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 The Effect of Rituximab on Mobilization With AMD3100 (Plerixafor) Plus G-CSF in Patients With Relapsed or Refractory Non-Hodgkin Lymphoma (NHL) or Hodgkin Disease (HD) (NCT00444912), a non-randomized phase 2 study. The primary outcome is Summary of Adverse Events (AEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00444912 |
| Phase | Phase 2 |
| Condition | Non-Hodgkin Lymphoma, Hodgkin Disease |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Genzyme, a Sanofi Company |
| Results posted | Yes |
2. Clinical Question
Population
Participants had relapsed or refractory non-Hodgkin lymphoma (NHL) or Hodgkin disease (HD) and were being considered for autologous hematopoietic progenitor cell (HPC) transplantation. The study enrolled 30 participants. The described safety population included participants who received at least 1 dose of G-CSF, plerixafor, or rituximab.
Intervention
Participants with CD20(+) lymphoma received rituximab with G-CSF and plerixafor. The brief summary describes weekly rituximab beginning 1 week before G-CSF and continuing until 2 weeks after its first dose. Participants with CD20(-) lymphoma received G-CSF and plerixafor.
Comparator
The other regimen was G-CSF and plerixafor without rituximab, assigned to participants with CD20(-) lymphoma. Because the regimens were assigned according to immunophenotype rather than by randomization, the groups are not a like-for-like control comparison.
Primary question
The posted primary outcome counts participants with adverse events from Day 1 through the day before chemotherapy, up to Day 59. It asks how many participants in each safety group had adverse events during this period; it does not, by itself, establish a causal effect of adding rituximab.
The study also listed collection and engraftment outcomes, including cumulative CD34+ cell collection, the change after plerixafor, apheresis days required to reach collection targets, and time to PMN, platelet, and lymphocyte engraftment. The registry record does not report numerical results for these outcomes. It also lists CD19+CD2-CD14- B-cell levels Six Months Post-Transplant, without a posted result.
3. Trial Design
This was a Phase 2 pilot cohort study with a parallel design and non-randomized allocation. The registry record lists enrollment of 30 and describes two treatment groups. Participants with CD20(-) lymphoma received G-CSF plus plerixafor; participants with CD20(+) lymphoma received those drugs plus rituximab. Since the treatment groups were defined by immunophenotype, treatment assignment was not independent of that characteristic. The registry record explicitly cautions that the arms are not a like-for-like control comparison.
The brief summary describes G-CSF twice daily for 4 days in both groups, followed by plerixafor on the evening of Day 4 and apheresis the next morning. G-CSF and evening plerixafor doses continued with subsequent apheresis, for up to a total of 4 aphereses or until the stated target of ≥5*10^6 CD34+ cells/kg was collected. The rituximab schedule for CD20(+) participants was weekly, beginning 1 week before G-CSF and continuing until 2 weeks after its first dose.
