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 17-AAG in Treating Patients With Relapsed or Refractory Anaplastic Large Cell Lymphoma, Mantle Cell Lymphoma, or Hodgkin's Lymphoma (NCT00117988), a na phase 2 study. The primary outcome is Number of Patients With Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00117988 |
| Phase | Phase 2 |
| Condition | Anaplastic Large Cell Lymphoma, Recurrent Adult Hodgkin Lymphoma, Recurrent Mantle Cell Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
The trial asked whether participants with relapsed or refractory anaplastic large cell lymphoma, mantle cell lymphoma, or classical Hodgkin's lymphoma experienced a complete or partial response while receiving 17-AAG. It was a phase II study with a single treatment group, not a randomized comparison. The registry record reports enrollment of 22 participants and identifies response as the primary outcome, assessed from baseline to best response.
Population
Participants had relapsed or refractory CD30+ anaplastic large cell lymphoma, relapsed or refractory mantle cell lymphoma, or relapsed or refractory classical Hodgkin's lymphoma. The registry record reports 22 enrolled participants. The primary-outcome population was defined as participants with a baseline and at least one post-baseline target lesion measurement; its actual size is not reported in the registry record.
Intervention
Participants received 17-AAG, also named tanespimycin in the intervention record. The treatment description specifies intravenous administration over 1 hour on days 1, 4, 8, and 11, repeated every 21 days for up to 8 courses if disease progression or unacceptable toxicity did not occur.
Comparator
There was no separate comparator group. The study is recorded as single-group, with no allocation and no masking. Consequently, its response observations describe participants receiving 17-AAG; they do not provide a concurrent control-group estimate of what would have happened under another treatment or no treatment.
Primary question
The primary outcome was the number of participants with a complete or partial response. The registry record gives response definitions based on decreases in lymph node masses and specifies assessment every 6 weeks. The number who responded, and any statistical estimate of response with its uncertainty, are not reported in the registry record.
3. Trial Design
This was a phase II, single-group study sponsored by the National Cancer Institute. The registry record lists the study as completed, with a start date of 2005-02 and primary completion in 2010-04. Enrollment was 22. The conditions listed were anaplastic large cell lymphoma, recurrent adult Hodgkin lymphoma, and recurrent mantle cell lymphoma; the official study title further specifies relapsed or refractory disease and classical Hodgkin's lymphoma.
All participants were assigned to the 17-AAG treatment group. The record describes intravenous treatment over 1 hour on days 1, 4, 8, and 11, with courses repeated every 21 days for up to 8 courses in the absence of disease progression or unacceptable toxicity. The treatment description also begins to describe possible treatment after disease regression following 8 courses, but that description is incomplete in the registry record, so further details cannot be established from it.
The primary outcome was a count of participants experiencing complete or partial response. The stated assessment period ran from baseline to best response, with assessments every 6 weeks. The analysis population was described as intention to treat, with inclusion requiring both a baseline and at least one post-baseline target lesion measurement. The number actually included in that outcome analysis is not reported in the registry record.
A single-group design can describe outcomes observed during treatment, but it cannot by itself separate the treatment experience from the natural course of disease or other influences. No randomized comparator, masking, or allocation process is recorded. The registry record does not report a detailed statistical analysis plan, an analysis of response, or a rationale for the enrollment target.
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 | 17-AAG |
|---|---|
| STARTED | 22 |
| COMPLETED | 18 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | 17-AAG |
|---|---|
| Death | 3 |
| Adverse Event | 1 |
Analysis population for the primary outcome, as defined in the registry: Analysis was intention to treat (ITT). All participants with baseline and at least one post baseline target lesion measurement were included.
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 | 17-AAG |
|---|---|---|
| Participants analysed | 22 | |
| Age, Continuous | years | 49.5 (25 to 76) |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 16 |
| Region of Enrollment: United States | participants | 22 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Response, measured over Baseline to time to best response; Every 6 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the number of participants with a complete or partial response. It is a binary participant-level outcome: each participant either meets the stated response definition or does not. Reporting a count can describe how many participants in the analysis population met the criterion, while an associated proportion and uncertainty interval can help readers understand the result relative to the size of that population. The registry record does not report the response count, proportion, or uncertainty interval.
The response definitions use changes in lymph node masses. The registry description defines partial response as a greater than 50% decrease and complete response as a greater than 75% decrease. The registry record does not explain the boundary wording further. Assessment was scheduled every 6 weeks, and the timeframe was baseline to time to best response. Thus, the endpoint concerns whether a participant reached either category during follow-up, rather than a direct comparison of average change between treatment groups.
