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 LSA4 Protocol for the Treatment of Advanced Pediatric and Adolescent Non-Hodgkins Lymphoma (NCT00610883), a na phase 2 study. The primary outcome is Complete Remission.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00610883 |
| Phase | Phase 2 |
| Condition | Non-Hodgkin's Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 17 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The registry title describes pediatric and adolescent patients with advanced Non-Hodgkin's Lymphoma. The listed condition is Non-Hodgkin's Lymphoma, and the record reports an enrollment of 17 participants. Further eligibility details, including age limits and disease-subtype criteria, are not reported in the registry record.
Intervention
The intervention is identified as LSA4 and lists Cyclophosphamide, Methotrexate, Daunomycin, L-asparaginase, and BCNU. The registry summary describes treatment in induction, consolidation, and maintenance parts. It says induction ends on day 19, consolidation ends on day 38 or 42, and maintenance may include up to 6 cycles. Additional dosing and administration details are not reported in the registry record.
Comparator
There is no separate comparator group. The study is recorded as a single-group design, with no allocation and no masking. It therefore does not provide a randomized or like-for-like control comparison. No control treatment or external comparator is reported in the registry record.
Primary question
The primary outcome is the number of participants who achieved complete remission as a result of treatment, with a timeframe of 330 Days. The record does not give the number who achieved remission or an estimate of uncertainty around that number. Consequently, the registry entry identifies what was intended to be counted, but does not report the result needed to describe the observed remission frequency or its precision.
3. Trial Design
This is a completed phase 2, single-group study sponsored by Memorial Sloan Kettering Cancer Center. The registry record lists 17 participants and identifies the study as concerning advanced pediatric and adolescent Non-Hodgkin's Lymphoma. The recorded start is 1990-05, and the primary completion is 2008-12. The record gives no separate randomized comparator arm, no allocation, and no masking.
The intervention is named LSA4, Cyclophosphamide, Methotrexate, Daunomycin, L-asparaginase, and BCNU. The registry summary describes a three-part approach: induction, consolidation, and maintenance. It says induction ends on day 19, consolidation ends on day 38 or 42, and maintenance may include up to 6 cycles. More detailed treatment procedures, including doses and criteria for moving between parts, are not reported in the registry record.
The primary outcome is complete remission, defined in the record as the number of patients who achieved a complete remission as a result of treatment. Its timeframe is 330 Days. The outcome result itself is not reported in the registry record. Thus, although the entry supplies a study design, an enrolled participant count, and the name and timeframe of the primary outcome, it does not provide the remission count or an accompanying statistical analysis. Other design details, including eligibility criteria, the analysis population, and a 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.
Overall Study
| Stage | All Patients |
|---|---|
| STARTED | 17 |
| COMPLETED | 17 |
| 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 | All Patients |
|---|---|---|
| Participants analysed | 17 | |
| Age, Customized: Median Age | years | 12 (2 to 19) |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Complete Remission, measured over 330 Days.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
Complete remission is the study's primary endpoint. The registry describes it as the number of patients who achieved complete remission as a result of treatment, measured over a timeframe of 330 Days. In plain terms, this endpoint is a count of participants meeting a remission criterion during the stated follow-up period. The specific clinical criteria used to determine complete remission are not reported in the registry record.
As a general statistical concept, a remission count can describe how many participants in a study met the endpoint. When the number of participants assessed is also known, the count can be interpreted in relation to the assessed group, and uncertainty can be quantified if an appropriate analysis is reported. That interpretation depends on knowing who was assessed, how missing outcome information was handled, and what analysis method was used.
The registry record does not report the remission count, the number assessed for this endpoint, or an uncertainty estimate. It therefore identifies the intended endpoint but does not permit a quantitative description of the observed remission result or its precision. The timeframe alone does not establish that every participant completed follow-up or had an outcome recorded.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record supports a limited description of the study: it was a completed, phase 2, single-group study, with 17 participants, and its primary endpoint was the number achieving complete remission at 330 Days. It does not report the number achieving that endpoint. Without that result, the central outcome cannot be summarized as an observed count, and no estimate of its uncertainty can be described.