Participants who underwent transplantation were to be monitored for PMN, platelet, and lymphocyte engraftment. Follow-up assessments were described at 100 days, and 6 and 12 months post-transplantation. The study started in 2006-02, had primary completion in 2009-06, and is listed as completed. Masking was none. A detailed protocol, eligibility criteria beyond the described disease and treatment context, and procedures for assigning participants within immunophenotype groups 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 | G-CSF and Plerixafor | G-CSF and Plerixafor + Rituximab |
|---|---|---|
| STARTED | 15 | 15 |
| Received Plerixafor | 15 | 15 |
| Received Transplant | 14 | 14 |
| COMPLETED | 12 | 13 |
| NOT COMPLETED | 3 | 2 |
Reasons for not completing this period:
| Reason | G-CSF and Plerixafor | G-CSF and Plerixafor + Rituximab |
|---|---|---|
| Death | 1 | 2 |
| Lost to Follow-up | 1 | 0 |
| Participant Failed Mobilization | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety Population: all participants who received at least 1 dose of study drug (G-CSF, plerixafor, or rituximab)
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 | G-CSF and Plerixafor | G-CSF and Plerixafor + Rituximab | Total |
|---|---|---|---|---|
| Participants analysed | 15 | 15 | 30 | |
| Age, Continuous | years | 39.3 (SD 11.3) | 46.5 (SD 14.9) | 42.9 (SD 13.5) |
| Sex: Female, Male: Female | Participants | 6 | 3 | 9 |
| Sex: Female, Male: Male | Participants | 9 | 12 | 21 |
| Race/Ethnicity, Customized: Caucasian | Participants | 12 | 10 | 22 |
| Race/Ethnicity, Customized: African-American | Participants | 3 | 1 | 4 |
| Race/Ethnicity, Customized: Asian | Participants | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Hispanic/Latino | Participants | 0 | 2 | 2 |
| Race/Ethnicity, Customized: Other | Participants | 0 | 2 | 2 |
| Disease Diagnosis: Hodgkin disease (HD) | Participants | 14 | 4 | 18 |
| Disease Diagnosis: Non-Hodgkin lymphoma (NHL) | Participants | 1 | 11 | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Summary of Adverse Events (AEs), measured over Day 1 and up to Day 59 (maximum time before start of chemotherapy).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is the number of participants with adverse events (AEs), rather than an event rate per unit of follow-up or a measure of event severity alone. The described safety population includes participants who received at least 1 dose of G-CSF, plerixafor, or rituximab. The observation window begins on Day 1, defined according to group as the start of G-CSF mobilization or the start of rituximab, and continues until the day before chemotherapy. The maximum stated period is Day 1 through Day 59.
The investigator graded AEs using the World Health Organization (WHO) Adverse Event Grading Scale and assessed seriousness and relatedness to study treatment. Counting participants with events is a direct way to describe how many people experienced an AE during the defined safety window. It does not show the total number of events per person, their duration, or the distribution of grades. Those details are not reported in the registry record.
Safety outcomes are relevant in a mobilization study because the treatment period includes exposure to the assigned drugs and procedures before chemotherapy. However, a count of participants with AEs is descriptive. In this non-randomized study, differences between groups cannot be attributed to rituximab alone, because the groups also differ by CD20 immunophenotype. The registry record does not report a formal comparative analysis of the primary outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary outcome gives participant counts for adverse events during the stated safety period. In the G-CSF and plerixafor group, 0 of 15 participants had a serious AE and 9 of 15 had an other AE. In the G-CSF and plerixafor plus rituximab group, 1 of 15 had a serious AE and 13 of 15 had an other AE. These are counts within the two observed groups, not estimates from a randomized comparison. The registry record does not report confidence intervals, hypothesis tests, or adjusted estimates for these counts.
The serious-event counts are sparse, so they provide limited precision about how often serious AEs might occur in a broader population. General statistical interpretation: with small groups and few events, a small change in the observed count can substantially alter the apparent pattern, and uncertainty around the underlying frequency can be wide. No confidence interval is posted for the serious-event counts, so their numerical precision cannot be assessed from the registry record.
The groups are not exchangeable treatment groups: participants entered the rituximab regimen because their lymphoma was CD20(+), while participants with CD20(-) lymphoma received G-CSF and plerixafor alone. Immunophenotype therefore distinguishes the groups at the same time that the treatment regimen differs. Any observed difference in AE counts could reflect treatment, immunophenotype, other group differences, or chance. The registry record does not report a design or analysis that separates these possibilities.
The registry lists cholelithiasis and lymphoma, each at 6.7%, among the top serious AE terms. These percentages are listed without group-specific attribution in the record, so they should not be assigned to either treatment group. Death counts are not reported in the registry record. The record also lists several collection and engraftment outcomes, but numerical results and analyses for them are not reported in the registry record. Consequently, the available results support a description of the recorded safety counts, not an assessment of comparative mobilization performance or transplant outcomes.
