrolled participants describes the enrolled cohort, while a disease-specific endpoint population focuses on the group for which the measure was defined. Without group-specific counts and the analysis rules, it is not possible to reconstruct the denominator or evaluate how exclusions and missing observations might affect the result.
Why might over-dispersion matter?
General: Over-dispersion means that observed variability is greater than a model expects under its assumptions. It can lead to standard errors that are too small if the model does not allow for the extra variability, making confidence intervals too narrow and tests too optimistic. This concept is most relevant to certain models for counts or clustered data; the registry’s primary endpoint is a participant percentage, and no model or over-dispersion assessment is reported. There is no basis in the registry record to say whether over-dispersion was relevant to the study’s analyses.
What can planning illustrations establish?
General: A sample-size calculation is a planning exercise based on specified assumptions about an effect, significance level, allocation, and statistical method. It does not show that a study had a particular probability of finding a result after the fact. The record’s planning assumptions describe a two-sided significance level of 0.05, equal allocation, and a normal approximation, with the target effect expressed as a fraction of an observed effect. For time-to-event illustrations, the stated planning quantity is the total number of events; other illustrations use participants per arm.
These assumptions are described as planning illustrations, not as the trial’s own design. The primary observed effect and any resulting planning values are not reported in the registry record. Such illustrations should not be described as observed power or as evidence that the trial was adequately sized for its actual outcomes.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Accelerated Phase Chronic Myeloid/Myelogenous Leuk | 77 | 50 | 64.9% | 53.2% to 75.5% | 53.8% to 74.7% | 53.8% to 74.7% |
| Accelerated Phase Chronic Myeloid/Myelogenous Leuk | 158 | 118 | 74.7% | 67.2% to 81.3% | 67.4% to 80.8% | 67.4% to 80.8% |
R code and output
arm <- c("Accelerated Phase Chronic Myeloid/Myelogenous Leuk", "Accelerated Phase Chronic Myeloid/Myelogenous Leuk")
n <- c(77, 158)
events <- c(50, 118)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Accelerated Phase Chronic Myeloid/Myelogenous Leuk 77 50 64.9 53.2 75.5 53.8 74.7 53.8 74.7
ARM Accelerated Phase Chronic Myeloid/Myelogenous Leuk 158 118 74.7 67.2 81.3 67.4 80.8 67.4 80.811. Multiplicity, Missing Data and Analysis Populations
The registry lists seven outcome measures, including the primary hematologic response measure and secondary measures covering cytogenetic response, time to response, response duration, progression to blast crisis, and overall survival by disease and by dose in the accelerated phase chronic myeloid/myelogenous leukemia group. It lists zero analyses. Outcome definitions and a list of measures do not establish which hypotheses were formally tested or how results were handled across multiple outcomes.
Multiplicity arises when several outcomes, groups, time points, or statistical tests are considered. In general, conducting many tests can increase the chance of at least one apparently unusual finding even when no true effect exists. Methods such as prespecifying one primary outcome or adjusting inference can address different aspects of this issue. The registry record does not report a multiplicity strategy, hierarchy of testing, or analysis plan, so it is not possible to determine whether or how multiplicity was addressed.
The primary population is described as all enrolled participants, while primary endpoint data were collected and analyzed only in the accelerated phase chronic myeloid/myelogenous leukemia 400 mg or 600 mg groups. The registry does not report the number of participants included in that endpoint analysis, the number with missing assessments, or the rules used to classify participants lacking an assessment. Missing-data methods and sensitivity analyses are not reported in the registry record.
The extension objective narrowed data collection to overall survival and serious adverse events. How participants’ follow-up and outcome availability were handled across the study parts is not reported in the registry record. Consequently, the completeness of endpoint follow-up and the effect of missing information on the reported outcomes cannot be assessed from the registry record.
12. 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 |
|---|---|---|---|
| STI571 | 20 | 293 | 6.8% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| STI571 | 0 of 0 |
Most frequent serious adverse event terms
| Event term | Organ system | STI571 |
|---|---|---|
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 2 of 293 |
| Death | General disorders | 2 of 293 |
| Subdural haematoma | Injury, poisoning and procedural complications | 1 of 293 |
| Transfusion reaction | Injury, poisoning and procedural complications | 1 of 293 |
| Breast cancer recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 293 |
| Gastrointestinal neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 293 |
| Malignant neoplasm progression | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 293 |
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | 1 of 293 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 1 of 293 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 293 |
Serious events with the highest arm-level rate: prostate cancer (0.7%), death (0.7%), subdural haematoma (0.3%), transfusion reaction (0.3%), breast cancer recurrent (0.3%).
The registry safety summary lists 293 participants at risk for serious adverse events and 20 participants affected. It also lists zero participants at risk and zero affected in the separate non-serious event category. These entries describe the safety fields in the registry record; they do not provide event counts by treatment dose or disease group.
The listed serious-event terms include prostate cancer at 0.7%, death at 0.7%, subdural haematoma at 0.3%, transfusion reaction at 0.3%, breast cancer recurrent at 0.3%, and gastrointestinal neoplasm at 0.3%. These are term-specific percentages as recorded. A participant may have more than one event, so term percentages should not be treated as mutually exclusive categories or added together.
