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 Antibody Therapy in Treating Patients With Refractory or Relapsed Non-Hodgkin's Lymphoma or Chronic Lymphocytic Leukemia (NCT00014560), a registered phase 1 study. The primary outcome is Clinical Toxicity.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00014560 |
| Phase | Phase 1 |
| Condition | Leukemia, Lymphoma |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Dartmouth-Hitchcock Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with refractory or relapsed non-Hodgkin's lymphoma or chronic lymphocytic leukemia (CLL). The registry record reports an enrollment of 3. It does not report further eligibility details or describe the participants' clinical characteristics.
Intervention
The listed biological interventions were bispecific antibody 4G7xH22 and sargramostim. The arm description identifies a single antibody arm. Details about how the agents were administered, including dose and schedule, are not reported in the registry record.
Comparator
No comparator arm is described. The record identifies a single arm and does not state an allocation method. This is not a randomized comparison, so there is no like-for-like control group against which to compare outcomes.
Primary question
The primary outcome was clinical toxicity over day 1-29. The record identifies this as the primary outcome but does not provide a numerical result, a toxicity grading method, or a definition of which events counted toward the outcome.
The study was a Phase I trial of intravenous bispecific antibody in patients with refractory or relapsed non-Hodgkin's lymphoma or CLL. Its stated purpose concerned antibody therapy in these conditions, while its registered primary outcome was clinical toxicity. The registry record also lists serum markers of macrophage activation as a secondary outcome.
The study was terminated. The primary outcome description says that the sponsor ended it prematurely after toxicities at other sites on unrelated trials led the sponsor to decide not to manufacture the investigational product. This explains the stated reason for ending the study, but it does not report toxicities among the participants in this trial.
Because the record describes only one arm and reports no primary outcome measurements, it cannot answer a comparative question about treatment versus another therapy or control. Its registered question is about toxicity monitoring in the enrolled participants; the posted results do not give the clinical toxicity findings needed to characterize that outcome.
3. Trial Design
This was a Phase I trial sponsored by Dartmouth-Hitchcock Medical Center. The title describes intravenous bispecific antibody 4G7xH22 in patients with refractory or relapsed non-Hodgkin's lymphoma or chronic lymphocytic leukemia (CLL). The listed conditions are leukemia and lymphoma. The record gives an enrollment of 3.
The study is described as having a single arm, labeled Antibody. The interventions list bispecific antibody 4G7xH22 and sargramostim, both categorized as biological interventions. Further details about the treatment regimen, including dose, schedule, and the relationship between the listed interventions, are not reported in the registry record. Masking is recorded as none. Allocation is not stated, and there is no control arm described.
The record lists clinical toxicity as the primary outcome, with a timeframe of day 1-29. Serum markers of macrophage activation are listed as a secondary outcome. Although the record indicates three outcome measures, the additional outcome details and the results for these measures are not reported in the registry record.
The study began on 2000-09-28, and its recorded primary completion date is 2003-02-14. Its status is terminated. The primary outcome description attributes premature termination to the sponsor Medarex's decision not to manufacture the investigational product after toxicities were experienced at other sites on unrelated trials. That account is not evidence of toxicity findings among this study's participants.
The record does not report a planned sample size rationale, statistical analysis plan, or intended comparison. With a single arm and no stated allocation method, the design does not provide a randomized treatment comparison. The registry record does not report protocol details needed to assess treatment exposure or to establish how the clinical toxicity outcome was defined and assessed.
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 | Single Arm |
|---|---|
| STARTED | 3 |
| COMPLETED | 0 |
| NOT COMPLETED | 3 |
Analysis population for the primary outcome, as defined in the registry: This study was prematurely ended by the sponsor Medarex (supplier of BsAb) due to toxicities experienced at other sites on unrelated trials leading to the decision that Medarex would not be manufacturing the investigational product (BsAb).
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 | Single Arm |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 2 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Clinical Toxicity, measured over day 1-29.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The registered primary endpoint is clinical toxicity, with a stated timeframe of day 1-29. In a Phase I study, a toxicity endpoint is used to describe safety and tolerability observations during treatment and follow-up. As a general statistical concept, such outcomes can help characterize the types and frequency of adverse events observed in the participants studied. The usefulness of that description depends on clear event definitions, assessment methods, and complete reporting.
The registry record does not state how clinical toxicity was defined, whether toxicity was graded, which events were included, or how the endpoint was summarized. It also does not provide numerical results for this outcome. Therefore, the endpoint title and timeframe identify what the study intended to monitor, but they do not establish what participants experienced.
