Chronic Myelogenous Leukemia or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemic Study: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Chronic Myelogenous Leukemia or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemic Study (NCT00978731), a na phase 1 study. The primary outcome is Number of Participants Who Died, Experienced Other Serious Adverse Events (SAEs), Adverse Events (AEs) and AEs Leading to Study Drug Discontinuation..

2. Clinical Question

The clinical question is therefore about long-term safety and tolerability during continued dasatinib exposure in a selected group previously treated on another protocol, rather than a comparison of dasatinib with another treatment. The record also names secondary outcomes concerning hematologic and cytogenetic responses, progression-free survival, and overall survival. Their names indicate which outcomes were of interest, but the registry entries available for this account do not include their result values. The registry record does not report a comparative estimate of benefit or harm against an untreated or alternative-treatment group.

3. Trial Design

This is a completed Phase 1 study with a single-group model, no masking, and allocation recorded as NA. The registered intervention is dasatinib. The official title describes long-term safety and efficacy among people with chronic myelogenous leukemia or Philadelphia chromosome positive acute lymphoblastic leukemia who experienced clinical benefit on protocol CA180-002. The brief summary emphasizes long-term safety and tolerability of dasatinib exposure in participants previously treated in CA180-002.

The registry lists enrollment of 46. It records a start date of 2005-12 and primary completion in 2008-09, with status completed. The primary-outcome population is described as all treated participants; the registry record does not report the treated population count in the registry record. The primary-outcome timeframe begins at study start and extends to up to 30 days after study participation ends. Median duration of exposure, described as on-study time, was 23.4 months.

Adverse-event results are divided into categories for accelerated-phase CML, chronic myelogenous leukemia with QD dosing, CML with BID dosing, and myeloid blast-phase CML. These are descriptive categories within the study, not randomized arms or like-for-like control groups. The record does not report a randomized allocation process or a masking procedure; masking is listed as none. Because there is no concurrent comparator, the design describes events observed during exposure but does not isolate the effect of dasatinib from the underlying disease, prior treatment, or other influences.

The registry lists 12 outcome measures and no analyses. A protocol-level statistical plan, sample-size justification, interim analyses, and detailed analysis methods are not reported in the registry record. The registry entries do not establish whether additional analyses were conducted outside the registry.

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.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All treated participants.

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.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants Who Died, Experienced Other Serious Adverse Events (SAEs), Adverse Events (AEs) and AEs Leading to Study Drug Discontinuation., measured over From start of study until up to 30 days after end of study participation. Median duration of exposure (on-study time) was 23.4 months..

The primary endpoint is a count of participants who died, experienced other serious adverse events, experienced adverse events, or discontinued study drug because of adverse events. It combines several safety-related summaries rather than expressing a treatment effect relative to a control. The record defines an adverse event as a new untoward medical occurrence or worsening of a pre-existing medical condition, whether or not it is considered related to study drug. A serious adverse event is defined by consequences including death, life threat, persistent or significant disability or incapacity, inpatient hospitalization or its prolongation, congenital anomaly or birth defect, or overdose.

Counting participants with these events is a way to describe how many people in the treated population had events during the specified follow-up. Such counts can help characterize the safety experience, while the definitions clarify which events qualify. They do not by themselves show how often events would have occurred without the study drug, nor do they establish that the drug caused an event. The registry also notes that study discontinuations due to adverse events were recorded and that these data differ from Participant Flow because the information came from separate case-report-form pages and was not reconciled.

The endpoint is appropriate to the stated safety and tolerability objective because it focuses on deaths, serious events, any events, and event-related discontinuation. However, numerical primary-endpoint results are not reported in the registry record. Without those values, the endpoint definition can be described, but its observed results cannot be quantified from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry presents this study as single-group and lists no analyses. That design description matters for interpretation: there is no randomized comparison to estimate how outcomes differ between dasatinib and a control treatment. The adverse-event categories are useful descriptions of the participants represented in each category, but they cannot substitute for a randomized comparison. General statistical interpretation of a single-group safety record is therefore descriptive: it concerns the events recorded among participants in the study, not a causal contrast.