This absence should not be interpreted as a count of zero, as evidence that no participants achieved remission, or as evidence that all participants achieved it. The outcome result is not reported in the registry record. The enrollment count of 17 is not a substitute for the number assessed at the primary endpoint; the analysis population and endpoint-specific follow-up are not reported either.
The single-group design also limits what the study can establish comparatively. There is no randomized control group in the registry record against which to assess the remission outcome. A count from a single group, even if reported, would describe the experience recorded for that group; by itself it would not separate the contribution of the intervention from other explanations or establish a comparative treatment effect. No treatment-effect estimate or statistical analysis is listed in the registry record.
Precision cannot be assessed from the posted endpoint information because neither the outcome count nor a confidence interval is reported. As a general statistical concept, a confidence interval communicates the uncertainty around an estimate under the assumptions of the analysis. It would not remove the limitations of a single-group design, and no such interval is reported for this primary outcome.
The safety information is a separate descriptive component. The registry lists 1 participant affected by a serious adverse event among 17 at risk, and 17 participants affected by other adverse events among 17 at risk. Ruptured Necrotic Tumor is listed as a leading serious adverse event term at 5.9%. The death count is not reported in the registry record. These safety counts do not supply the missing remission result and should not be treated as an efficacy comparison.
Overall, the record tells readers the endpoint that was selected and the study structure in which it was assessed, but it does not contain the endpoint result or a statistical analysis. Any stronger quantitative interpretation would require information not reported in the registry record.
9. Statistical Methods Explained
The design determines the kinds of conclusions a study can support. This record describes a single-group phase 2 study with no allocation and no masking. A single-group study can document outcomes among its participants, but it has no concurrent comparison group in the registry record. As a general statistical concept, a randomized comparison helps distinguish differences associated with an intervention from differences that may arise because groups differ in other ways. That comparative structure is absent here, so the record does not provide a randomized treatment-effect estimate.
What does the primary count represent?
The primary endpoint is a count of participants achieving complete remission within the stated timeframe of 330 Days. A count is a descriptive outcome. To interpret it fully, readers would need to know the count, the number of participants whose outcome was assessed, and the definition and assessment process for remission. The registry record identifies the endpoint and timeframe, but does not report the remission count, the assessed population, or the remission criteria.
As a general statistical concept, a binary outcome records whether each participant did or did not meet a defined condition. A count of participants meeting that condition can be used to estimate a proportion when the denominator is known. An uncertainty interval can then describe how much sampling variability may affect the estimate. Those quantities cannot be reconstructed from the registry entry because the outcome count and analysis details are not reported. No recalculated result is available in the registry record.
Why does a confidence interval matter?
In general, a confidence interval gives a range of values compatible with an estimate under a specified statistical method and its assumptions. A narrower interval indicates greater precision under those assumptions, while a wider interval reflects more uncertainty. A confidence interval is not a guarantee that the true value lies in the reported interval, and it cannot correct design limitations such as the lack of a comparator. No confidence interval for complete remission is reported in the registry record.
What can a significance test add?
In general, a significance test evaluates compatibility between data and a specified null hypothesis under a statistical model. A p-value is not the probability that a treatment works, nor is it a measure of the size or clinical importance of an effect. The registry record lists no statistical analyses for the primary outcome and reports no p-value. There is therefore no test result to interpret, and the record does not report a prespecified statistical plan or null hypothesis.
How should the planning assumptions be understood?
The record's sample-size assumptions describe a planning illustration using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They also describe the effect to detect as a stated fraction of an observed effect. This is a hypothetical two-arm planning framework, whereas the study design is recorded as single-group. No numerical sample-size planning results are reported in the registry record, and no effect estimate is reported from which to assess such planning.
As a general statistical concept, sample-size calculations depend on assumptions about the outcome, target effect, variability, allocation, and error rates. A planning calculation does not show what the study actually observed. It is not observed power. The record's assumptions therefore should not be read as evidence that the enrolled study had a particular probability of detecting an effect, especially since no numerical planning result or primary outcome result is reported.
The planning description also distinguishes time-to-event outcomes, for which event totals may be used, from other outcomes, for which participants per arm may be used. The registered primary endpoint is a participant count for complete remission, not a reported time-to-event estimate. The record does not provide a numerical calculation to interpret for this endpoint.
