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 Treatment With AMD3100 (Plerixafor) in Non-Hodgkin's Lymphoma and Multiple Myeloma Patients (NCT00322842), a non-randomized phase 2 study. The primary outcome is Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00322842 |
| Phase | Phase 2 |
| Condition | Lymphoma, Non-Hodgkin, Multiple Myeloma |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 35 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Genzyme, a Sanofi Company |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled participants with non-Hodgkin lymphoma (NHL) or multiple myeloma (MM). The registry record reports an enrollment of 35 and identifies the safety population as all participants who received at least 1 dose of plerixafor. The condition-specific at-risk groups in the adverse-event summary are 4 participants with NHL and 31 with MM.
Intervention
The intervention was G-CSF plus plerixafor. The arm descriptions state that participants received G-CSF 10 µg/kg/day for 4 days, followed by plerixafor 240 µg/kg on the evening of day 4. G-CSF was given the next morning, followed by apheresis. The descriptions are truncated after the shared regimen, so later dosing details are not reported in the registry record.
Comparator
This was a non-randomized, single-group study with no masking. It did not include a concurrent control group. A secondary outcome describes comparison with historical controls treated with G-CSF alone or chemotherapy and G-CSF, but details of that comparison are not reported in the registry record. The study therefore does not provide a randomized, like-for-like treatment comparison.
Primary question
The primary endpoint counts participants with treatment-emergent adverse events (TEAEs), collected from the start of G-CSF mobilization on Day 1 until the day before chemotherapy, approximately day 38. The study also prespecified an exploratory question about whether an interval of 10-11 hours between plerixafor dosing and apheresis would be associated with at least a 2-fold increase in circulating CD34+ cells. The primary endpoint's overall result is not reported in the registry record.
3. Trial Design
NCT00322842 is recorded as a phase 2, non-randomized, unmasked, single-group study. Its title describes treatment with AMD3100, also identified as plerixafor, in people with NHL and MM. The registry record lists enrollment of 35, a start date of 2004-09, primary completion in 2007-02, and status as completed. The sponsor is Genzyme, a Sanofi Company.
The intervention was G-CSF plus plerixafor. For both disease groups, the arm descriptions state that G-CSF was administered at 10 µg/kg/day for 4 days, then plerixafor 240 µg/kg was given on the evening of day 4. G-CSF was given the next morning, followed by apheresis. The descriptions end mid-sentence when describing subsequent dosing, so the full treatment schedule is not reported in the registry record.
The study aimed to evaluate safety and other outcomes described as exploratory. A prespecified outcome concerned the interval of 10-11 hours between plerixafor dosing and the start of apheresis, and whether circulating CD34+ cells would increase by at least 2-fold. The registry summary says that data were intended to assist in determining dosing schedules for future studies.
There was no concurrent randomized comparator. A secondary outcome refers to historical controls treated with G-CSF alone or chemotherapy and G-CSF, but the record does not report the details or results of that comparison. The design can describe outcomes observed during the study regimen, but it cannot isolate a treatment effect through a randomized comparison. Details of the protocol's sample-size justification, allocation procedures beyond the non-randomized designation, and any interim analyses 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 | Non-Hodgkin's Lymphoma (NHL) | Multiple Myeloma (MM) |
|---|---|---|
| STARTED | 4 | 31 |
| COMPLETED | 2 | 29 |
| NOT COMPLETED | 2 | 2 |
Reasons for not completing this period:
| Reason | Non-Hodgkin's Lymphoma (NHL) | Multiple Myeloma (MM) |
|---|---|---|
| Withdrawal by Subject | 0 | 1 |
| Lost to Follow-up | 0 | 1 |
| Refused transplant | 1 | 0 |
| Death | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety population - all participants who received at least 1 dose of plerixafor.
