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 and Combination Chemotherapy in Treating Patients With Newly Diagnosed Acute Lymphoblastic Leukemia (NCT00844298), a na phase 2 study. The primary outcome is Proportion of Patients Achieving Hematologic and Molecular Complete Remission (CR) After Induction Therapy.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00844298 |
| Phase | Phase 2 |
| Condition | Leukemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 91 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Asan Medical Center |
| Results posted | Yes |
2. Clinical Question
The clinical question concerns a treatment plan combining nilotinib with multi-agent chemotherapy for adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL). The registry record identifies the study as phase II and names the primary endpoint as the proportion of patients achieving hematologic and molecular complete remission after induction therapy. It does not report a numerical remission result, so the record cannot establish the observed proportion or its precision.
Population
Adults with newly diagnosed Philadelphia chromosome-positive ALL treated in the study. The record lists leukemia as the condition.
Intervention
Nilotinib combined with the mVPD multi-agent chemotherapy treatment plan.
Comparator
No separate comparator arm is recorded. The design is single-group, with allocation listed as not applicable.
Primary question
What proportion of analyzable participants achieved hematologic and molecular complete remission after induction therapy?
This is a description of the study’s stated question, not an estimate of treatment effect. A single-group study can describe outcomes among its participants, but it does not provide a concurrent control group for distinguishing the treatment plan’s contribution from other explanations. The registry record does not report the primary endpoint value, a confidence interval, or an analysis comparing outcomes with another treatment. The question is therefore clinically framed as remission after the combined treatment, while the posted record leaves the size and precision of that outcome unknown.
3. Trial Design
The registry record describes a phase II, single-group study of nilotinib plus mVPD chemotherapy in patients with newly diagnosed Philadelphia chromosome-positive adult ALL. Asan Medical Center is listed as the sponsor. The study is marked completed, with enrollment of 91. Its recorded start is 2009-01 and its primary completion is 2012-06.
Allocation is listed as not applicable, masking as none, and the model as single group. These entries indicate that the registry record does not describe a randomized, masked comparison between treatment arms. The intervention is named Nilotinib+mVPD, and the arm description says participants received the nilotinib plus mVPD treatment plan. No separate control arm is recorded.
The primary outcome is the proportion achieving hematologic and molecular complete remission after induction therapy, with a timeframe of one month and an approximate assessment point described as recovery of cytopenia. Disease or relapse-free survival and overall survival are listed as secondary outcomes. The record identifies three outcome measures, but no outcome analyses are posted. It does not report treatment doses, detailed eligibility criteria, the chemotherapy schedule, follow-up duration, or the protocol’s statistical plan.
Because there is no concurrent comparison group, the design supports description of outcomes within the treated group, subject to the completeness and definitions of the posted data. It does not directly estimate how those outcomes would differ under another treatment or under no treatment. The absence of reported outcome analyses also means that the registry entry cannot be used to assess the observed remission or survival results.
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
| Stage | Nilotinib+mVPD |
|---|---|
| STARTED | 91 |
| COMPLETED | 91 |
| NOT COMPLETED | 0 |
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+mVPD |
|---|---|---|
| Participants analysed | 90 | |
| Age, Continuous | years | 47.0 (17 to 71) |
| Sex: Female, Male: Female | Participants | 45 |
| Sex: Female, Male: Male | Participants | 45 |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Patients Achieving Hematologic and Molecular Complete Remission (CR) After Induction Therapy, measured over 1 month.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of analyzable subjects |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is titled as the proportion of patients achieving hematologic and molecular complete remission after induction therapy. In general, a proportion describes how many participants in a defined analysis population meet an outcome criterion, relative to the number assessed in that population. Hematologic and molecular remission refer to distinct dimensions of response; the registry title presents them together as the endpoint but does not supply detailed criteria for either component.
The recorded unit is percentage of analyzable subjects, while the parameter is listed as a number. This metadata does not resolve how the result was intended to be displayed or whether both remission components had to be met for a participant to count. The endpoint description gives a timeframe of one month and an approximate timing of recovery of cytopenia. It does not define the analyzable population, explain how missing assessments were handled, or provide the endpoint’s observed value.