The term “Death” in the serious adverse event list is not an overall survival estimate. Overall survival is a separate outcome in the registry, and its result is not reported in the registry record. The safety summary also does not establish whether an event was caused by treatment. Attribution details, event timing, severity grades, and dose-specific safety results are not reported in the registry record.
13. What the Result Does, and Does Not, Mean
The registry record establishes that the study was a completed, non-randomized Phase 2 study of STI571 in adults with Philadelphia chromosome-positive leukemia, with 293 participants enrolled. It describes a primary hematologic response endpoint for accelerated phase chronic myeloid/myelogenous leukemia and lists additional response, progression, and survival outcomes. It also provides a summary of serious adverse events.
However, the record does not report the primary response percentage, its 95% confidence interval, or numerical results for the listed efficacy outcomes. The endpoint definition therefore explains what was intended to be measured, but it does not show how many participants responded or how precise an estimate was. The registry record also does not provide the information needed to judge the completeness of endpoint data or the details of the statistical analyses.
The non-randomized design means that dose and disease groups should not be interpreted as randomized treatment comparisons. The registry does not describe a like-for-like control group. An outcome observed in a treated cohort, even if a numerical estimate were available, would not by itself establish a causal difference from an alternative treatment or no treatment.
The serious adverse event figures describe recorded safety reporting, not comparative risk or treatment causation. The listed “Death” term is not a substitute for the separately named overall survival outcome. Overall survival results are not reported in the registry record.
Accordingly, the record supports a description of the study’s objectives, populations, endpoint definitions, and safety summary, but not a conclusion about the magnitude or precision of the primary response result. It also does not support claims about statistical significance, comparative efficacy, or the causal effect of STI571.
14. Why This Trial Matters Statistically
This study illustrates why an endpoint definition and a result are different kinds of information. The registry describes hematologic response using clinical and laboratory criteria and specifies a confidence interval, but does not report the response estimate or interval. Without the result, readers cannot assess the observed response frequency or its statistical precision.
It also highlights the importance of matching the analysis population to the clinical question. Although the registry lists 293 enrolled participants and describes an ITT population, the primary endpoint data were collected and analyzed in specified accelerated phase chronic myeloid/myelogenous leukemia dose groups. Overall enrollment should not be mistaken for the primary endpoint denominator.
The non-randomized design is another central statistical feature. Disease groups and doses appear in parallel categories, but the registry does not describe randomized allocation or a like-for-like control comparison. Any comparison among those groups would need careful interpretation because group differences may reflect more than dose.
Finally, the study distinguishes early objectives from the extension’s narrower data collection objective. The first part included response and other clinical outcomes, while the extension focused on access to study drug, overall survival, and serious adverse events. This makes clarity about study parts, outcome timeframes, and available results essential when interpreting a long-running study record. The posted safety summary supplies some event information, but it does not replace efficacy estimates or survival results.
15. 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 statistical interpretation is that the registry record does not report the primary outcome result. It gives the hematologic response definition and a 95% confidence interval as the dispersion measure, but no estimate, interval limits, or endpoint denominator. The listed secondary outcomes likewise have no numerical results in the registry record.
The study was non-randomized and did not describe a like-for-like control comparison. This limits causal interpretation of differences between groups, especially because the record includes multiple leukemia categories and dose groups. The registry does not report an adjustment strategy or enough group-specific information to assess comparability.
Although the primary population is described as all enrolled participants, primary endpoint data were collected and analyzed only in specified accelerated phase chronic myeloid/myelogenous leukemia groups. The number of participants in those groups, the number assessed, and the handling of missing assessments are not reported in the registry record. This prevents evaluation of endpoint completeness and the impact of missing data.
The registry lists seven outcome measures and zero analyses, but does not report the detailed statistical analysis plan, multiplicity handling, interim analyses, or sample-size justification. These methods are not reported in the registry record. The planning assumptions described for illustrations should not be confused with the trial’s design or with observed power.
The safety summary is aggregate and does not provide dose-specific event results, event timing, or attribution details. The listed serious adverse event terms cannot establish causation, and the “Death” term does not provide an overall survival estimate. Numerical overall survival results are not reported in the registry record.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: An Extension Study to Determine the Safety and Anti-Leukemic Effects of Imatinib Mesylate in Adult Participants With Ph+ Leukemia — NCT00171249.
- Registry record last updated 2021-07-22; status last verified 2021-07.
- Results first posted 2021-07-22.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
The registry record describes NCT00171249 as a completed Phase 2, non-randomized, unmasked, parallel study sponsored by Novartis Pharmaceuticals. It lists enrollment of 293 participants, a start date of 1999-08-09, and a primary completion date of 2013-09-23. The study concerned adults with Philadelphia chromosome-positive leukemia and STI571 at 400 mg or 600 mg.
The primary endpoint was the percentage with hematologic response in accelerated phase chronic myeloid/myelogenous leukemia, assessed up to 3 years after treatment started. Response included complete hematologic remission, no evidence of leukemia, or return to chronic phase. The primary estimate and its 95% confidence interval are not reported in the registry record.
The registry lists seven outcome measures and zero analyses. Its safety summary reports 20 participants affected by serious adverse events among 293 at risk. Detailed statistical methods, missing-data handling, and numerical efficacy and survival results are not reported in the registry record.