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 | Non-Hodgkin's Lymphoma (NHL) | Multiple Myeloma (MM) | Total |
|---|---|---|---|---|
| Participants analysed | 4 | 31 | 35 | |
| Age, Continuous | years | 59.8 (SD 8.1) | 58.8 (SD 7.3) | 58.9 (SD 7.3) |
| Sex: Female, Male: Female | Participants | 3 | 12 | 15 |
| Sex: Female, Male: Male | Participants | 1 | 19 | 20 |
| Race/Ethnicity, Customized: Caucasian | participants | 4 | 30 | 34 |
| Race/Ethnicity, Customized: Other | participants | 0 | 1 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE), measured over Day 1 to approximately Day 38 (before start of chemotherapy).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants in the overall safety summary who experienced treatment-emergent adverse events (TEAEs). It is a participant count, not a measure of how many separate events each person had. The specified collection period begins on Day 1, when G-CSF mobilization starts, and continues to the day before chemotherapy, approximately day 38. The safety population includes all participants who received at least 1 dose of plerixafor.
Investigators graded adverse events using the World Health Organization (WHO) Adverse Event Grading Scale and assessed severity as mild, moderate, or severe. They also assessed relatedness to study treatment on a scale from “not related” to “definitely related.” These assessments give context about the type and judged severity of reported events; they do not by themselves establish that treatment caused an event.
A TEAE count is used to describe the safety experience during a defined observation period. It is not an efficacy endpoint and does not quantify the benefit of treatment. The primary endpoint's overall result is not reported in the registry record. The posted adverse-event summaries give some group-specific counts, but they do not substitute for a reported overall primary endpoint result or provide the full severity and relatedness breakdown described in the endpoint definition.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a completed phase 2 study of G-CSF plus plerixafor in participants with NHL or MM. Its primary endpoint concerns safety: the number of participants with TEAEs during the period from Day 1 to approximately day 38, before chemotherapy. Although the endpoint is defined, its overall result is not reported in the registry record. The record therefore does not support a numerical summary of the primary endpoint as a whole.
The adverse-event summaries report separate at-risk groups. Among the 4 participants with NHL, 2 had a serious adverse event and 4 had other adverse events. Among the 31 participants with MM, 0 had a serious adverse event and 20 had other adverse events. These are descriptive counts for the recorded groups. They should not be interpreted as a comparison between disease groups: the groups are not randomized treatment arms, and their sizes differ. The record does not report the detailed clinical profiles needed to explain differences between them.
The most frequently listed serious adverse-event terms are Pyrexia and Diabetes mellitus non-insulin-dependent, each shown as 25.0%. The registry record does not provide enough detail in the posted summary to connect those term-level percentages to a complete account of event counts, timing, severity, or treatment relatedness. Deaths are not reported in the registry record.
The study's single-group design limits causal interpretation. Without a concurrent randomized control group, observed events cannot be separated from background events or from differences in the underlying conditions and care. The secondary outcome mentioning historical controls does not resolve this limitation; its analysis and results are not reported in the registry record.
The record also describes an exploratory CD34+ cell question based on an interval of 10-11 hours and a target of at least a 2-fold increase. This is a stated study question, not a result. Results for that outcome and the other secondary outcomes are not reported in the registry record. No effect estimate, confidence interval, or statistical test result for the primary endpoint is reported there, so the precision of the overall safety estimate cannot be judged from the record.
9. Statistical Methods Explained
Reading a single-group safety summary
In a single-group study, outcomes are described among participants who received the intervention rather than contrasted with a randomized control group. For a binary outcome such as whether a participant experienced an event, a count tells how many participants met the outcome. A proportion can also describe the share of the group affected, but a count alone does not convey the uncertainty around that share. General statistical interpretation therefore distinguishes the observed count from an estimate of the underlying event frequency.
For this study, the primary endpoint is a participant count for TEAEs over a defined observation period. The registry record gives the endpoint definition and identifies the safety population, but does not report the overall endpoint result. The adverse-event summaries provide group-specific counts for serious and other events; these should be read as descriptive safety information rather than as a treatment contrast. No confidence interval or other precision estimate for the primary endpoint is reported in the registry record.