The endpoint description includes the sponsor's reason for ending the study: toxicities at other sites on unrelated trials led to a decision not to manufacture the investigational product. This is a report about the sponsor's decision and events elsewhere, not a numerical result for this trial's primary endpoint. The registry record does not report a separate participant-level clinical toxicity result for the enrolled group.
Clinical toxicity is used in early-phase research to monitor potential harms, but a primary endpoint label alone cannot convey the severity, timing, or frequency of events. In this record, the lack of endpoint measurements and assessment details prevents a statistical interpretation of the trial's clinical toxicity outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a small, single-arm Phase I study with an enrollment of 3 and a primary outcome of clinical toxicity over day 1-29. It does not report a numerical primary outcome result, an effect estimate, a confidence interval, or an analysis. Consequently, the posted results do not quantify clinical toxicity among the participants or show how precisely any such quantity was estimated.
The record says that the study was prematurely ended by the sponsor. The stated reason was toxicities at other sites on unrelated trials and a subsequent decision not to manufacture the investigational product. Those events explain the study's termination as recorded, but they should not be read as outcomes observed in this trial. No clinical toxicity counts or descriptions for this study are reported in the registry record.
The adverse-event results list a single-arm group with 3 participants at risk for serious adverse events and 0 affected. The corresponding other-adverse-event row also lists 3 at risk and 0 affected. The record does not provide serious adverse event terms, and deaths are not reported in the registry record. These entries describe the recorded adverse-event categories; they do not fill the gap in the separately registered primary outcome.
There is no comparator arm. As a general statistical point, a single-arm study can describe observations in its participants, but it cannot estimate a randomized between-group treatment difference. The absence of a control group also means that changes or events cannot be compared with a concurrent control condition. This trial's record does not report a comparative analysis.
The enrollment of 3 limits how much can be learned from the absence of recorded events. As a general statistical concept, estimates based on very few participants are imprecise, and zero recorded events in a small group does not establish that an event cannot occur. No interval estimates are reported, so the degree of uncertainty cannot be expressed from the registry results.
The study's termination and missing endpoint results are central to interpretation. The record documents that the study ended early, but does not state how many participants received treatment, whether all planned assessments were completed, or why participant-level outcome results are absent. Those matters are not reported in the registry record. The available information therefore supports describing the design, enrollment, recorded status, sponsor's stated reason for termination, and adverse-event entries, but not drawing conclusions about the primary clinical toxicity outcome.
9. Statistical Methods Explained
The statistical information in the registry record is limited. It identifies a Phase I, single-arm study with an enrollment of 3 and a clinical toxicity primary outcome over day 1-29. No statistical analyses are listed. The registry record does not report a statistical analysis plan, a sample-size justification, or an inferential method used to evaluate the primary outcome. The explanations below give general context for interpreting such information, not a description of analyses documented for this study.
What can a single-arm study estimate?
As a general statistical matter, a single-arm study can describe outcomes observed in the enrolled participants. If event counts and a clear denominator are reported, descriptive summaries can show the recorded experience in that group. Such summaries do not establish what would have happened under a different treatment or under a control condition. A randomized comparison is designed to compare groups under the study's allocation process; no such comparison is described in this record.
Here, the adverse-event rows report participants at risk and participants affected for serious and other adverse events. Those counts are descriptive. The record does not report an adjusted or comparative analysis, and the primary clinical toxicity result itself is not numerically reported. The adverse-event entries should therefore not be treated as a substitute for an analysis of the primary endpoint.
Why does a confidence interval matter?
As a general statistical concept, a confidence interval expresses the uncertainty around an estimate under a specified statistical method. A narrow interval indicates greater precision than a wide interval, while an interval that spans a broad range of plausible values leaves substantial uncertainty. The registry record gives no confidence interval for the primary endpoint or adverse-event counts. It is therefore not possible to cite interval-based precision for the outcomes.
With very few participants, even a clearly reported proportion or rate would generally be subject to considerable uncertainty. This is a general point about small samples, not a calculated estimate for this study. The record's enrollment is 3, and no interval estimate is posted. Neither the absence of a reported event nor an empty event-term list demonstrates that the underlying risk is zero.
What does the early termination change?