In general, a remission endpoint can provide a concise measure of response at a specified assessment point. Its interpretation depends on clear outcome criteria, a defined denominator, and complete ascertainment. Those details and a numerical result are not reported in the registry record, so the endpoint’s result and precision cannot be evaluated from this entry.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the study question and treatment group, but it does not post a numerical result for the primary remission endpoint. Consequently, no observed remission proportion, confidence interval, or endpoint-specific statistical test can be interpreted from this record. The endpoint title alone is not evidence that a particular proportion achieved remission.
The record lists disease or relapse-free survival and overall survival as secondary outcomes. It does not provide their estimates, event counts, follow-up summaries, or analyses. The entry reports three outcome measures and zero analyses. Thus, the named outcomes indicate what was registered, but they do not establish what was observed.
Enrollment was 91, and the adverse-event record reports 18 participants with serious adverse events among 91 at risk, as well as 79 with other adverse events among 91 at risk. Febrile neutropenia is listed among the leading serious adverse-event terms at 19.8%. These safety entries describe reported counts and a reported percentage; they do not supply the remission result or demonstrate a comparison with another regimen. Deaths are not reported in the registry record.
In general, an estimate is more informative when accompanied by a measure of uncertainty, such as a confidence interval. A confidence interval indicates the range of values reasonably compatible with the data and the analysis assumptions; it does not correct for bias, missing observations, or lack of a control group. No confidence interval for remission or survival is reported, so the precision of those outcomes cannot be assessed.
The single-group design is central to interpretation. Outcomes observed among participants receiving nilotinib plus mVPD cannot, by themselves, isolate the effect of nilotinib, chemotherapy, or their combination. Differences in participant characteristics, disease course, supportive care, and assessment practices can influence observed outcomes in clinical studies generally. The registry entry does not give comparative data that would address those possibilities.
The record supports a narrow conclusion: it describes a completed phase II, single-group study and records some adverse-event counts, but it does not post analyses of the registered efficacy outcomes. Any claim about the magnitude, precision, or comparative effect of the treatment plan would go beyond the registry results available in this entry.
9. Statistical Methods Explained
The registry record does not report a statistical analysis plan or outcome analyses. The concepts below explain how results of this kind are generally interpreted; they are not a description of methods confirmed to have been used in this study.
What does a remission proportion represent?
For a binary endpoint such as achieving or not achieving a defined remission, the basic summary is a proportion: the number meeting the endpoint divided by the number included in the analysis population. A clear report should state both the numerator and denominator, define the analysis population, and explain how participants without an evaluable assessment were treated. The registry describes the primary endpoint as a percentage of analyzable subjects, but does not report the count, denominator, or definition of analyzable subjects. It also does not provide detailed criteria for hematologic or molecular remission.
Why does uncertainty matter?
A point estimate alone does not show how much uncertainty surrounds an outcome. In general, a confidence interval expresses the range of values compatible with the observed data under a specified statistical model and its assumptions. A narrower interval generally indicates greater precision, while a wider interval indicates less precision. It cannot establish that an estimate is unbiased, and it does not address design limitations such as the lack of a comparator. No remission estimate or confidence interval is reported in this registry record.
What can a single-group design estimate?
A single-group study can describe outcomes in the participants who received the treatment plan. Without a concurrent comparison group, it does not directly estimate a contrast between that plan and another option. This distinction is important: an observed outcome in a treated group is not itself a treatment effect. In general, causal comparisons require a design and analysis that support a defensible counterfactual comparison. The registry lists allocation as not applicable and the model as single group; it does not describe a separate control arm.
How should survival endpoints be analyzed?
In general, time-to-event outcomes account for both whether an event occurred and when it occurred. Participants whose event has not been observed by the end of available follow-up may be censored, meaning their full event time is unknown but their observed follow-up still contributes information. Interpretation depends on event definitions, follow-up, censoring, and the analysis method. The registry lists disease or relapse-free survival and overall survival as secondary outcomes, but does not report event definitions, follow-up summaries, censoring details, or analyses. These general principles therefore cannot be used to infer what analysis was conducted.