Response is used in studies of this kind as a way to summarize observed tumor change. In general, a prespecified response criterion makes the outcome more reproducible than an informal judgment. However, the endpoint alone does not describe how long a response lasted, what happened after the best response, or how outcomes compare with a control group. Those results are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the number of participants with response as the primary outcome, but it does not report how many participants achieved complete or partial response. It also records no analyses. Therefore, the efficacy result cannot be quantified from this record: there is no posted response count, response proportion, confidence interval, or statistical test to interpret. The absence of a reported analysis is not evidence that no participant responded; it means the registry record does not give the result needed to assess that question.
Enrollment was 22, but the endpoint population was defined as participants with a baseline and at least one post-baseline target lesion measurement. The number meeting that definition is not reported in the registry record. Enrollment and the endpoint analysis denominator should therefore not be treated as interchangeable. Without the denominator and outcome count, neither the observed response frequency nor its precision can be determined.
In general, a confidence interval expresses the range of values reasonably compatible with an estimate under the statistical model. A narrow interval indicates greater precision than a wide interval, while neither interval width nor a point estimate resolves the limitations of a single-group design. No confidence interval is reported for this outcome. No p-value or model-based estimate is reported either. Statistical significance, uncertainty, and the stability of any response estimate therefore cannot be evaluated from the registry results.
The study had no comparator group. Even if a response count were available, the single-group result would describe what was observed among participants receiving 17-AAG, not the difference in outcome relative to another treatment. It would not establish how much of the observed response was attributable to treatment rather than other influences. The response definition and scheduled assessments give the endpoint a clear clinical subject, but they do not supply a counterfactual comparison.
The registry includes safety counts: 3 of 22 participants were recorded in the serious adverse event group, and 21 of 22 in the other adverse event group. Death is listed among the top serious adverse event terms at 13.6%. These figures are safety descriptions, not substitutes for the missing response result. The record does not give enough detail to determine from those entries the timing, relationship to treatment, or individual overlap of events.
Any planning figures based on an observed effect should be read as planning illustrations, not as evidence of study power or as observed results. The registry describes a calculation using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those assumptions do not describe the actual single-group design. With no response estimate or analysis reported in the registry record, the clinical and statistical interpretation remains limited to the endpoint definition, study structure, and reported safety counts.
9. Statistical Methods Explained
The primary outcome is a count of participants meeting a response definition. Since the outcome is whether each participant did or did not meet that definition, it is binary at the participant level. A count can be paired with a denominator to describe the observed response proportion. The registry record supplies enrollment but does not supply the response count or the actual number of participants in the stated analysis population, so the response proportion cannot be interpreted from the record.
What does a response estimate describe?
In general, an estimated proportion summarizes the frequency of an event in the population used for analysis. Its interpretation depends on a clear event definition and an appropriate denominator. Here, the event is complete or partial response, and the response criteria are described in terms of decreases in lymph node masses. The analysis population was described as intention to treat, but eligibility for that analysis required a baseline and at least one post-baseline target lesion measurement. The actual denominator is not reported in the registry record.
In general, a confidence interval communicates uncertainty around an estimate. It reflects that a result based on a limited set of observed participants may not precisely identify the underlying response frequency. The width of an interval depends on the estimate, the amount of information, and the method used. No confidence interval or method for calculating one is reported for the primary endpoint. It is therefore not possible to judge the precision of an efficacy estimate from the registry record.
Why does the study structure matter?
This is a single-group study, with no randomized comparator. In general, randomization can help make groups comparable with respect to measured and unmeasured influences, while a concurrent comparator can provide a reference for interpreting outcomes. Neither feature is present in the recorded design. A response observed in the treatment group could be described, but a treatment-versus-control effect cannot be estimated from a group that has no control arm. This design limitation is distinct from statistical uncertainty: even a precisely estimated response frequency would not by itself provide a comparative treatment effect.
The record states that response was assessed every 6 weeks from baseline to best response. In general, repeated assessments provide opportunities to identify whether an outcome criterion has been met over follow-up. Interpretation also depends on the assessment rules, how missing visits are handled, and how best response is determined. Those operational details, beyond the stated schedule and response definitions, are not reported in the registry record. Nor is a duration-of-response analysis reported.
What can be inferred from the analysis record?
The registry record lists one outcome measure and no analyses. It gives no efficacy estimate, statistical test, model, or adjustment procedure. A p-value, if reported, would address compatibility of data with a specified statistical hypothesis under model assumptions; it would not measure the size or clinical importance of an effect. No such p-value is available for the response endpoint. Likewise, no model assumptions or diagnostics are reported, so there is no basis for evaluating a fitted efficacy model.
In general, a count of participants with an event is commonly analyzed as a proportion when a denominator is known. More complex approaches may account for covariates or different study structures, but the registry record does not specify that any such approach was used. Over-dispersion is a concern in some count or rate models when observed variability exceeds the model's expectation; that general issue does not establish that such a model was used in this trial. The endpoint is described as a participant count, and no dispersion estimate or model is reported.