What a confidence interval would add
General explanation: a confidence interval describes the uncertainty of an estimated quantity under a statistical method. A narrow interval indicates greater statistical precision than a wide interval, while neither width nor location alone establishes causation. In a single-group study, an interval around an event proportion would describe uncertainty about that group's event frequency. It would not create a comparison with an untreated group or remove confounding. The registry record does not report a confidence interval for the primary endpoint.
Why design determines the comparison
Random allocation aims to create groups that are comparable, on average, with respect to measured and unmeasured characteristics. This study is recorded as non-randomized and single-group, so there is no concurrent randomized treatment contrast. The mention of historical controls in a secondary outcome indicates a proposed comparison with prior treatment experience, but the record does not report how those controls were selected, how differences were handled, or what the comparison found. General statistical caution is warranted because historical groups can differ from a current study group in ways that affect outcomes.
Interpreting the planning assumptions
The registry record describes planning illustrations based on an observed effect, using 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 calculations, the stated approach uses the total number of events with the Schoenfeld method; other calculations are described as participants per arm. These are assumptions for planning illustrations, not evidence that the trial itself used those design choices. They do not establish the trial's original sample-size justification, which is not reported in the registry record.
General explanation: a planning calculation asks what design size might be needed to detect a chosen effect under specified assumptions. Its result depends on the assumed effect, variability or event frequency, allocation, significance level, and analysis method. When the assumed effect comes from an observed study result, the calculation is conditional on that estimate and can be optimistic if the observed effect is imprecise. Such planning figures are not observed power and do not show that a completed study had adequate power. The registry record does not report the protocol's sample-size justification.
Multiplicity and outcome interpretation
The record lists a primary safety endpoint and multiple secondary outcomes, including a relative increase in peripheral blood CD34+ cells after the first dose, PMN engraftment timing after transplant, and a comparison of CD34+ cell increases with historical controls. When several outcomes are examined, the chance of at least one apparently unusual finding can increase if each is tested separately without a multiplicity strategy. General methods include prespecifying a primary outcome, adjusting inference across tests, or treating secondary findings as exploratory. Which approach was used for this study is not reported in the registry record. The record reports zero analyses, so it does not provide test results or an analysis plan for these outcomes.
Event counts and over-dispersion
General explanation: count outcomes can vary more than a simple statistical model expects, a feature called over-dispersion. If present, it can make standard errors too small and confidence intervals too narrow under a model that assumes less variability. This concern is distinct from a binary participant endpoint, which records whether a person had an event rather than the number of events. The primary endpoint is defined as a participant count with TEAEs, and the record does not report a model-based analysis or a check for over-dispersion.
Analysis population and missing information
The endpoint definition identifies a safety population consisting of participants who received at least 1 dose of plerixafor. The record does not report the number excluded from that population, participant-level missing outcome information, or a missing-data method. General statistical practice is to describe who was analyzed and how missing observations were handled, because those choices can affect interpretation. Without those details, the completeness of the endpoint data and the influence of missingness cannot be assessed from the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies a primary safety endpoint and lists secondary outcomes involving the relative increase in peripheral blood CD34+ cells after the first dose, PMN engraftment by the stated post-transplant time window, and comparison of CD34+ cell increases with historical controls. Examining several outcomes raises a general multiplicity issue: separate statistical tests can increase the chance of at least one apparently notable result unless the analysis plan addresses that possibility. The record does not report whether tests were adjusted, whether a hierarchy was used, or how exploratory outcomes were to be interpreted. It reports zero analyses, and results for these secondary outcomes are not reported in the registry record.
The safety endpoint defines the analysis population as all participants who received at least 1 dose of plerixafor. The record does not report a participant flow accounting, the number with missing outcome data, or a missing-data strategy. It is therefore not possible to assess from the registry record whether every eligible participant contributed complete data to the primary endpoint or how any incomplete observations were treated.