Early termination can affect the amount and completeness of information collected. In general, participants may have different durations of exposure or follow-up, and planned assessments may not all be completed. Whether that occurred in this study is not reported in the registry record. The documented reason for termination concerns toxicities at other sites on unrelated trials and the sponsor's manufacturing decision; it does not describe the completeness of this study's participant follow-up.
The record gives a primary completion date and a terminated status, but it does not explain how those dates relate to the collection or analysis of outcomes. It also does not report whether the study's original enrollment target was met, whether there were interim analyses, or whether any analysis plan changed. These details cannot be inferred from the termination statement.
How should planning calculations be understood?
As a general statistical concept, sample-size planning asks how much information a study might need to detect a prespecified effect under chosen assumptions. A planning illustration is not an observed outcome, and it is not evidence that a study had adequate power. The registry record does not report the trial's own sample-size justification or planning assumptions.
The stated planning framework for illustrative calculations uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect expressed as a fraction of an observed effect. It also distinguishes total events for time-to-event calculations from participants per arm for other calculations. These are properties of the planning illustration, not the design of this single-arm trial. No observed effect is reported for this study, and no two-arm comparison is described. Therefore, such planning assumptions cannot be interpreted as the trial's own design choices or as evidence of observed power.
What does the record permit?
The record supports a descriptive account of the registered design, enrollment, outcome timeframe, termination status, and posted adverse-event counts. It does not support an inferential treatment comparison, a precise estimate of clinical toxicity, or conclusions about the unreported endpoint assessments. General statistical principles can explain why a small, uncontrolled study has limited capacity for comparative inference, but they cannot replace missing study results.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists clinical toxicity as the primary outcome and serum markers of macrophage activation as a secondary outcome. It also indicates three outcome measures, but further outcome details are not reported in the registry record. The record lists no statistical analyses. It does not report how multiple outcomes were handled, whether any outcome was prioritized for formal testing beyond its primary designation, or whether any adjustment for multiple testing was planned.
As a general statistical concept, testing multiple outcomes can increase the chance of obtaining an apparently notable result by chance when each is considered separately. Prespecified outcome hierarchy or a multiplicity adjustment can address that issue, but no such approach is reported for this study. With no outcome analyses or numerical endpoint results posted, there are no reported hypothesis tests to interpret for multiplicity.
Missing-data procedures are not reported in the registry record. The record does not state whether the participants completed the planned day 1-29 assessments, how incomplete observations were handled, or whether any outcomes were unavailable. It also does not report reasons for missing outcome data. The study's early termination makes completeness an important interpretive question, but the record does not establish whether data were missing or how any gaps were treated.
The analysis population is not described. The registry record does not state whether results were analyzed among all enrolled participants, only participants who received treatment, or another defined group. Because no primary endpoint results are reported, the population contributing to any endpoint analysis cannot be identified. The posted adverse-event rows give 3 participants at risk for each listed category, but do not describe an analysis population for the clinical toxicity outcome or the secondary measure.
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 |
|---|---|---|---|
| Single Arm | 0 | 3 | 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 |
|---|---|
| Single Arm | 0 of 3 |
The adverse-event results list a single arm. For serious adverse events, the row reports 3 participants at risk and 0 affected. For other adverse events, it also reports 3 at risk and 0 affected. These are the counts recorded for the two categories in the registry results.
The serious-event term list is empty. That means no serious event terms are listed in the posted results; the record does not supply a narrative event list. Deaths are not reported in the registry record. The record also does not define the criteria used to classify serious versus other adverse events, provide event-level details, or describe follow-up completeness.
As a general statistical point, zero recorded events among a small group does not establish that the true risk is zero. The counts are descriptive and should be read in the context of an enrollment of 3 and the absence of a comparator. They are separate from the registered primary clinical toxicity endpoint, for which no numerical result is reported. The sponsor's termination explanation refers to toxicities at other sites on unrelated trials, not adverse events recorded among this trial's participants.
12. What the Result Does, and Does Not, Mean
The record establishes that this was a Phase I, single-arm study of bispecific antibody 4G7xH22 in patients with refractory or relapsed non-Hodgkin's lymphoma or CLL, with sargramostim also listed as an intervention. It reports an enrollment of 3, identifies clinical toxicity over day 1-29 as the primary outcome, and records the study as terminated. The sponsor's stated reason for ending the study concerns toxicities at other sites on unrelated trials and a decision not to manufacture the investigational product.
The posted adverse-event results record 0 affected among 3 at risk for serious adverse events and 0 affected among 3 at risk for other adverse events. These entries describe the recorded categories in the registry. They do not provide a complete clinical toxicity result, and the record does not report the primary endpoint numerically. It also does not report deaths or serious adverse event terms.