9. Statistical Methods Explained
Statistical methods should be interpreted in light of how participants entered each group and what outcomes were reported. This study was non-randomized, and treatment was assigned according to CD20 immunophenotype. The posted arm counts therefore describe different groups of participants; they are not the equivalent of a randomized estimate of the effect of adding rituximab.
What does a participant count measure?
For a binary outcome such as whether a participant had an AE, each participant is classified as having or not having the event during the stated period. The posted results give the number with an event and the number at risk in each group. This is an intuitive descriptive summary, but it does not convey how many separate events occurred, whether events recurred, or when they occurred. A participant with several AEs still contributes as a participant with an AE to a count of affected participants. The registry record does not provide event-level details for the primary outcome.
Serious AEs and other AEs are reported separately. These categories answer different descriptive questions and should not be combined without a defined reason. The investigator assessed seriousness, grade, and relatedness, but the registry record does not give the full distribution of grades or relatedness assessments. The posted counts therefore cannot describe the severity mix or establish whether events were judged related to a study drug.
Why does non-randomized allocation matter?
Random allocation is used in comparative studies to make treatment groups more comparable, on average, with respect to measured and unmeasured characteristics. That protection is not present. Participants with CD20(+) lymphoma received rituximab with G-CSF and plerixafor, while participants with CD20(-) lymphoma received G-CSF and plerixafor. The same characteristic that determines the assigned regimen also defines a difference between the groups. A direct contrast in observed AE counts therefore combines any regimen differences with differences associated with the groups themselves.
Statistical adjustment can sometimes account for measured differences, but it cannot guarantee removal of confounding, especially when groups are defined by a treatment-selection characteristic. The registry record reports zero analyses and does not report an adjusted comparative model. No claim about a causal treatment effect follows from the raw group counts.
How should uncertainty be understood?
A confidence interval is a range of values compatible with the data under a specified statistical method. General explanation: it communicates sampling uncertainty and is often more informative than a point estimate alone. No confidence intervals for the primary AE counts are reported in the registry record. Without them, the precision of a group frequency or a contrast cannot be judged from posted inferential statistics.
For sparse outcomes, standard large-sample approximations may perform poorly; exact or other suitable methods may be considered, depending on the analysis question. This is general statistical guidance, not a description of a method used in this study. The registry record does not report the method used to analyze the primary outcome, nor does it report a statistical analysis plan.
What do planning calculations represent?
A planning illustration based on an observed effect is not a measure of the trial's achieved power. Power is a design quantity tied to assumed effects, variability, sample size, and a statistical test; calculating it after observing the results does not establish how informative the study was. The described planning assumptions use a two-sided significance level of 0.05, equal allocation, a normal approximation, and an effect to detect defined as a stated fraction of the observed effect. For time-to-event calculations, the described output is a total number of events using the Schoenfeld approach; other calculations are expressed as participants per arm.
These assumptions are planning illustrations, not evidence of the study's own design rationale. The trial's sample-size justification and any prespecified power target are not reported in the registry record. No planning result should be interpreted as an observed study outcome or as proof that the trial could detect a clinically important difference.
What analyses are available?
The registry lists 12 outcome measures and zero analyses. The primary AE counts are posted, but the registry record does not report a formal test, effect estimate, confidence interval, adjustment, or detailed missing-data method. A statistical plan, including any interim analyses, is not reported in the registry record. Interpretation should therefore remain descriptive and should not imply analyses that are not documented.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 12 outcome measures, including the primary AE outcome and secondary measures of CD34+ cell collection, apheresis days, engraftment, and B-cell levels after transplant. When a study assesses many outcomes, the chance of at least one apparently notable result can increase if each is tested separately. General statistical explanation: multiplicity procedures may control a chosen error rate or may distinguish confirmatory questions from exploratory ones. The registry record does not report a multiplicity strategy, hierarchy of testing, or prespecified statistical analysis plan. It lists zero analyses, and numerical results for the secondary outcomes are not reported in the registry record.