What do participant counts convey?

A count of participants with an event indicates how many people in a defined group were recorded as having that event. The registry gives both the number at risk and the number affected for serious adverse events and other adverse events in the listed categories. These counts retain a direct connection to the participants, but do not on their own describe the timing of events, repeated events in the same person, or the amount of observation time for each person. The record’s median exposure duration of 23.4 months gives a summary of on-study time, but it does not replace participant-level follow-up information.

Counts can be helpful for describing the observed safety experience, particularly when paired with clear event definitions and a stated population. Here, the endpoint description says the primary population is all treated participants, while the adverse-event results are broken out by disease and dosing categories. The registry entries do not provide a reconciliation between those categories and the full primary-outcome population, and they do not report the numerical primary endpoint. The registry also warns that adverse-event-related discontinuations differ from Participant Flow because the records came from separate case-report-form pages and were not reconciled. That caveat limits any attempt to combine those sources.

What can a confidence interval add?

In general, a confidence interval expresses the statistical uncertainty around an estimate under a specified analysis. A narrow interval indicates greater precision under that analysis, while a wide interval indicates less precision. A confidence interval does not remove bias, establish causality, or account automatically for differences between groups. No confidence intervals are reported in the registry entries for the adverse-event counts or the named secondary outcomes, so their precision cannot be assessed from this information.

Why does over-dispersion matter?

In general, over-dispersion occurs when observed variability exceeds what a statistical model assumes. If a model does not account for that extra variability, its uncertainty estimates may be too optimistic. This is a general consideration for count data; the registry entries do not report a count model, an over-dispersion assessment, or a model-based analysis for these outcomes. It would therefore be inappropriate to say that over-dispersion was assessed or corrected in this study.

How should time-to-event outcomes be understood?

The listed secondary outcomes include median duration of complete hematologic response, median duration of major cytogenetic response, median progression-free survival, and median overall survival. The outcome names specify the Kaplan-Meier method for several duration outcomes. In general, Kaplan-Meier methods estimate the distribution of time until an event while accounting for participants whose event time is not observed during follow-up through censoring. A median time is the point at which the estimated event-free proportion reaches its midpoint, when the data allow that median to be estimated. The record details do not include the resulting medians, event definitions in detail, censoring summaries, or confidence intervals. Those outcomes therefore cannot be numerically interpreted.

The registry’s sample-size assumptions describe a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. For time-to-event outcomes, the illustration uses a total number of events; for other outcomes, it gives participants per arm. These are planning assumptions, not evidence that the study used a two-arm design or that it had a particular observed power. No numerical sample-size illustrations or observed-effect values are included in the registry record for this account. The assumptions cannot be used to infer the study’s actual design rationale or its precision.

More broadly, the registry does not report a protocol statistical plan, a sample-size justification, interim analyses, or detailed inferential methods. It also lists no analyses. General statistical concepts can help clarify what the outcome definitions and design permit, but they cannot fill in those absent methods or results.

10. Multiplicity, Missing Data and Analysis Populations

The record lists 12 outcome measures, including the primary safety outcome and secondary outcomes concerning hematologic response, cytogenetic response, progression-free survival, and overall survival. When a study evaluates multiple outcomes, general statistical practice may address the possibility that testing many questions increases the chance of at least one apparently unusual result. Whether and how that issue was handled depends on the prespecified analysis plan. A multiplicity strategy is not reported in the registry record, and no analyses are listed. It is therefore not possible to determine whether outcome testing was adjusted, hierarchical, exploratory, or otherwise structured.

Missing-data handling is also not reported in the registry record. The registry entries do not state how participants with incomplete follow-up, unavailable measurements, or unknown outcome status were handled. This matters for both participant counts and time-to-event outcomes: the interpretation depends on how the analysis treated people whose data were incomplete or whose event time was not observed. The Kaplan-Meier method appears in the names of several listed duration outcomes, but the record details do not describe censoring rules or the amount of missing follow-up.