The record also includes planning illustrations based on an observed effect. It states that those calculations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. These are planning assumptions, not a report of the trial's design: the actual study was single-group. Planning calculations are not observed power, and they do not establish the probability that the study detected a real effect. The numeric planning outputs and the effect estimate on which they rely are not reported in the registry record.
In general, sample-size planning asks how much information a future study would need under specified assumptions about the effect, variability, significance level, and desired power. If assumptions change, the resulting planning estimate can change. Such an illustration should not be interpreted as an observed participant count, an efficacy result, or a retrospective measure of success. It also cannot repair the absence of an outcome analysis in the registry record.
Finally, an analysis label does not settle how every participant was handled. The stated requirement for at least one post-baseline target lesion measurement means the named analysis population is defined more narrowly than simply all enrolled participants. The registry does not state how participants without post-baseline measurements, incomplete assessments, or other missing data were handled. Any interpretation should distinguish the planned endpoint and population description from the results and methods that are actually reported.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one outcome measure and no analyses. It does not report a multiplicity strategy, such as whether testing across outcomes or subgroups was planned, or how any resulting error rates would be addressed. The presence of a single listed outcome measure does not establish that no additional analyses were conducted or planned; such details are not reported in the registry record. With no reported efficacy analyses, there are no p-values to assess for multiplicity.
The primary analysis population is described as intention to treat, with inclusion limited to participants who had a baseline and at least one post-baseline target lesion measurement. The record does not state how many participants met these criteria. It also does not explain how participants without qualifying post-baseline measurements were handled, whether missing lesion measurements were imputed, or whether any sensitivity analysis was performed. These details are not reported in the registry record.
In general, missing outcome data can affect an estimated response frequency if participants with missing assessments differ from those with observed assessments. The direction and extent of any effect depend on the missingness pattern and assumptions used to handle it. Without the analysis denominator, missing-data counts, or handling method, the potential influence cannot be assessed. The intention-to-treat label does not, by itself, specify how missing outcome information was addressed, particularly given the stated measurement requirement for inclusion.
The registry record also does not report a statistical analysis plan, a prespecified hierarchy among outcomes, or interim analyses. No conclusions about these procedures should be inferred from their omission. The record supports describing the endpoint and its stated analysis population, but not reconstructing unreported multiplicity or missing-data methods.
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 |
|---|---|---|---|
| 17-AAG | 3 | 22 | 13.6% |
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 |
|---|---|
| 17-AAG | 21 of 22 |
Most frequent serious adverse event terms
| Event term | Organ system | 17-AAG |
|---|---|---|
| Death | General disorders | 3 of 22 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 17-AAG |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 8 of 22 |
| Fatigue | General disorders | 7 of 22 |
| Nausea | Gastrointestinal disorders | 6 of 22 |
| Fever | General disorders | 6 of 22 |
| Pain | General disorders | 5 of 22 |
| Vomiting | Gastrointestinal disorders | 5 of 22 |
| Neuropathy | Nervous system disorders | 5 of 22 |
| Hypomagnesemia | Metabolism and nutrition disorders | 4 of 22 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 4 of 22 |
| Blurred Vision | Eye disorders | 3 of 22 |
Serious events with the highest arm-level rate: death (13.6%).
The registry lists 22 participants at risk for the reported serious adverse event grouping, with 3 affected. For the other adverse event grouping, it lists 22 at risk and 21 affected. These are participant counts and should be read as safety summaries for the 17-AAG group, not as efficacy outcomes. The registry also lists Death as a top serious adverse event term at 13.6%.
The 13.6% entry is reported as a term percentage. The registry record does not explain its calculation details or state how that term relates to the 3 participants in the serious adverse event grouping. It should not be converted into a participant count or assumed to identify the number of deaths. The record does not report the timing of these events, individual event histories, or whether events were considered related to treatment.
In general, adverse event counts describe events recorded during a study and do not, by themselves, establish causation. Interpretation also depends on definitions, severity, duration, timing, exposure, and comparison with an appropriate reference. Those details are not reported. The safety entries therefore support only a limited description of the recorded counts and term; they do not show whether 17-AAG caused an event or permit a comparative safety assessment.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase II, single-group study enrolled 22 participants with relapsed or refractory lymphoma and used complete or partial response as its primary outcome. It describes the response criteria, the assessment schedule, and the population intended for the endpoint analysis. It also reports safety counts for serious and other adverse event groupings and identifies Death among the top serious adverse event terms.