The primary outcome is defined for a safety population of participants who received at least 1 dose of study drug. The record gives group denominators of 15 for the posted AE summaries. That definition does not establish how missing outcome data, incomplete follow-up, or participants with partial exposure were handled. The handling of missing data and the amount of missing data are not reported in the registry record.
Only the safety population is explicitly described. The registry record does not report a separate efficacy analysis population, an intention-to-treat population, or rules for inclusion in analyses of the collection and engraftment outcomes. Without those details, the analysis populations and their relationship to all enrolled participants cannot be fully assessed. These omissions matter because different population definitions can change which participants contribute to an outcome summary.
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 |
|---|---|---|---|
| G-CSF and Plerixafor | 0 | 15 | 0.0% |
| G-CSF and Plerixafor + Rituximab | 1 | 15 | 6.7% |
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 |
|---|---|
| G-CSF and Plerixafor | 9 of 15 |
| G-CSF and Plerixafor + Rituximab | 13 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | G-CSF and Plerixafor | G-CSF and Plerixafor + Rituximab |
|---|---|---|---|
| Cholelithiasis | Hepatobiliary disorders | 0 of 15 | 1 of 15 |
| Lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 15 | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | G-CSF and Plerixafor | G-CSF and Plerixafor + Rituximab |
|---|---|---|---|
| Nausea | Gastrointestinal disorders | 1 of 15 | 4 of 15 |
| Paraesthesia oral | Gastrointestinal disorders | 2 of 15 | 2 of 15 |
| Injection site erythema | General disorders | 3 of 15 | 1 of 15 |
| Pyrexia | General disorders | 1 of 15 | 3 of 15 |
| Diarrhoea | Gastrointestinal disorders | 1 of 15 | 2 of 15 |
| Bone pain | Musculoskeletal and connective tissue disorders | 1 of 15 | 2 of 15 |
| Anaemia | Blood and lymphatic system disorders | 1 of 15 | 1 of 15 |
| Vomiting | Gastrointestinal disorders | 0 of 15 | 2 of 15 |
| Catheter site pain | General disorders | 2 of 15 | 0 of 15 |
| Injection site pain | General disorders | 1 of 15 | 1 of 15 |
Serious events with the highest arm-level rate: cholelithiasis (6.7%), lymphoma (6.7%).
The serious AE figures are participant counts: 0 of 15 in the G-CSF and plerixafor group, and 1 of 15 in the G-CSF and plerixafor plus rituximab group. The other AE counts are 9 of 15 and 13 of 15, respectively. Each denominator is the number at risk reported for that group. The figures describe the safety population during the stated period, from Day 1 until the day before chemotherapy, up to Day 59.
These raw counts should not be treated as a randomized comparison. Group assignment followed CD20 immunophenotype, so the groups differ in a characteristic linked to the regimen. The counts alone do not establish that rituximab caused a difference in AEs. With only 15 participants at risk in each group and few serious events, the serious-event frequency is especially uncertain; no confidence intervals or comparative tests are reported in the registry record.
Cholelithiasis and lymphoma are each listed at 6.7% among the top serious AE terms, but the registry record does not identify a group for either term. Deaths are not reported in the registry record. AE grades, relatedness results, recurrent-event counts, and a full event listing are also not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record describes a completed Phase 2 pilot cohort study of mobilization regimens in participants with relapsed or refractory NHL or HD before autologous HPC transplantation. Its primary posted outcome is a count of participants with AEs during the mobilization-to-chemotherapy safety period. The record reports serious AE counts of 0 of 15 with G-CSF and plerixafor and 1 of 15 with G-CSF and plerixafor plus rituximab, alongside other AE counts of 9 of 15 and 13 of 15.
Those observations describe what was recorded in these groups. They do not show that either regimen caused more or fewer AEs, because assignment was non-randomized and depended on CD20 status. Nor do the counts establish that one regimen is safer overall: the group sizes are limited, serious events are sparse, and the record does not report uncertainty intervals or a formal comparative analysis. The top serious AE terms are listed without group-specific attribution.