The record describes planning illustrations using a two-sided significance level of 0.05 and equal allocation between two arms, but these assumptions do not establish the study's original sample-size justification or analysis plan. The trial is listed as single-group and non-randomized, so those illustrative assumptions should not be mistaken for the trial's design. Details of interim analyses, multiplicity procedures, and the handling of missing data are not 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 |
|---|---|---|---|
| Non-Hodgkin's Lymphoma (NHL) | 2 | 4 | 50.0% |
| Multiple Myeloma (MM) | 0 | 31 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Non-Hodgkin's Lymphoma (NHL) | 4 of 4 |
| Multiple Myeloma (MM) | 20 of 31 |
Most frequent serious adverse event terms
| Event term | Organ system | Non-Hodgkin's Lymphoma (NHL) | Multiple Myeloma (MM) |
|---|---|---|---|
| Pyrexia | General disorders | 1 of 4 | 0 of 31 |
| Diabetes mellitus non-insulin-dependent | Metabolism and nutrition disorders | 1 of 4 | 0 of 31 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Non-Hodgkin's Lymphoma (NHL) | Multiple Myeloma (MM) |
|---|---|---|---|
| Hypokalaemia | Metabolism and nutrition disorders | 0 of 4 | 5 of 31 |
| Nausea | Gastrointestinal disorders | 1 of 4 | 3 of 31 |
| Pyrexia | General disorders | 1 of 4 | 3 of 31 |
| Diarrhoea | Gastrointestinal disorders | 1 of 4 | 2 of 31 |
| Vomiting | Gastrointestinal disorders | 0 of 4 | 3 of 31 |
| Anaemia | Blood and lymphatic system disorders | 0 of 4 | 2 of 31 |
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 4 | 2 of 31 |
| Headache | Nervous system disorders | 0 of 4 | 2 of 31 |
| Paraesthesia | Nervous system disorders | 1 of 4 | 1 of 31 |
| Sleep disorder | Psychiatric disorders | 0 of 4 | 2 of 31 |
Serious events with the highest arm-level rate: pyrexia (25.0%), diabetes mellitus non-insulin-dependent (25.0%).
The serious adverse-event summaries distinguish participants at risk from participants affected. In the NHL group, 4 participants were at risk and 2 were reported with serious adverse events; 4 were at risk for other adverse events and 4 were affected. In the MM group, 31 participants were at risk, 0 were reported with serious adverse events, and 20 were reported with other adverse events. These are counts, not comparative estimates of treatment risk.
The term-level serious adverse-event list names Pyrexia at 25.0% and Diabetes mellitus non-insulin-dependent at 25.0%. The registry record does not give further detail in this summary about the denominator or event-level overlap behind these percentages. It also does not report deaths. The endpoint definition says investigators assessed event severity and relatedness to treatment, but the posted group counts do not give a complete breakdown by those assessments.
Because the study had no randomized control group, these observations describe events recorded during treatment; they do not establish that treatment caused them or show how the counts compare with another regimen. The small NHL at-risk group also means its counts should be read as counts in that group, not as stable estimates applicable to a wider population.
12. What the Result Does, and Does Not, Mean
The registry record describes a study of G-CSF plus plerixafor in participants with NHL and MM, with safety as the stated primary focus. The reported adverse-event counts show that events were recorded in both disease groups, including serious events in the NHL group and none in the MM group. Those observations describe the participants included in the registry summaries. They do not establish that the intervention caused an event, or that a difference between groups reflects a difference in treatment effect.
The overall primary TEAE result is not reported in the registry record. Nor are results reported for the exploratory CD34+ cell question or the listed secondary outcomes. Consequently, the record does not provide a complete account of the primary safety endpoint, the magnitude and precision of any change in circulating CD34+ cells, or the historical-control comparison.
The study's single-group, non-randomized design is important when interpreting its observations. Without a concurrent randomized comparator, there is no direct basis for estimating how outcomes would have differed under another treatment approach. Historical controls are mentioned for a secondary outcome, but details of their use and results are not reported in the registry record. The study's stated intent to inform dosing schedules for future studies should not be confused with evidence from a randomized comparison.
