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 Stage I/II Nasal NK Cell Lymphoma (NCT01321008), a na phase 1, phase 2 study. The primary outcome is Progression-free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01321008 |
| Phase | Phase 1, Phase 2 |
| Condition | Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Newly diagnosed patients with Stage I/II nasal NK cell lymphoma. The registry lists lymphoma as the condition and describes the intended population as patients with stage 1 and/or 2 NK cell lymphoma.
Intervention
Radiation therapy followed by CHOP chemotherapy. The listed regimen includes cyclophosphamide, doxorubicin, vincristine, and prednisone.
Comparator
There is no separate control group in the registered design. The study is listed as a single-group trial, with allocation recorded as NA and masking as none.
Primary question
The primary outcome was progression-free survival, defined as time from treatment initiation to first documented progressive disease or death due to disease. The registry also describes safety as a study goal.
The clinical question was whether radiation therapy followed by chemotherapy could help control Stage I/II nasal NK cell lymphoma, while also studying the safety of the regimen. This is a question about outcomes after a combined treatment strategy in the enrolled study group, rather than a comparison of that strategy with another treatment or with no treatment.
The registry record lists one study group, named Radiation + Chemotherapy, and an enrollment of 1. It also states that the study terminated early and that no analysis is available. Consequently, the record does not provide an analyzed estimate of progression-free survival or a statistical comparison that answers the question about disease control. The posted safety counts are also limited and do not establish the safety profile of the regimen.
3. Trial Design
The registry describes a Phase 1 and Phase 2, single-group study titled Stage I/II Nasal NK Cell Lymphoma. Its official title describes radiation therapy followed by chemotherapy for newly diagnosed patients with Stage I/II nasal NK cell lymphoma. The listed sponsor is M.D. Anderson Cancer Center. The recorded start was 2011-05, the primary completion was 2013-05, and the current status is terminated.
The study has one listed treatment group, Radiation + Chemotherapy. The intervention description specifies radiation therapy at a total dose of 50.4 to 54 Gy over 28 to 30 treatments, followed by CHOP chemotherapy. CHOP is described as cyclophosphamide 750 mg/m2 IVPB, doxorubicin 50 mg/m2 IVPB, and vincristine 1.4 mg/m2, with a maximum dose of 2 mg, IVPB on Day 1, plus oral prednisone 100 mg daily on days 1-5 for four 21-day cycles.
Allocation is recorded as NA, masking as none, and the model as single group. There is no registered like-for-like control group, so this design does not support a randomized comparison of treatment strategies. The registry records enrollment of 1 and states that the study terminated early, with no analysis available. The reason for termination and details of any changes to the planned study are not reported in the registry record.
The record describes progression-free survival as the primary outcome. It does not report a statistical analysis plan, sample-size justification, or methods for handling departures from the intended treatment or follow-up. Those details should not be inferred from the phase labels or the treatment description.
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: Study was terminated early due to extensive revisions to the study.
Overall Study
| Stage | Radiation + Chemotherapy |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Radiation + Chemotherapy |
|---|---|
| Early Study Termination | 1 |
Analysis population for the primary outcome, as defined in the registry: Study terminated early, no analysis available.
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 | Radiation + Chemotherapy |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival (PFS), measured over Day 1 to disease progression or death (up to 5+ years).
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is progression-free survival (PFS). The registry defines PFS as the time from treatment initiation to the first documented progressive disease or death due to disease. It gives the timeframe as Day 1 to disease progression or death, up to 5+ years.
In general, PFS is a time-to-event measure: it captures how long it takes for a specified event to occur. It is used when the timing of disease progression or death is relevant, rather than only whether either event occurred by a single assessment date. A PFS analysis ordinarily depends on clear definitions of the event, follow-up, and the way observations without a recorded event are handled. The registry describes the endpoint and its follow-up schedule, but the specific statistical handling of such observations is not reported in the registry record.
The registry says PFS was reviewed with each 21-day treatment cycle, followed every 3-4 months for the first 2 years, and annually thereafter. These are the stated assessment intervals, not evidence that all planned assessments took place. The record says the study terminated early and no analysis is available. It therefore provides no estimated PFS, event-time summary, confidence interval, or comparison with another group. The endpoint definition identifies what the study intended to measure; it does not, by itself, show what happened to participants.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the registry record reports no analysis for the primary endpoint. It lists one outcome measure, progression-free survival, but its population description says the study terminated early and no analysis is available. There is therefore no posted PFS estimate to interpret and no estimate of how precisely an outcome was measured.