Why can over-dispersion matter?
As a general statistical concept, over-dispersion occurs when observed variability exceeds what a chosen model assumes. If a model understates variability, standard errors may be too small and uncertainty intervals or tests may be too optimistic. Whether over-dispersion is relevant depends on the outcome and model. The registry record does not report a model, dispersion estimate, or analysis from which to assess it.
In sum, statistical interpretation requires the endpoint value, the population analyzed, and the method used. The record supplies the outcome definition and timeframe, but does not report the remission result, an uncertainty estimate, or an inferential analysis. General statistical methods explain what those items would mean; they cannot fill in values that are not reported.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome measure, complete remission, and no statistical analyses. It does not report whether additional outcomes were prespecified in a protocol, whether multiple comparisons were planned, or whether any adjustment for multiplicity was used. These details are not reported in the registry record. The listed count of one outcome measure does not establish that no other analyses were considered or performed.
As a general statistical concept, multiplicity arises when several outcomes, time points, subgroups, or hypotheses are evaluated. Repeated testing can increase the chance of obtaining a nominally notable result by chance. Prespecification and appropriate methods can help interpret such analyses. Since no inferential results or analysis plan are reported, the impact of multiplicity cannot be evaluated for this study.
Missing-data methods and analysis populations are also not reported in the registry record. The enrollment is 17, but the record does not state how many participants had an assessable complete-remission outcome at 330 Days, whether any participants lacked outcome data, or how missing outcomes were handled. It also does not identify an intention-to-treat, per-protocol, or other analysis population. These labels should not be inferred from enrollment alone.
As a general statistical concept, missing outcomes can affect an estimate when the participants with observed data differ from those without observed data. Different assumptions about missingness can lead to different interpretations. Without the analysis population and missing-data approach, it is not possible to assess the extent or direction of this potential uncertainty. No interim-analysis plan or interim results are reported in the registry record.
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 |
|---|---|---|---|
| All Patients | 1 | 17 | 5.9% |
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 |
|---|---|
| All Patients | 17 of 17 |
Most frequent serious adverse event terms
| Event term | Organ system | All Patients |
|---|---|---|
| Ruptured Necrotic Tumor | Cardiac disorders | 1 of 17 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Patients |
|---|---|---|
| Hematological Toxicity | Blood and lymphatic system disorders | 17 of 17 |
| Hepatic Toxicity | Renal and urinary disorders | 17 of 17 |
Serious events with the highest arm-level rate: ruptured necrotic tumor (5.9%).
The adverse-event record lists all patients as the group, with 17 participants at risk for both serious and other adverse events. The serious adverse-event affected count is 1, while the other adverse-event affected count is 17. These are descriptive registry counts; they are not a comparison between treatment groups, because the study is recorded as single-group.
Ruptured Necrotic Tumor is listed among the leading serious adverse event terms, at 5.9%. The registry record does not provide further context for this term in the adverse-event entry, such as timing, attribution, severity grading details, or whether an individual could contribute more than one event. Those details are not reported in the registry record.
Serious adverse events and other adverse events are separate categories in the record and should not be combined or interpreted as interchangeable measures. The death count is not reported in the registry record. These data describe adverse events recorded for the group; they do not establish causation, comparative risk, or the relationship between adverse events and the primary remission outcome.
12. What the Result Does, and Does Not, Mean
The registry entry establishes that a phase 2, single-group study of the LSA4 regimen enrolled 17 participants and designated complete remission at 330 Days as its primary outcome. It also records an intervention name, a three-part treatment summary, and adverse-event counts. Those are meaningful elements of the study record, but they do not amount to a reported primary-outcome result.
The remission count is not reported in the registry record. Therefore, readers cannot tell from this entry how many participants achieved complete remission, what proportion of assessed participants did so, or how precise any estimate might be. The absence of a reported result is not evidence of a particular outcome. It should not be interpreted as either success or failure.