General statistical interpretation separates description from inference. Counts describe what was observed in the analyzed participants; estimates and confidence intervals would help convey statistical uncertainty, while a suitable comparison would be needed to support a treatment contrast. The registry record does not report a confidence interval or test result for the primary endpoint. The available safety summaries therefore support a limited descriptive reading, not a conclusion about comparative safety or treatment benefit.
13. Why This Trial Matters Statistically
This trial is statistically instructive because it makes clear how much interpretation depends on design and reporting. The study is recorded as phase 2, non-randomized, unmasked, and single-group. Its safety endpoint is defined in terms of participants with TEAEs, but the overall primary endpoint result is not reported in the registry record. Endpoint definitions and observed results serve different purposes: the definition tells readers what was intended to be measured, while the result is needed to describe what was observed.
The separate NHL and MM safety counts also illustrate why denominators matter. The groups have different reported at-risk counts, and they are disease cohorts rather than randomized treatment arms. Their event counts cannot be read as a controlled comparison. General statistical reasoning also cautions that small groups yield imprecise estimates; the registry record does not report confidence intervals that would quantify that uncertainty for the summaries.
The study's exploratory question about the interval between plerixafor dosing and apheresis sets out a measurable biological objective, but a prespecified question is not itself a finding. The registry record does not report results for that question or the other listed secondary outcomes. This distinction helps prevent an objective from being mistaken for evidence of an observed effect.
Finally, the planning assumptions described for sample-size illustrations are not a substitute for the protocol's design rationale. They use assumptions that include equal allocation between two arms, whereas the study is recorded as single-group. Such illustrations can be useful for thinking about future research, but they are not observed power and do not explain the completed study's sample size. The original justification is not reported in the registry record.
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 consequential limitation is the absence of a reported overall result for the primary safety endpoint. The endpoint definition specifies a safety population and collection period, but the registry record does not give the overall number of participants with TEAEs or a precision estimate. The group-specific adverse-event summaries cannot fill that gap because they present serious and other events by disease group rather than a complete overall TEAE result.
The study is non-randomized and single-group, with no concurrent control. This prevents a like-for-like comparison and limits causal interpretation of event counts. The secondary outcome's reference to historical controls does not remove that concern: control selection, comparability, analysis, and results are not reported in the registry record.
The at-risk group sizes differ, and the NHL group is small. Counts from such groups are descriptive and may be imprecise; the registry record does not provide intervals to show that uncertainty. The posted safety summary also does not give the full breakdown by investigator-assessed severity or relatedness, although those assessments are described in the endpoint definition. Deaths are not reported in the registry record.
Results for the exploratory CD34+ cell outcome and other secondary outcomes are not reported. Details of the sample-size justification, interim analyses, multiplicity approach, missing-data handling, and participant flow are also not reported in the registry record. The arm descriptions are truncated after the shared initial regimen, so later treatment details are incomplete. These reporting gaps limit assessment of outcome completeness, precision, and the interpretation of the regimen's observed safety experience.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Treatment With AMD3100 (Plerixafor) in Non-Hodgkin's Lymphoma and Multiple Myeloma Patients — NCT00322842.
- Registry record last updated 2014-03-13; status last verified 2014-02.
- Results first posted 2010-11-25.
- 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.
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18. Record Summary
NCT00322842 is a completed phase 2 study of G-CSF plus plerixafor in participants with NHL or MM. The record lists enrollment of 35, a start date of 2004-09, and primary completion in 2007-02. Its design is non-randomized, unmasked, and single-group.
The primary endpoint is the number of participants with TEAEs during the period from Day 1 through approximately day 38, before chemotherapy. Its overall result is not reported in the registry record. The adverse-event summary reports serious and other event counts separately for the NHL and MM groups. The study also describes exploratory and secondary outcomes, including changes in peripheral blood CD34+ cells, but their results are not reported in the registry record.
No concurrent randomized comparator is listed. The record mentions historical controls for a secondary outcome but does not report the comparison results or details of the analysis. The available information is therefore descriptive and does not establish comparative treatment effects.