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 Mobilization of Stem Cells With AMD3100 (Plerixafor) in Multiple Myeloma Patients (NCT00103662), a randomized phase 3 study. The primary outcome is Proportion of Participants Achieving a Target of ≥ 6*10^6 CD34+ Cells/kg in 2 or Fewer Days of Apheresis..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00103662 |
| Phase | Phase 3 |
| Condition | Multiple Myeloma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 302 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Genzyme, a Sanofi Company |
| Results posted | Yes |
2. Clinical Question
Population
People with multiple myeloma who were being considered for autologous transplantation. The primary outcome description identifies the analysis population as the Intent-to-Treat Population. The registry record does not provide further eligibility details in the available information.
Intervention
Granulocyte colony-stimulating factor (G-CSF) plus plerixafor, also called AMD3100. The registry record describes the study purpose as mobilizing and collecting stem cells for autologous transplantation.
Comparator
G-CSF plus placebo. This comparison was intended to distinguish the addition of plerixafor from G-CSF alone under the trial conditions. Detailed dosing and treatment schedules are not reported in the registry record.
Primary question
What proportion of participants achieved a collection target of at least 6*10^6 CD34+ cells/kg within 2 or fewer days of apheresis, up to Day 6? The primary outcome is described, but its arm-specific results and the estimated between-group effect are not reported in the registry record.
The central statistical question is whether the proportion meeting this collection target differed between the randomized groups. The endpoint focuses on both the amount of collected CD34+ cells and the time allowed to collect them. It is therefore a threshold-based outcome: a participant either meets the stated target within the specified apheresis period or does not. The registry record does not give a numerical result for this primary comparison, so the size, direction, and precision of any observed difference cannot be assessed from the information recorded.
3. Trial Design
This was a Phase 3, randomized, parallel-group, placebo-controlled trial with quadruple masking. The registry record lists an enrollment of 302 and identifies the condition as multiple myeloma. One group received G-CSF plus plerixafor, and the other received G-CSF plus placebo. The record describes the trial as multicenter, comparative, and double-blind in its official title; the masking field separately records quadruple masking.
The study purpose was to compare the regimens for mobilizing and collecting stem cells in people with multiple myeloma preparing for autologous transplantation. The official title specifies a collection target of at least 6*10^6 CD34+ cells/kg. The primary endpoint narrowed that target to achievement within 2 or fewer days of apheresis. Its timeframe is listed as up to Day 6, with central laboratory data from Days 5 to 6 of the Treatment/Apheresis period.
The registry record lists a start of 2005-01, a primary completion of 2006-10, and a completed status. It does not report the allocation ratio, the sequence-generation procedure, concealment details, site count, treatment doses, or the detailed schedule of visits and assessments. Although the enrollment figure is recorded, arm-specific enrollment for the primary endpoint is not reported in the registry record.
Randomization is intended to make treatment groups comparable on average, while masking is intended to reduce the influence of expectations on care, outcome assessment, and reporting. Those are general design principles; the record does not describe how these protections were implemented or assess whether masking was maintained. The primary outcome population is named as the Intent-to-Treat Population, but the record does not supply a participant flow account or explain how missing endpoint observations were handled.
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 Plus Plerixafor | G-CSF Plus Placebo |
|---|---|---|
| STARTED | 148 | 154 |
| COMPLETED | 129 | 121 |
| NOT COMPLETED | 19 | 33 |
Reasons for not completing this period:
| Reason | G-CSF Plus Plerixafor | G-CSF Plus Placebo |
|---|---|---|
| Entered Rescue Procedure | 0 | 7 |
| Death | 7 | 7 |
| Elective Withdrawal | 5 | 5 |
| Lost to Follow-up | 0 | 1 |
| Other | 6 | 9 |
| Failed mobilization | 0 | 4 |
| Intercurrent illness | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Intent-to-Treat Population
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 Plus Plerixafor | G-CSF Plus Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 148 | 154 | 302 | |
| Age, Continuous | years | 58.2 (SD 8.4) | 58.4 (SD 8.6) | 58.3 (SD 8.5) |
| Sex: Female, Male: Female | Participants | 48 | 47 | 95 |
| Sex: Female, Male: Male | Participants | 100 | 107 | 207 |
| Race/Ethnicity, Customized: Caucasian | participants | 117 | 128 | 245 |
| Race/Ethnicity, Customized: African-American | participants | 18 | 14 | 32 |
| Race/Ethnicity, Customized: Asian | participants | 1 | 3 | 4 |
| Race/Ethnicity, Customized: Hispanic/Latino | participants | 11 | 4 | 15 |
| Race/Ethnicity, Customized: Other | participants | 1 | 5 | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Participants Achieving a Target of ≥ 6*10^6 CD34+ Cells/kg in 2 or Fewer Days of Apheresis., measured over up to Day 6.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | proportion of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the proportion of participants achieving a target of at least 6*10^6 CD34+ cells/kg in 2 or fewer days of apheresis. The registry record defines the target on the basis of cells collected relative to body weight. It says that central laboratory data from Days 5 to 6 of the Treatment/Apheresis period were used and that each participant’s value was calculated as the sum of daily values collected over the 2 apheresis days. The stated timeframe is up to Day 6.