The enrollment is 1, and the study is registered as a single-group design. A single-group record can describe outcomes observed after a treatment strategy, but without a control group it cannot provide a like-for-like estimate of how outcomes would differ under another strategy. Here, the record does not even provide an analyzed outcome summary for the enrolled group. No treatment effect, statistical significance, or comparative conclusion can be drawn from the primary endpoint information.
The safety information is similarly limited. For the Radiation + Chemotherapy group, the record lists 1 at risk and 0 affected in the serious adverse event category. It lists 1 at risk and 1 affected in the other adverse event category. Deaths are recorded as null. These entries are counts in the registry record, not a complete characterization of the types, timing, or severity of all possible events. The list of top serious terms is empty. A null deaths entry should not be treated as a reported count of zero.
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. They do not describe the registered single-group design. Because no primary analysis is available, the record supplies no analyzed observed effect that would make such an illustration interpretable as a trial-specific result. Planning calculations are not observed outcomes or observed power.
Overall, the registry supports a description of the intended question, treatment, endpoint, and limited posted safety counts. It does not support a conclusion about PFS or a comparative treatment effect. The reason for early termination, the extent of follow-up actually completed, and the detailed statistical procedures are not reported in the registry record.
9. Statistical Methods Explained
The design and available results determine which statistical interpretations are possible. The registry lists a single-group model, allocation as NA, and masking as none. It records one study group rather than a randomized treatment comparison. The record also states that the study terminated early and no analysis is available. The methods below explain general statistical concepts relevant to the registered endpoint and design; they should not be read as a description of procedures that the investigators used unless the registry record states them.
What does a single-group design estimate?
In general, a single-group study describes outcomes observed among participants who receive the study intervention. A treatment comparison requires an appropriate group representing a different strategy or a defined comparator. Without that group, an observed outcome cannot establish what would have happened to comparable participants under an alternative strategy. This is why the absence of a control group matters for interpretation, even when an outcome is clearly defined.
For this record, the named group received radiation plus chemotherapy, and the registered design is single group. The registry does not offer a randomized, like-for-like control comparison. More importantly, it reports no primary-outcome analysis. Thus, neither an outcome estimate for the group nor a comparative treatment estimate is available in the record.
How is progression-free survival understood?
In general, a time-to-event endpoint measures the time from a defined starting point until a specified event. Here, the registry defines the starting point as treatment initiation and the event as first documented progressive disease or death due to disease. The endpoint is called progression-free survival because it concerns the time until one of those defined events, not simply a count of events at a single visit.
Time-to-event methods can use information from participants whose event has not been documented by the end of their observation, provided the analysis specifies how that information is handled. In general, this requires an appropriate rule for the observation period and for censoring. The registry describes when PFS was to be reviewed but does not report the censoring rules or the analysis method. No event-time analysis is available to assess.
What can a confidence interval tell a reader?
In general, a confidence interval expresses statistical uncertainty around an estimate under the assumptions of the analysis method. A narrower interval indicates greater precision than a wider interval, while the interval's position relative to a relevant value can help readers assess compatibility with possible effects. The interpretation depends on the endpoint, study design, and analysis assumptions; an interval does not remove limitations such as lack of a comparator or incomplete information.
The registry record contains no PFS estimate or confidence interval. Precision therefore cannot be assessed for the primary endpoint. It would be inappropriate to infer precision from the enrollment count alone, or to treat the absence of a reported interval as evidence that an effect is either small or large.
What does statistical significance mean in general?
In general, a significance test evaluates data against a specified null hypothesis using a chosen statistical model and significance level. Its result depends on the design, endpoint, observed data, and analysis assumptions. A p-value is not a measure of clinical importance, and it does not state the probability that a treatment works. A test also cannot compensate for a design that lacks a suitable comparator.
No primary-outcome test or analysis is reported in the registry record. The planning assumption of a two-sided significance level of 0.05 is part of a sample-size illustration, not a report of a test result from this study. There is no PFS p-value to interpret.
How should the sample-size illustration be read?