The trial also lists clinically relevant collection and engraftment outcomes. However, their numerical results are not reported in the registry record. The available information therefore cannot establish differences in CD34+ collection, apheresis requirements, engraftment timing, or post-transplant B-cell levels. It also cannot establish how findings might apply beyond the enrolled pilot cohort.
In short, the posted evidence supports a narrow descriptive account of the recorded AE counts and study design. It does not support a causal comparison of the regimens or a conclusion about comparative efficacy. The unreported analyses, missing-data procedures, and detailed outcome results limit further statistical interpretation.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its two regimens were not assigned at random. The treatment groups were defined by CD20 immunophenotype: CD20(-) participants received G-CSF and plerixafor, while CD20(+) participants received those drugs with rituximab. This makes the biological group distinction inseparable from the treatment contrast in the simple arm summaries. A difference in observed outcomes cannot be read as the isolated effect of adding rituximab.
The study also illustrates how a pilot cohort can provide descriptive safety information while leaving substantial uncertainty about comparative effects. The reported safety denominators are 15 per group, and serious AE counts are 0 and 1. Such counts are useful for documenting observations in the enrolled participants, but they offer limited precision about event frequencies beyond this cohort. Confidence intervals and formal comparative analyses are not reported in the registry record.
A further statistical lesson is the distinction between prespecified analysis and post hoc planning. The record describes sample-size planning assumptions based on a fraction of an observed effect, but does not report the trial's own design justification or analysis plan. Planning illustrations can help readers think about what a future study might need to detect; they do not show that this completed study had adequate power or establish an observed treatment effect.
Finally, the registry lists multiple collection, engraftment, and safety outcomes but reports zero analyses. That gap separates outcome definitions from evidence about outcome results. A careful reading distinguishes what the study intended to measure, what counts are posted, and what remains unreported.
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 principal design limitation is non-randomized allocation by CD20 immunophenotype. The treatment groups therefore differ by a characteristic that determined regimen, preventing a direct like-for-like comparison. The registry record does not describe a method that separates treatment effects from group differences, and it reports zero analyses.
The enrolled cohort was 30 participants, with 15 at risk in each posted safety group. The serious AE counts are sparse, and no confidence intervals or comparative tests are reported. AE results are limited to participant counts; event-level detail, grade distributions, relatedness findings, and the handling of recurrent events are not reported in the registry record.
Although the registry lists collection and engraftment outcomes, numerical results for those outcomes are not reported in the registry record. The record also does not report missing-data procedures, analysis populations beyond the safety population, a multiplicity approach, an interim analysis plan, or a statistical analysis plan. The stated follow-up schedule describes assessments after transplantation, but does not supply numerical follow-up findings. These limitations restrict both the precision and scope of conclusions that can be drawn from the posted results.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: The Effect of Rituximab on Mobilization With AMD3100 (Plerixafor) Plus G-CSF in Patients With Relapsed or Refractory Non-Hodgkin Lymphoma (NHL) or Hodgkin Disease (HD) — NCT00444912.
- Registry record last updated 2014-03-13; status last verified 2014-02.
- Results first posted 2010-07-29.
- 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
NCT00444912 is a completed Phase 2, non-randomized, parallel pilot cohort study sponsored by Genzyme, a Sanofi Company. It enrolled 30 participants with relapsed or refractory NHL or HD. Regimens were assigned according to CD20 immunophenotype: G-CSF and plerixafor for CD20(-) lymphoma, and those drugs plus rituximab for CD20(+) lymphoma.
The primary outcome counts participants with AEs from Day 1 until the day before chemotherapy, up to Day 59. Posted serious AE counts are 0 of 15 and 1 of 15; other AE counts are 9 of 15 and 13 of 15, respectively. The registry lists 12 outcome measures and zero analyses. Numerical results for the listed collection and engraftment outcomes are not reported in the registry record. Because treatment assignment was non-randomized and tied to immunophenotype, the safety counts are descriptive rather than a like-for-like causal comparison.