The results do not establish the frequency or severity of clinical toxicity in the study beyond the posted adverse-event entries. They cannot establish that toxicity did not occur, that an event is impossible, or that the intervention is safe or unsafe. Such conclusions would require clearly defined and complete outcome data, and the registry record does not provide those data.
Because there is no comparator arm, the record also cannot estimate a treatment effect relative to another therapy or a control condition. No statistical analyses, confidence intervals, or comparative results are reported. The enrollment is small, and the reported counts do not support broad generalization to other patients or settings.
The most defensible interpretation is therefore limited: the registry documents the study's design and early termination, while the primary clinical toxicity result and analysis are not reported. The sponsor's reason for termination should not be confused with participant outcomes in this trial. The adverse-event counts are descriptive registry entries, not a comparative efficacy finding or a comprehensive safety assessment.
13. Why This Trial Matters Statistically
This trial illustrates why design and results must be considered together when reading clinical research. It is a Phase I study with a single arm, an enrollment of 3, and a primary outcome focused on clinical toxicity. Those features indicate a study intended to monitor toxicity rather than a randomized comparison between treatment groups. The registry record does not report comparative analyses or numerical results for the primary outcome.
It also illustrates the distinction between a reason for study termination and a study outcome. The recorded explanation refers to toxicities at other sites on unrelated trials and the sponsor's decision not to manufacture the investigational product. That information is important to the study history, but it is not a report of toxicity among the participants enrolled in this trial.
The adverse-event entries show why counts need context. The record reports no affected participants in the serious or other adverse-event categories, while the primary clinical toxicity outcome has no numerical result. As a general statistical matter, sparse observations and small groups provide limited precision. An absence of recorded events in a small group is not proof that the underlying risk is absent.
Finally, the record demonstrates the limits of registry results when outcome definitions, analysis methods, and missing-data handling are not described. A reader can identify the registered question and the reported counts, but cannot assess endpoint estimation, uncertainty, or comparative effects from the posted information. Clear reporting of participant outcomes and analytic methods is necessary for those statistical questions.
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 enrollment is 3, and the study has no comparator arm. These features limit the amount of information available and prevent a like-for-like comparison. The registry record does not report baseline characteristics, so the clinical profile of the enrolled participants cannot be assessed beyond the stated eligibility population.
The primary outcome is labeled clinical toxicity and assigned a day 1-29 timeframe, but the registry record does not define the toxicity criteria, grading approach, event collection method, or summary measure. It reports no numerical primary outcome result. The secondary outcome of serum markers of macrophage activation is listed, but the corresponding results and measurement details are not reported in the registry record.
The study was terminated early, but the record does not report the number of participants treated, exposure duration, completion of follow-up, or whether planned outcome assessments were completed. It also does not describe missing-data handling or the analysis population. These omissions make it difficult to assess whether the posted adverse-event entries reflect complete observation of the enrolled group.
The record provides no statistical analysis plan, sample-size justification, confidence intervals, or outcome analyses. It does not state whether interim analyses occurred or how multiplicity was considered. The reason for termination refers to events at other sites on unrelated trials; it should not be treated as trial-specific outcome evidence. Overall, the registry record allows a limited description of the study and its recorded adverse-event counts, but not a detailed assessment of clinical toxicity, precision, or comparative effects.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Antibody Therapy in Treating Patients With Refractory or Relapsed Non-Hodgkin's Lymphoma or Chronic Lymphocytic Leukemia — NCT00014560.
- Registry record last updated 2018-05-03; status last verified 2018-04.
- Results first posted 2013-08-05.
- 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
NCT00014560 is a Phase I, single-arm study sponsored by Dartmouth-Hitchcock Medical Center. It enrolled 3 patients with refractory or relapsed non-Hodgkin's lymphoma or CLL and listed bispecific antibody 4G7xH22 and sargramostim as interventions. The primary outcome was clinical toxicity over day 1-29; serum markers of macrophage activation were listed as a secondary outcome.
The study began on 2000-09-28, had a primary completion date of 2003-02-14, and is recorded as terminated. The sponsor's stated reason for ending it was a decision not to manufacture the investigational product after toxicities at other sites on unrelated trials. The registry record does not report numerical results for the primary outcome or statistical analyses. Adverse-event entries report 0 affected among 3 at risk for serious events and 0 affected among 3 at risk for other events.