This is a binary, threshold-based endpoint. For each participant, the outcome represents whether the specified collection target was achieved within the allowed apheresis period. The group-level measure is the proportion of participants who met that criterion. Such an endpoint can express whether a clinically specified collection goal was reached within a defined period, rather than describing only the amount collected or time taken separately. That is a general explanation of the measure, not a claim about the trial’s results.
The endpoint’s definition includes the target, time limit, laboratory source, and calculation approach. The registry record does not report the numbers meeting the target in either group, the arm-specific proportions, an effect estimate, a confidence interval, or a test result. Consequently, the recorded endpoint definition alone does not establish whether the groups differed or how precisely any difference was estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record provides a clear description of the primary outcome but no numerical primary-outcome results. It therefore does not support an estimate of the proportion reaching the collection target in either group, the contrast between groups, or the uncertainty around that contrast. The direction and magnitude of the primary comparison are not reported in the registry record. No conclusion about the comparative outcome can be drawn from the endpoint definition or the trial design alone.
The primary question concerns the proportion meeting a threshold of at least 6*10^6 CD34+ cells/kg within 2 or fewer days of apheresis. The record names the Intent-to-Treat Population and describes use of central laboratory data from Days 5 to 6. It does not include the outcome counts, analysis estimate, confidence interval, or p-value needed to interpret the result statistically. The registry record also does not state the precise method used to compare proportions.
Confidence intervals, as a general statistical concept, describe a range of values compatible with the data under a specified analysis method. They help readers judge precision: a narrower interval indicates greater precision than a wider one, while an interval spanning a range of plausible effects may leave uncertainty about the direction or importance of the comparison. No confidence interval for the primary endpoint is reported in the registry record, so its precision cannot be evaluated.
The record lists secondary outcomes covering target achievement under other collection thresholds or time limits, time to collection and engraftment, graft durability, and adverse events. It does not report numerical results for those listed outcomes in the information available for this account. Nor does it provide estimates or uncertainty measures for secondary comparisons. The presence of outcome names alone is not evidence that a particular effect was observed.
Safety information is recorded separately. The registry reports serious adverse-event counts and counts for other adverse events by treatment group, along with selected serious-event terms and percentages. Those data should be read as safety summaries, not as substitutes for the primary efficacy comparison. The registry record does not report deaths in the available safety fields. These omissions limit what can be assessed from the recorded information; they do not establish that an event or analysis did not occur.
The study is listed as completed, but completion status is not itself a result. A trial can be completed while the registry entry still lacks outcome estimates or statistical details. For readers evaluating the evidence, the key limitation is therefore the absence of reported primary-outcome results and accompanying uncertainty. The record supports description of what the investigators intended to compare and how the endpoint was defined, but not a numerical interpretation of the primary treatment contrast.
9. Statistical Methods Explained
The statistical descriptions below explain general principles relevant to the outcome and design. They should not be read as a report of methods actually used unless the registry record explicitly names them. The record identifies a randomized, parallel-group trial and a binary primary endpoint, but does not report the specific statistical test, model, confidence-interval method, covariate adjustment, or analysis plan.
What does a proportion measure?
A proportion is the number of participants meeting a defined criterion relative to the number assessed for that criterion. For a binary outcome, each participant is classified according to whether the target was achieved. The group-level proportion describes the frequency of that outcome in the group. In this trial, the criterion was achieving at least 6*10^6 CD34+ cells/kg within 2 or fewer days of apheresis. The registry record does not report the numerator or denominator for the primary endpoint by group, so the proportions cannot be interpreted from the recorded information.
What would a between-group effect represent?