It does not report how many participants achieved a response, the number included in the response analysis, or an estimate of response frequency. No confidence interval, statistical test, or efficacy analysis is recorded. Consequently, the posted registry information does not allow a reader to quantify the observed response or its precision. It also does not establish that no response occurred; the result is simply not reported in the registry record.
Even if the response count were available, the single-group design would support a description of observed outcomes among participants receiving 17-AAG, not a direct estimate of the difference between 17-AAG and another treatment. It could not, by itself, determine what would have happened to comparable participants without 17-AAG. The response endpoint also does not answer how long any response lasted, whether responses were sustained, or what other clinical outcomes occurred; those results are not reported in the registry record.
The safety counts likewise need a limited interpretation. They document recorded event groupings, but do not establish that treatment caused an event. The record lacks detail on event timing, attribution, and individual patterns. Overall, the information supports an account of the trial's design and recorded endpoint and safety information, but it does not support a conclusion about treatment efficacy or comparative safety.
13. Why This Trial Matters Statistically
This trial illustrates an important distinction between a clearly named endpoint and a reported endpoint result. The registry identifies response as the primary outcome, describes its assessment schedule, and specifies a population definition. Those elements help readers understand what the study intended to measure. Yet without the response count and analysis denominator, the core efficacy result cannot be quantified. A stated endpoint is not a substitute for an estimate and its uncertainty.
The design also matters to the meaning of any response result. A single-group study can describe what was observed among participants receiving treatment, but it cannot provide a randomized, concurrent comparison. This distinction is fundamental: precision about a single-group response frequency would not eliminate the absence of a control group or support a comparative treatment effect.
The record also shows why analysis-population labels need to be read alongside their operational definitions. Although the population is described as intention to treat, inclusion in the primary outcome analysis required a baseline and at least one post-baseline target lesion measurement. The actual analysis population size and missing-data handling are not reported. A label alone does not tell readers which participants contributed outcome information or how incomplete measurements affected analysis.
Finally, planning illustrations should be kept separate from observed evidence. The registry describes assumptions for hypothetical two-arm calculations, while the trial itself had a single treatment group. These calculations are not observed power and do not fill the gap left by an unreported response analysis. The example highlights the value of distinguishing design, planning assumptions, safety descriptions, and actual outcome estimates when interpreting clinical trial records.
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 direct limitation of the registry record is that it does not report the primary response result. Although the response endpoint and assessment schedule are stated, there is no response count, analysis denominator, proportion, confidence interval, or test. The record therefore does not permit an efficacy estimate or an assessment of its statistical precision.
The single-group design limits comparative interpretation. There is no randomized control group against which response frequency or safety can be compared. The record also does not provide a statistical analysis plan, model specification, covariate adjustment, or analysis diagnostics. These methods are not reported in the registry record and should not be inferred.
The primary analysis population is defined by having baseline and at least one post-baseline target lesion measurement, but the number meeting that definition is unavailable. The record does not report participant-level missingness, reasons for missing assessments, imputation, or sensitivity analyses. The intention-to-treat description therefore does not resolve how incomplete outcome data were handled.
The response criteria are presented as decreases in lymph node masses, but the wording of the complete-response threshold is not clarified further in the registry record. Although assessments were scheduled every 6 weeks through best response, details about assessment procedures, independent review, response duration, and follow-up after best response are not reported. The treatment description is also incomplete after beginning to address what may happen following regression after 8 courses.
Safety reporting is limited to grouped counts and a listed serious event term. The record does not give event timing, treatment attribution, individual overlap, or the basis for the Death percentage. These limitations prevent a detailed safety assessment and do not support causal conclusions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: 17-AAG in Treating Patients With Relapsed or Refractory Anaplastic Large Cell Lymphoma, Mantle Cell Lymphoma, or Hodgkin's Lymphoma — NCT00117988.
- Registry record last updated 2014-05-30; status last verified 2013-02.
- Results first posted 2011-05-30.
- 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 describes a completed phase II, single-group study of 17-AAG in relapsed or refractory anaplastic large cell lymphoma, mantle cell lymphoma, and classical Hodgkin's lymphoma. The National Cancer Institute is listed as sponsor. Enrollment was 22, with a start date of 2005-02 and primary completion in 2010-04. Treatment was intravenous 17-AAG on specified days, repeated every 21 days for up to 8 courses under the stated conditions.
The primary outcome was the number of participants with complete or partial response, assessed every 6 weeks from baseline to best response. The record defines the analysis population by baseline and at least one post-baseline target lesion measurement. It does not report the response count, analysis denominator, efficacy analysis, or confidence interval. Safety entries include 3 of 22 participants in the serious adverse event grouping, 21 of 22 in the other adverse event grouping, and Death listed at 13.6% among the top serious adverse event terms. The record does not provide details needed to interpret causality or comparative safety.