Even if the remission count were available, a single-group design would not provide a randomized comparison with another treatment or a control condition. As a general statistical concept, an observed outcome in one group can describe what happened among those participants, but it cannot on its own establish what would have happened under a different treatment or no treatment. The registry record does not report a comparative effect estimate.
The safety information likewise has a descriptive scope. It records 1 participant affected by serious adverse events among 17 at risk, 17 affected by other adverse events among 17 at risk, and a leading serious adverse-event term of Ruptured Necrotic Tumor at 5.9%. It does not report the death count. The entry does not provide enough information to determine causation or compare safety with another group.
Accordingly, the record is useful for identifying the study design, population description, intended primary outcome, and some safety counts. It does not support a quantitative conclusion about the primary remission endpoint or a comparative conclusion about treatment effects. Additional outcome and methods information is not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why a registry entry's design and outcome result need to be read separately. The record identifies a phase 2, single-group study and names complete remission at 330 Days as the primary endpoint. Yet the remission count is not reported in the registry record. A clearly stated endpoint does not, by itself, provide evidence about the observed endpoint value.
The single-group design is statistically important because it defines the scope of inference. No randomized comparator is listed, so the entry cannot support a direct treatment-versus-control contrast. This distinction matters even when a study records a participant count and a clinically framed outcome: descriptive outcomes in one group and comparative effects answer different questions.
The record also shows why denominators and analysis populations matter. Enrollment is 17, while the number assessed for the primary outcome is not reported in the registry record. Without that information, a reader cannot determine how the outcome count should be interpreted or how missing endpoint information was handled. Statistical precision cannot be evaluated without the outcome result and an uncertainty estimate.
Finally, the record's sample-size assumptions describe a hypothetical two-arm planning framework, while the study is recorded as single-group. That contrast is a reminder that planning illustrations are not observed findings and should not be mistaken for evidence about the trial's actual statistical power. With no numerical planning results or primary outcome analysis reported, the statistical interpretation remains limited to the design and the outcomes recorded descriptively.
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 primary limitation for interpreting the main clinical question is that the complete-remission result is not reported in the registry record. The endpoint definition and timeframe are listed, but the remission count and an uncertainty estimate are absent. The clinical criteria for complete remission are also not reported, making it impossible to assess how the endpoint was operationalized from the registry entry alone.
The single-group design has no concurrent comparator in the registry record. It therefore does not provide a randomized treatment contrast. The record also does not report eligibility criteria, participant characteristics, disease subtypes, or details of the analysis population. Although the title specifies pediatric and adolescent patients with advanced lymphoma, further description of who was enrolled is not reported in the registry record.
Endpoint-specific follow-up and missing-data handling are not reported. The enrollment count of 17 does not establish that all participants contributed an assessable primary outcome at 330 Days. The record does not identify an analysis plan, statistical model, confidence interval, p-value, or interim analysis. Without these details, precision, inferential assumptions, and the effect of missing outcomes cannot be assessed.
The intervention is named and the summary describes treatment parts, but more detailed dosing and implementation information is not reported in the registry record. For safety, the entry provides affected counts and a leading serious adverse-event term, but does not report attribution, timing, or the death count. These limitations leave the record informative about study structure but insufficient for a complete assessment of efficacy or comparative safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: LSA4 Protocol for the Treatment of Advanced Pediatric and Adolescent Non-Hodgkins Lymphoma — NCT00610883.
- Registry record last updated 2023-09-28; status last verified 2023-09.
- Results first posted 2016-01-25.
- 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 describes a completed phase 2, single-group study sponsored by Memorial Sloan Kettering Cancer Center, with 17 participants. The title concerns treatment of advanced pediatric and adolescent Non-Hodgkin's Lymphoma. The recorded start is 1990-05, and primary completion is 2008-12.
The intervention is listed as LSA4 with Cyclophosphamide, Methotrexate, Daunomycin, L-asparaginase, and BCNU. The primary endpoint is the number achieving complete remission at 330 Days. That result is not reported in the registry record, and no statistical analyses are listed.
The adverse-event entry lists 1 participant affected by serious events among 17 at risk and 17 affected by other events among 17 at risk. Ruptured Necrotic Tumor is listed as a leading serious adverse-event term at 5.9%. The death count is not reported in the registry record.