What does over-dispersion mean?
In general, over-dispersion arises when count data vary more than a statistical model expects. If it is ignored, standard errors and uncertainty intervals can be too small, making results appear more precise than the data justify. The registry’s primary endpoint is a remission proportion, not a reported count-model analysis, and the record does not report a model for that endpoint. Over-dispersion should not be assumed to have been assessed or to be relevant to an unreported analysis.
How should planning illustrations be read?
Sample-size calculations are planning tools, not observations from the trial. The registry’s stated planning illustration uses an observed effect as its reference and describes assumptions including a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also distinguishes planning for a stated fraction of that effect. These are illustrations based on the stated assumptions, not evidence that the trial randomized participants between arms or had a particular probability of detecting an effect. The study itself is recorded as single-group. Such illustrations are not observed power, and the record does not report the trial’s own sample-size justification.
In general, any planning calculation depends on the chosen effect, variability assumptions, analysis method, and design. A planning result should not be mistaken for an observed outcome or for a substitute for an uncertainty interval around an actual estimate. The registry does not post an efficacy estimate from which readers could assess the planning illustration against observed results.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary remission outcome and the secondary outcomes disease or relapse-free survival and overall survival. It identifies three outcome measures overall but posts no analyses. The statistical plan, including any planned hierarchy of outcomes, adjustment for multiple testing, or distinction between confirmatory and exploratory analyses, is not reported in the registry record.
In general, testing multiple outcomes can increase the chance of finding at least one apparently notable result by chance. A prespecified strategy may address this through an outcome hierarchy or an adjustment to the interpretation of statistical tests. Without a reported plan and results, it is not possible to determine whether multiplicity was addressed or how it should affect interpretation.
The primary endpoint refers to analyzable subjects, but the criteria defining that population are not reported. The record does not explain whether participants with missing or unevaluable remission assessments were excluded, counted as not achieving remission, or handled in another way. It also does not report the amount or pattern of missing data, the analysis population for secondary outcomes, or any sensitivity analysis. These omissions matter because the denominator and handling of unavailable assessments can change the interpretation of a proportion.
Interim analyses, stopping rules, and the detailed statistical analysis plan are not reported in the registry record. Their absence from the entry should not be taken as evidence that a particular procedure was or was not used. With no posted efficacy analyses, readers cannot assess the consequences of these unanswered questions for the reported 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+mVPD | 18 | 91 | 19.8% |
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+mVPD | 79 of 91 |
Most frequent serious adverse event terms
| Event term | Organ system | Nilotinib+mVPD |
|---|---|---|
| Febrile neutropenia | Infections and infestations | 18 of 91 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Nilotinib+mVPD |
|---|---|---|
| Jaundice | Hepatobiliary disorders | 79 of 91 |
Serious events with the highest arm-level rate: febrile neutropenia (19.8%).
The safety entries are recorded for the Nilotinib+mVPD group. Serious adverse events are reported for 18 participants among 91 at risk. Other adverse events are reported for 79 participants among 91 at risk. These are participant counts in the registry record; the entry does not provide event-level totals or explain whether an individual could contribute to more than one category.
Febrile neutropenia is listed as a leading serious adverse-event term, with a reported value of 19.8%. The registry record does not provide further detail on the term’s event count, timing, severity criteria, or relationship to treatment. Deaths are not reported in the registry record.
In general, adverse-event counts describe events recorded under a study’s definitions and observation procedures. They are not, on their own, a comparison of risk between treatments. Because this study is single-group and no comparator is recorded, the listed figures cannot establish how these events compare with another regimen. The entry also does not report exposure duration or additional context needed to interpret event frequency over time.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II study of nilotinib plus mVPD in newly diagnosed Philadelphia chromosome-positive adult ALL was completed and enrolled 91 participants. It identifies hematologic and molecular complete remission after induction therapy as the primary endpoint, with a stated timeframe of one month and an approximate assessment point at recovery of cytopenia. It also names disease or relapse-free survival and overall survival as secondary outcomes.