The primary population is described as all treated participants, but the number treated and the analysis population for each secondary outcome are not reported in the registry entries. Enrollment is listed as 46; enrollment should not be assumed to equal the treated population or the number included in every analysis. The adverse-event description also notes that study-drug discontinuations due to adverse events differ from Participant Flow because data from separate case-report-form pages were not reconciled. That specific inconsistency cautions against treating the record’s separate participant summaries as interchangeable.

These gaps prevent a full assessment of analysis populations, missingness, and multiplicity. They do not show that such issues were ignored; rather, the relevant methods are not reported in the registry record.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: cardio-respiratory arrest (25.0%), pneumonia (25.0%), herpes zoster disseminated (25.0%), hernia (25.0%), basal cell carcinoma (25.0%).

The adverse-event results give category-specific numbers at risk and affected. For serious adverse events, the listed counts are 3 of 5 in accelerated-phase CML, 10 of 22 in chronic myelogenous leukemia; QD dosing, 9 of 15 in CML; BID dosing, and 3 of 4 in myeloid blast-phase CML. For other adverse events, the counts are 5 of 5, 21 of 22, 15 of 15, and 4 of 4, respectively, in those same categories. These should be read as descriptive participant counts over the study’s recorded follow-up, not as a comparison between randomized treatments.

The registry defines adverse events broadly, whether or not considered related to study drug, and defines serious events by specified consequences. The listed serious-event terms and percentages are CARDIO-RESPIRATORY ARREST, PNEUMONIA, HERPES ZOSTER DISSEMINATED, HERNIA, and BASAL CELL CARCINOMA at 25.0% each, and PERICARDIAL EFFUSION at 20.0%. The denominator for these term percentages is not reported in the registry record, so the percentages should not be interpreted as full-cohort risks.

Deaths are included in the primary endpoint title, but death counts for the adverse-event categories are null in the record details. Numerical results for the overall primary endpoint are also not reported. The registry notes that event-related discontinuation data differ from Participant Flow and were not reconciled. These qualifications limit how completely the safety record can be summarized from the posted counts.

12. What the Result Does, and Does Not, Mean

The registry record documents a study intended to characterize long-term safety and tolerability during dasatinib exposure among participants previously treated in CA180-002. It gives descriptive counts of participants with serious and other adverse events within disease and dosing categories, along with a median exposure duration of 23.4 months. Those are observations from the study population and study period. They can help readers understand what was recorded, but do not establish that dasatinib caused each event.

The single-group design is central to the limits of interpretation. Without a concurrent control group and randomized allocation, the study cannot directly compare event occurrence under dasatinib with what would have happened under another treatment or no study drug. The listed disease and dosing categories are not like-for-like randomized comparators. Differences between their counts may reflect differences in participants, disease category, follow-up, or other factors, and the registry entries do not provide adjusted comparisons that address these possibilities.

The record also does not provide numerical values for its primary safety endpoint or for the named secondary efficacy outcomes. It is therefore not possible to quantify the overall number of deaths, serious events, adverse events, or discontinuations from the primary endpoint based on these registry entries, or to report response and survival estimates. The outcome titles alone do not show the magnitude, precision, or direction of an outcome.

The serious-event percentages are listed for selected terms, but the denominator is not reported in the registry record. The counts are more directly interpretable as category-specific participant counts, yet they remain descriptive and are not exposure-adjusted rates. The record’s note about unreconciled adverse-event discontinuation information further limits comparisons with participant-flow summaries.

Thus, the posted information supports a careful account of the study design and the reported event counts, while leaving important questions about comparative effects, outcome precision, missing data, and analysis methods unanswered. It does not support a treatment recommendation or a conclusion that the drug is effective or ineffective.