A treatment comparison for a binary endpoint can be expressed in different ways, such as an absolute difference in proportions or a relative measure. These summaries answer related but distinct questions. An absolute difference describes how far apart the observed proportions are; a relative measure describes their relationship on a ratio scale. The registry record does not report a primary effect estimate or specify which scale was used. No such quantity can be reconstructed from the trial enrollment figure or the safety counts.
Randomization provides a basis for comparing assigned groups because treatment assignment is determined by chance rather than by participant characteristics. In general, randomization can reduce systematic differences between groups, though it does not guarantee that every characteristic will be balanced in a particular trial. The record identifies randomization but does not describe the sequence-generation or allocation-concealment procedures, or provide baseline characteristics with which to assess group comparability.
Masking is another general design safeguard. When participants, clinicians, assessors, or other study personnel are unaware of assigned treatment, expectations may have less opportunity to influence treatment delivery or outcome assessment. The registry record lists quadruple masking and its official title describes a double-blind trial. It does not define which roles were masked, how masking was maintained, or whether it was assessed. These design labels describe the recorded design, not proof that masking was successful throughout the trial.
Why does uncertainty matter?
An observed estimate is subject to sampling variability: a different group of participants could produce a different estimate even under the same underlying conditions. A confidence interval is a general way to communicate that uncertainty alongside an estimate. A p-value, when reported, addresses compatibility of the observed data with a specified null hypothesis under the assumptions of the test; it does not measure the probability that a treatment is effective or the importance of an effect. Neither a primary confidence interval nor a p-value is reported in the registry record.
For a binary endpoint, the precision of a proportion depends on the amount of outcome information and the distribution of outcomes. A reported enrollment figure alone cannot establish the precision of the primary comparison: the number assessed for the endpoint and the observed outcome counts also matter. The registry record does not provide those primary-endpoint details. It would therefore be inappropriate to infer precision or statistical significance from enrollment alone.
What role does the analysis population play?
The primary outcome description names the Intent-to-Treat Population. As a general principle, an intention-to-treat approach analyzes participants according to their randomized assignment, regardless of whether treatment was fully received, when the data and protocol support that approach. This can preserve the benefits of randomization and address the effect of assignment. The registry record does not define the population further, state which participants were included in the primary analysis, or explain the handling of missing measurements and deviations from assigned treatment.
Missing outcome data can affect a binary comparison if the chance of missingness differs across groups or is related to the outcome. General approaches include transparent reporting of missingness, prespecified assumptions, and sensitivity analyses. The record does not report the amount or pattern of missing primary-outcome data, the assumptions used, or any sensitivity analyses. It is not possible to determine from the record whether missingness influenced the reported result.
How should planning calculations be read?
The record describes a planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation, and a normal approximation. It says that the effect to detect is a stated fraction of the observed effect and distinguishes participant-per-arm quantities from total event quantities for time-to-event outcomes. These are assumptions for planning illustrations, not a report of the trial’s original design justification. No actual planning quantities are reported in the registry entries, and the primary observed effect is not reported. Such illustrations are not observed power and do not replace an estimate with its confidence interval.
The registry record does not report a statistical analysis plan, any interim analysis, or the method used to address multiplicity. These absences matter because the chosen model, endpoint hierarchy, and analysis decisions can affect interpretation. General statistical principles can explain why those details are useful, but they cannot establish which methods this trial used. The appropriate reading is therefore descriptive: the endpoint and broad design are documented, while important inferential details and the primary result are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 10 outcome measures and names secondary outcomes that include adverse events, target achievement under other collection conditions, time to collection, time to engraftment, and graft durability at stated follow-up points. When a trial assesses several outcomes or makes several comparisons, the chance of at least one apparently unusual result can increase. This is a general multiplicity concern, not a finding about the trial. The registry record does not report a multiplicity strategy, a hierarchy of outcomes, or adjustments to account for multiple testing.
The primary endpoint is explicitly identified, but its numerical results are not reported in the information available for this account. The registry record also does not provide a statistical analysis plan, the tests used for the primary or secondary outcomes, or any interim-analysis details. It is therefore not possible to assess whether the reported analyses followed prespecified procedures or how secondary findings were intended to be interpreted.
The primary outcome description names the Intent-to-Treat Population. The record does not report the number with evaluable primary outcome data in each group, the extent or pattern of missingness, exclusions from analysis, or the method used to handle missing observations. It also does not report sensitivity analyses that might show whether conclusions depend on assumptions about missing data. These details are relevant because incomplete outcome information can affect both the estimated proportions and their uncertainty.