What the record does not establish is the observed remission proportion, the survival results, or the precision of any efficacy estimate. No outcome analyses are posted. The listed adverse-event counts provide limited descriptive safety information, but they do not constitute a comparison with another treatment and do not supply a complete account of safety.
In general, treatment effects are assessed by comparing outcomes under different treatment conditions using a suitable design and analysis. This registry entry describes a single group and no separate comparator. As a result, even if a remission result were available, the design would not by itself show how outcomes would have differed under another treatment. The entry does not report enough outcome data to make even a descriptive estimate of remission.
The distinction between a registered endpoint and a reported result is important. Naming a primary outcome tells readers what the study intended to assess; it does not show the outcome value. Likewise, naming secondary outcomes does not establish that their analyses were completed or that results were favorable or unfavorable. The registry record supports description of the design, participants enrolled, endpoint definitions at a broad level, and selected adverse-event figures. It does not support a conclusion about the size or comparative effect of the treatment plan.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of why design and results must be read separately. The registry identifies a clear treatment group and a remission question, but the design is single-group and no efficacy analyses are posted. Those features limit what can be concluded even before considering the absence of numerical endpoint results.
A proportion is interpretable only with its outcome definition and denominator. Here, the endpoint title combines hematologic and molecular remission, while the recorded unit refers to a percentage of analyzable subjects and the parameter is listed as a number. The record does not clarify the remission criteria or define analyzable subjects. This illustrates how registry metadata can name an endpoint without supplying all details needed to reproduce or interpret its calculation.
The study also illustrates the difference between planning and evidence. Sample-size illustrations are tied to assumptions and are not observed outcomes, nor do they create a comparison group in a single-group study. In general, planning figures cannot replace a reported estimate with uncertainty or resolve limitations in the design.
The adverse-event entries demonstrate another interpretive boundary: counts from a treated group can describe what was recorded, but without a concurrent comparator they do not quantify relative risk. A rigorous statistical reading therefore distinguishes the registry’s design information, registered outcomes, posted safety counts, and missing efficacy analyses rather than treating them as interchangeable evidence.
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 efficacy is that the registry record posts no outcome analyses. The primary remission endpoint has no reported value or uncertainty interval, and the named secondary survival outcomes have no reported estimates or event summaries. The record therefore cannot support an assessment of efficacy magnitude or precision.
The single-group design has no separate control arm, so it cannot directly estimate a comparative treatment effect. Allocation is listed as not applicable and masking as none. The record does not report detailed eligibility criteria, the chemotherapy schedule or doses, follow-up duration, or the statistical analysis plan. It also does not define the analysis population or explain how missing or unevaluable endpoint assessments were handled.
The endpoint metadata leave some ambiguity: the title describes a proportion, the unit refers to a percentage of analyzable subjects, and the parameter is listed as a number. Detailed criteria for hematologic and molecular remission are not reported. The record also does not explain the relationship between the overall enrollment and the analyzable population.
Safety interpretation is limited to the reported participant counts and febrile neutropenia percentage. Deaths are not reported in the registry record, and further detail on event timing, duration of exposure, and definitions is not given. These limitations are specific to what this registry entry records; they should not be read as claims about information that may exist outside the entry.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Nilotinib and Combination Chemotherapy in Treating Patients With Newly Diagnosed Acute Lymphoblastic Leukemia — NCT00844298.
- Registry record last updated 2015-09-07; status last verified 2015-08.
- Results first posted 2015-09-07.
- 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.
18. Record Summary
The registry record describes a completed phase II, single-group study sponsored by Asan Medical Center, with enrollment of 91. It evaluates nilotinib plus mVPD chemotherapy in newly diagnosed Philadelphia chromosome-positive adult ALL. The primary endpoint is the proportion achieving hematologic and molecular complete remission after induction therapy, with a timeframe of one month and approximate assessment at recovery of cytopenia.
Disease or relapse-free survival and overall survival are listed as secondary outcomes. The record reports 18 participants with serious adverse events and 79 with other adverse events among 91 at risk; febrile neutropenia is listed at 19.8%. It does not post efficacy outcome analyses, define the analyzable population, or report a comparative arm. The remission and survival results, their precision, and deaths are not reported in the registry record.