13. Why This Trial Matters Statistically

This study is statistically notable for the distinction between a safety objective and the evidence a single-group design can provide. A descriptive safety record can document events among exposed participants, but it cannot separate treatment effects from the clinical context without a suitable comparator. The registry’s single-group model and allocation recorded as NA make that distinction essential when interpreting the adverse-event categories.

The record also illustrates why outcome definitions, populations, and data provenance matter. The primary endpoint groups deaths, serious adverse events, adverse events, and event-related discontinuations, while the outcome population is identified as all treated participants. Yet numerical primary-outcome values are not reported in the registry entries. The registry separately warns that discontinuation data differ from Participant Flow because the information was collected on separate case-report-form pages and not reconciled. A discrepancy of this kind can affect how readers connect event summaries to participant disposition.

Another important statistical issue is the gap between naming outcomes and reporting their results. The record lists response and time-to-event outcomes, including Kaplan-Meier outcomes, but their numerical results are not included in the registry record. Without estimates and measures of uncertainty, readers cannot judge precision or compare outcome magnitudes. The same applies to the sample-size planning assumptions: an illustration based on an observed effect does not establish the trial’s actual sample-size rationale or observed power.

Finally, the adverse-event categories show how descriptive data may be divided by disease and dosing while remaining non-comparative. The category counts can be reported transparently, but should not be treated as causal contrasts. This distinction is broadly important in clinical research: the statistical meaning of a result depends not only on its numerical value, but also on design, population, follow-up, and the way outcomes were collected and analyzed.

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 registry entries leave several limitations specific to this study. First, the design is single-group, with allocation listed as NA and masking as none. There is no concurrent randomized comparator, so the adverse-event counts cannot establish a treatment contrast. The disease and dosing categories are descriptive groupings rather than randomized arms.

Second, the primary endpoint’s numerical results are not reported in the registry record, despite the endpoint definition and the description of the all-treated population. Death counts in the adverse-event category results are null. The registry entries also do not report numerical values for the listed secondary response and survival outcomes. As a result, the available information does not permit a full quantitative assessment of the stated safety and efficacy outcomes.

Third, the registry explicitly notes that adverse-event-related study discontinuation data differ from Participant Flow because information was captured on separate case-report-form pages and was not reconciled. This makes it difficult to integrate the discontinuation summary with participant disposition. The record does not give further details on the extent of that difference.

Fourth, the serious-event term percentages lack a reported denominator in the registry record. The record does not provide exposure-adjusted rates, uncertainty intervals, detailed missing-data procedures, or analysis populations for each outcome. The median on-study exposure duration of 23.4 months does not describe the individual follow-up distribution.

Finally, a protocol statistical plan, sample-size justification, interim analyses, and detailed methods are not reported in the registry record. The record lists no analyses. These absences restrict assessment of precision, multiplicity, and the handling of incomplete data; they do not establish what may have been done outside the registry.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Chronic Myelogenous Leukemia or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemic Study — NCT00978731.
• Registry record last updated 2011-04-28; status last verified 2011-04.
• Results first posted 2011-04-19.
• 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.

18. Record Summary

NCT00978731 is a completed Phase 1, single-group study of dasatinib in participants previously treated in CA180-002 who experienced clinical benefit. The registry lists enrollment of 46, a start date of 2005-12, and primary completion in 2008-09. The primary objective concerns long-term safety and tolerability; the primary endpoint counts participants with deaths, serious adverse events, adverse events, and adverse-event-related discontinuation. Median on-study exposure was 23.4 months.

Posted adverse-event results report serious and other adverse-event counts across four disease and dosing categories. The record also lists secondary response and survival outcomes, including outcomes named with the Kaplan-Meier method, but their numerical results are not reported in the registry entries. The primary endpoint’s numerical results are likewise not reported. The study has no randomized comparator, and the registry notes that adverse-event discontinuation data were not reconciled with Participant Flow. A statistical plan, missing-data procedures, and detailed analyses are not reported in the registry record.