The registry's sample-size assumptions describe a planning illustration computed from an observed effect. They specify 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 tables, the stated quantity is the total number of events, using the Schoenfeld method; other tables are described as participants per arm.
These are planning assumptions, not evidence about the registered trial's design or its results. The actual record is single group, not an equal-allocation two-arm comparison. The record also says no analysis is available, so it supplies no analyzed observed effect on which to base a trial-specific interpretation. Any sample-size figures generated under those assumptions are planning illustrations only, not observed power and not a record of the number enrolled. The registry does not report the trial's own sample-size justification.
Which analysis details remain unknown?
Statistical methods depend on prespecified choices, such as the analysis population, endpoint rules, handling of missing observations, and any planned interim or subgroup analyses. Those choices influence how estimates and uncertainty should be understood. The registry does not report a statistical analysis plan, analysis populations, or procedures for missing data. Interim analyses and subgroup analyses are not reported in the registry record. These absences mean that the intended analytic framework cannot be reconstructed from the posted record.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study evaluates multiple hypotheses or outcomes and draws conclusions across them. In general, repeated testing can increase the chance of a false-positive finding unless the analysis plan addresses that issue. A prespecified strategy may identify a primary outcome, define the testing sequence, or otherwise explain how multiple tests will be interpreted.
The registry lists one outcome measure, with progression-free survival designated as primary. It reports zero analyses. No multiplicity procedure or broader testing strategy is reported in the registry record. With no analysis available, there are no posted hypothesis-test results for which the effect of multiplicity adjustment can be assessed. The listing of one outcome measure alone does not establish whether other analyses were planned or performed.
Missing-data methods and analysis populations are also not reported in the registry record. In general, missing outcome information can affect an estimate if the chance that information is missing is related to participants' outcomes or other characteristics. The handling of incomplete follow-up, withdrawals, or unavailable assessments therefore matters, particularly for a time-to-event endpoint. The endpoint description gives planned assessment intervals, but it does not specify how incomplete observation would be treated statistically.
The record does not describe an interim-analysis plan, any stopping boundary, or the reason for termination. It states that the study terminated early and that no analysis is available; the timing and circumstances of that decision are not reported in the registry record. No analysis population, such as an intention-to-treat or per-protocol population, is identified. These omissions prevent readers from evaluating how participants were intended to contribute to an analysis or how incomplete information might affect the results.
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 |
|---|---|---|---|
| Radiation + Chemotherapy | 0 | 1 | 0.0% |
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 |
|---|---|
| Radiation + Chemotherapy | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Radiation + Chemotherapy |
|---|---|---|
| Erythema Multiforme | Skin and subcutaneous tissue disorders | 1 of 1 |
| Sinus Disorder | Infections and infestations | 1 of 1 |
| Fatigue | General disorders | 1 of 1 |
| Respiratory, Thoracic and Mediastinal Disorders | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
The registry lists adverse event counts for the Radiation + Chemotherapy group. In the serious adverse event category, 1 participant is listed as at risk and 0 as affected. In the other adverse event category, 1 is listed as at risk and 1 as affected. The deaths entry is null, and the list of top serious terms is empty.
These entries should be read as the safety counts recorded in the registry, not as a full account of event types or clinical context. The record does not identify the other adverse event in the figures shown, and an empty list of top serious terms does not supply a description of events. A null value for deaths means the record does not report a count there; it should not be rewritten as zero deaths.
In general, adverse event counts describe what was recorded in a study and are not, by themselves, proof that an intervention caused an event. Here, the limited counts and the lack of an outcome analysis do not establish a comprehensive safety profile. The registry does not report further details on event timing, duration, severity beyond the serious-versus-other categorization, or attribution.
12. What the Result Does, and Does Not, Mean
The record documents a clinical study intended to assess radiation therapy followed by chemotherapy in newly diagnosed patients with Stage I/II nasal NK cell lymphoma. It identifies progression-free survival as the primary endpoint and describes how that endpoint was defined and scheduled for review. It also records limited adverse event counts for the listed treatment group. These registry entries describe the study's registration and the results fields available in the record.
The record does not show whether PFS was longer or shorter, how many disease-progression or disease-related death events occurred, or how precisely an outcome was estimated. The primary-outcome population description explicitly states that the study terminated early and no analysis is available. There is no posted PFS estimate, confidence interval, or test result from which to draw an efficacy conclusion.