These gaps should be understood as limits of what is reported in the registry record, not proof that analyses or procedures were not conducted. Without the missing information, readers cannot assess multiplicity control, missing-data assumptions, or the robustness of the primary comparison.
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 Plus Plerixafor | 4 | 147 | 2.7% |
| G-CSF Plus Placebo | 6 | 151 | 4.0% |
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 |
|---|---|
| G-CSF Plus Plerixafor | 140 of 147 |
| G-CSF Plus Placebo | 140 of 151 |
Most frequent serious adverse event terms
| Event term | Organ system | G-CSF Plus Plerixafor | G-CSF Plus Placebo |
|---|---|---|---|
| Nausea | Gastrointestinal disorders | 0 of 147 | 2 of 151 |
| Bone pain | Musculoskeletal and connective tissue disorders | 1 of 147 | 1 of 151 |
| Atrial fibrillation | Cardiac disorders | 1 of 147 | 0 of 151 |
| Vomiting | Gastrointestinal disorders | 0 of 147 | 1 of 151 |
| Enterobacter bacteraemia | Infections and infestations | 0 of 147 | 1 of 151 |
| Dehydration | Metabolism and nutrition disorders | 0 of 147 | 1 of 151 |
| Hemiparesis | Nervous system disorders | 1 of 147 | 0 of 151 |
| Muscle spasticity | Nervous system disorders | 0 of 147 | 1 of 151 |
| Agitation | Psychiatric disorders | 0 of 147 | 1 of 151 |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | 0 of 147 | 1 of 151 |
Most frequent non-serious adverse event terms
| Event term | Organ system | G-CSF Plus Plerixafor | G-CSF Plus Placebo |
|---|---|---|---|
| Bone pain | Musculoskeletal and connective tissue disorders | 53 of 147 | 64 of 151 |
| Nausea | Gastrointestinal disorders | 51 of 147 | 39 of 151 |
| Fatigue | General disorders | 40 of 147 | 41 of 151 |
| Diarrhoea | Gastrointestinal disorders | 47 of 147 | 29 of 151 |
| Paraesthesia | Nervous system disorders | 33 of 147 | 34 of 151 |
| Headache | Nervous system disorders | 30 of 147 | 35 of 151 |
| Back pain | Musculoskeletal and connective tissue disorders | 23 of 147 | 34 of 151 |
| Hypokalaemia | Metabolism and nutrition disorders | 19 of 147 | 29 of 151 |
| Injection site erythema | General disorders | 32 of 147 | 5 of 151 |
| Catheter site pain | General disorders | 14 of 147 | 19 of 151 |
Serious events with the highest arm-level rate: nausea (1.3%), atrial fibrillation (0.7%), bone pain (0.7%), hemiparesis (0.7%), deep vein thrombosis (0.7%).
The registry record reports serious adverse events for the two treatment groups. In the G-CSF Plus Plerixafor group, 4 of 147 participants are recorded with a serious adverse event; in the G-CSF Plus Placebo group, 6 of 151 are recorded with a serious adverse event. The record also lists 140 participants in each group under other adverse events. These are registry counts and denominators; they should not be treated as a complete description of event severity, timing, duration, or causal attribution.
The listed serious-event terms include nausea at 1.3%, and atrial fibrillation, bone pain, hemiparesis, deep vein thrombosis, and vomiting, each at 0.7%. The registry record does not give additional context for these percentages in the available information, such as event-specific counts or timing. The selected terms do not establish that other serious events did not occur.
Deaths are not reported in the registry record. The recorded safety figures do not establish whether an event was caused by treatment, and they do not by themselves determine the overall balance of benefits and risks. They should be interpreted as the safety information recorded for the trial, with the lack of further detail kept in view.
12. What the Result Does, and Does Not, Mean
The registry record establishes the question the trial set out to address: whether adding plerixafor to G-CSF changed the proportion of participants with multiple myeloma who achieved a specified CD34+ cell collection target within 2 or fewer days of apheresis. It also documents a randomized, masked, placebo-controlled design and identifies the primary analysis population as the Intent-to-Treat Population. These registry entries describe the planned comparison and endpoint, not the observed treatment effect.