The design also limits comparative interpretation. Because the study is listed as single group and has no like-for-like control group, it cannot estimate a treatment contrast against another strategy. Even if an outcome estimate had been reported for the treatment group, a single-group result would not by itself determine what would have happened under a different treatment. In this record, the absence of an analyzed outcome means even a descriptive PFS result is unavailable.
The safety counts should not be expanded into a general claim about safety. They are limited registry entries, with a null deaths value and no serious-event terms listed. The record does not provide enough detail to characterize all events or assess their relationship to treatment. The reason for study termination, completed follow-up, missing-data handling, and analysis populations are not reported in the registry record.
The appropriate reading is therefore narrow: the registry records an intended treatment strategy and endpoint, but no analyzed primary result. It neither establishes benefit nor establishes lack of benefit, and it does not support a comparative or definitive safety conclusion.
13. Why This Trial Matters Statistically
This record illustrates why a clear endpoint definition and an available analysis are both necessary for statistical interpretation. Progression-free survival is defined as time from treatment initiation to first documented progressive disease or death due to disease, and the registry specifies planned review intervals. That information clarifies what investigators intended to measure. It does not reveal the observed endpoint values or the uncertainty around them.
The study's single-group design is also central. A single-group study and a randomized comparison answer different statistical questions. The former can describe outcomes in the group receiving the intervention; the latter can estimate a contrast between groups under the design's assumptions. The registry does not list a control group, and it reports no analysis. Readers therefore have neither an analyzed group outcome nor a comparative estimate.
The record further demonstrates why planning calculations should be separated from trial results. The sample-size assumptions describe a hypothetical two-arm planning framework, while the registered study is single group. Planning illustrations do not indicate what was observed, do not represent observed power, and cannot fill in for a missing endpoint analysis. They also cannot explain why a study terminated.
Finally, the safety fields show the importance of preserving the distinction between a zero and a missing value. The serious adverse event count affected is recorded as 0, whereas deaths are recorded as null. Those entries have different meanings in the record and should not be conflated. Together, these design and reporting features make the study statistically important as an example of the boundary between what a registry can document and what its results can support.
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 most important limitation is the lack of an analyzed primary outcome. The registry states that the study terminated early and no analysis is available. The record therefore does not provide an estimate of progression-free survival, its precision, or a statistical test. The reason for termination is not reported in the registry record, so it cannot be attributed to efficacy, safety, recruitment, or any other cause.
The registered design is single group, with allocation recorded as NA and masking as none. It does not include a like-for-like control comparison. Thus, the record cannot support a randomized comparative treatment effect. Enrollment is listed as 1, which also means the posted record offers very limited information about outcomes and safety in the intended population.
The registry describes a planned endpoint assessment schedule, but it does not establish how much follow-up was completed or whether the planned assessments occurred. The statistical analysis plan, analysis populations, missing-data procedures, censoring rules, interim analyses, and multiplicity approach are not reported in the registry record. These omissions limit assessment of bias, precision, and the intended interpretation of any analysis.
The safety fields are sparse: counts are listed for serious and other adverse events, the deaths value is null, and no top serious terms are listed. The record does not provide detail sufficient to characterize event type, timing, severity, or attribution. The sample-size assumptions refer to a two-arm planning illustration and should not be mistaken for the single-group trial's own design or for evidence about its results.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Stage I/II Nasal NK Cell Lymphoma — NCT01321008.
- Registry record last updated 2015-02-05; status last verified 2014-05.
- Results first posted 2014-06-23.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry record for NCT01321008 describes a Phase 1 and Phase 2, single-group study of radiation therapy followed by CHOP chemotherapy for newly diagnosed patients with Stage I/II nasal NK cell lymphoma. It lists one treatment group, enrollment of 1, and progression-free survival as the primary outcome. The study is marked terminated, and the primary-outcome description states that no analysis is available.
The record defines PFS as time from treatment initiation to first documented progressive disease or death due to disease, with planned review during treatment and follow-up. It reports limited adverse event counts, but no analyzed PFS estimate or comparative result. The reason for termination, statistical analysis plan, missing-data methods, and analysis populations are not reported in the registry record.