The primary endpoint results are not reported in the registry record. Without arm-specific outcome counts or proportions, an effect estimate, and an interval describing its precision, readers cannot determine the direction or magnitude of the primary comparison from this information. The record therefore does not establish whether the groups differed on the primary endpoint, nor how uncertain an estimated difference would be.
The stated enrollment and completed status do not fill that gap. Enrollment is not a measure of treatment effect, and completion does not indicate a positive, negative, or statistically conclusive result. Likewise, the outcome definitions and the biological purpose described in the trial summary do not substitute for observed outcome data.
Safety counts are recorded, but they are not a complete account of event context and do not establish causality. The registry record does not report deaths in the available safety fields. The primary outcome and safety summaries should therefore be treated separately, with neither used to infer information that is absent from the other.
In short, this record supports a description of the trial design, target population, interventions, endpoint definition, and selected safety figures. It does not support a conclusion about the primary comparative result or its precision. Any interpretation of efficacy would require the missing outcome results and relevant analysis details; those are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for a focused, threshold-based primary outcome in a randomized, parallel-group comparison. The endpoint combines a defined cell-collection target with a limit on the number of apheresis days. That makes the outcome easy to state as a participant-level criterion, while also making its interpretation dependent on the exact target, measurement window, and endpoint-analysis population. The registry record gives those core definitions and identifies central laboratory data as the source for the primary measure.
The design also illustrates how randomized assignment, masking, and a placebo comparator are intended to support a fair comparison. These are general reasons such design features matter; the registry record does not provide enough detail to assess their implementation or success. It records quadruple masking and randomization, but does not describe allocation procedures, baseline balance, or whether masking remained intact.
For statistical interpretation, the absence of reported primary results is decisive. An endpoint definition cannot answer the treatment-comparison question without the observed outcome proportions and an estimate of their uncertainty. This is a useful distinction in registry reading: a completed trial record can document design and outcomes of interest while still omitting information needed to evaluate the main result.
The listed secondary measures also show why multiplicity and outcome hierarchy matter in clinical research. Several outcomes can address different aspects of collection, recovery, durability, and safety, but they do not all answer the same question. The registry record does not report how those analyses were prioritized or how multiple comparisons were handled. The statistical significance or clinical relevance of any unreported secondary result cannot be inferred from its inclusion in the outcome list.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most important trial-specific limitation for interpretation is that the registry record does not report numerical results for the primary endpoint. It gives neither the number meeting the collection target in each group nor the estimated comparison and its precision. This prevents assessment of the primary treatment contrast, regardless of the trial’s randomized design or completed status.
The endpoint description specifies central laboratory data from Days 5 to 6 and a sum of daily values over the 2 apheresis days. However, the record does not report arm-specific evaluable denominators, missing primary measurements, or how incomplete collections and other missing values were handled. The extent to which these issues affected the comparison cannot be assessed from the registry record.
Other details important for evaluating the analysis are not reported in the registry record: the statistical test or model, confidence-interval method, covariate adjustment, prespecified analysis plan, allocation ratio, and details of sequence generation and concealment. The record also does not describe the roles included in quadruple masking or whether masking was assessed. These limitations concern available documentation and should not be taken as evidence that a procedure was not used.
The registry lists multiple secondary outcomes but does not report their numerical results or how multiplicity was addressed. Safety reporting includes selected counts and serious-event terms, but not the detailed event-level context, timing, or attribution needed for a broader safety assessment. Deaths are not reported in the registry record. Together, these reporting gaps limit the statistical and clinical interpretation possible from this record alone.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Mobilization of Stem Cells With AMD3100 (Plerixafor) in Multiple Myeloma Patients — NCT00103662.
- Registry record last updated 2014-03-13; status last verified 2014-02.
- Results first posted 2010-09-29.
- 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
The registry record describes a completed, randomized, parallel-group, placebo-controlled Phase 3 trial of G-CSF plus plerixafor versus G-CSF plus placebo in people with multiple myeloma. It records quadruple masking and enrollment of 302. The primary outcome was the proportion achieving at least 6*10^6 CD34+ cells/kg in 2 or fewer days of apheresis, with the Intent-to-Treat Population named for the primary analysis.
The record identifies secondary outcomes related to adverse events, collection targets, time to collection and engraftment, and graft durability. It also records serious adverse-event counts and selected serious-event terms. The primary outcome results, associated effect estimate and precision, detailed statistical analysis methods, and handling of missing data are not reported in the registry record. The design and endpoint can be described, but the primary comparative result cannot be evaluated from the information recorded.