Non-inferiority Study of XM02 Filgrastim (Granix) and Filgrastim (Neupogen) in Combination With Plerixafor for Autologous Stem Cell Mobilization in Patients With Multiple Myeloma or Non-Hodgkin Lymphoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Non-inferiority Study of XM02 Filgrastim (Granix) and Filgrastim (Neupogen) in Combination With Plerixafor for Autologous Stem Cell Mobilization in Patients With Multiple Myeloma or Non-Hodgkin Lymphoma (NCT02098109), a randomized phase 2 study. The primary outcome is Comparison of the Mean Day 5 CD34+Cells/kg Yield Between the Two Arms.

2. Clinical Question

The clinical question is whether stem cell mobilization using XM02 filgrastim (Granix) with plerixafor produces a different mean Day 5 CD34+ cell yield from mobilization using filgrastim (Neupogen) with plerixafor. The trial concerns autologous stem cell mobilization in patients with multiple myeloma or non-Hodgkin lymphoma. CD34+ cell yield is reported in cells/kg and is the primary endpoint.

The comparison is between two combination regimens, not between filgrastim and no filgrastim: both arms include plerixafor. The registry describes a randomized, open-label, parallel-group Phase 2 study. Its title identifies the question as non-inferiority, but the record does not report a non-inferiority margin or a formal non-inferiority analysis. The posted primary result is a comparison of mean yields; it should therefore be read as an estimate of the observed difference, not as a standalone demonstration of non-inferiority.

The primary endpoint is accompanied by secondary outcomes concerning safety, engraftment, readmission and collection thresholds. The registry lists 9 outcome measures, while numerical results are posted for the primary outcome and selected secondary outcomes. Results for outcomes without numerical estimates are not reported in the registry record. The question addressed by the posted evidence is consequently narrower than every question listed in the study record: it describes the observed Day 5 mean yield and a subset of related outcomes, with uncertainty around the main comparison.

3. Trial Design

The registry describes a randomized, open-label, parallel-group Phase 2 study sponsored by Washington University School of Medicine. Its status is completed. The record gives a total enrollment of 100 and lists multiple myeloma and non-Hodgkin lymphoma as the conditions studied. The recorded start date is 2014-08-20, and the primary completion date is 2016-06-10.

One group received XM02 filgrastim (Granix) with plerixafor, and the comparison group received filgrastim (Neupogen) with plerixafor. The listed arm descriptions specify filgrastim at 10 mg/kg on Days 1 through 4, with additional days potentially required if the target collection goal had not been met. Plerixafor is listed at 0.24 mg/kg on Day 4, with additional days potentially required for the same reason. Apheresis is listed for Day 5, with later procedures potentially required if the target collection goal had not been met. The available arm-description text is truncated, so the complete procedural details are not reported in the registry record.

Masking is listed as none. Participants and study personnel therefore were not described as masked in the registry. The study title calls the comparison non-inferiority, but the registry record does not report the original design assumptions, the non-inferiority margin, or a sample-size justification. The outcome summaries include different denominators across outcomes; the registry record does not explain the reasons for those denominators or report participant flow and analysis-population definitions.

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

Reasons for not completing this period:

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.

6. Primary Endpoint

The registry lists the primary outcome as Comparison of the Mean Day 5 CD34+Cells/kg Yield Between the Two Arms, measured over Day 5.

The primary endpoint is the mean Day 5 CD34+ cell yield, measured in cells/kg. It describes the average number of collected CD34+ cells relative to body weight at the stated time point. The registry lists a mean and standard deviation for each arm. This is a continuous outcome: each participant’s yield can take a range of values, and the arm mean summarizes the central value of those observations.

Collection yield is relevant to a mobilization study because the study is comparing regimens intended to mobilize stem cells for collection. A mean comparison uses information across the observed yield values rather than reducing every participant to a threshold category. The reported standard deviations describe the spread of individual yields around each arm’s mean; they are not confidence intervals for the mean.

The registry reports a mean of 11.6 cells/kg for XM02 filgrastim with plerixafor and 10.0 cells/kg for filgrastim with plerixafor. The listed mean difference, expressed as the latter arm relative to the former, is -1.6 cells/kg, with a 95% confidence interval from -4.289 to 1.089. The interval includes zero, so the posted estimate does not precisely distinguish the means. Although the trial title uses the term non-inferiority, a mean comparison alone cannot establish non-inferiority without a reported margin and an appropriate analysis. Those details are not reported in the registry record.

7. Results Posted in the Registry

• Filgrastim (Neupogen) and Plerixafor: mean 10.00 versus 11.60 on control; difference -1.60 (95% CI -4.29 to 1.09), standardized effect d = -0.24; z-test p = 0.244.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Comparison of the Readmission Rate Between the Two Arms

• Endpoint type: Binary: the number of participants with the event, compared as proportions.
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Filgrastim (Neupogen) and Plerixafor: 18.0% (9/50) versus 25.0% (11/44) on control; risk difference -7.0 percentage points (95% CI -23.6 to 9.6), risk ratio 0.72 (0.33–1.57); unadjusted two-proportion z-test p = 0.408.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Filgrastim (Neupogen) and Plerixafor and the control. The interval gives the range of effects that remain compatible with the data.

Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Filgrastim (Neupogen) and Plerixafor: 96.1% (49/51) versus 95.7% (44/46) on control; risk difference 0.4 percentage points (95% CI -7.5 to 8.4), risk ratio 1.00 (0.92–1.09); unadjusted two-proportion z-test p = 0.916.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Filgrastim (Neupogen) and Plerixafor and the control. The interval gives the range of effects that remain compatible with the data.

Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Filgrastim (Neupogen) and Plerixafor: 90.2% (46/51) versus 97.8% (45/46) on control; risk difference -7.6 percentage points (95% CI -16.8 to 1.6), risk ratio 0.92 (0.83–1.02); unadjusted two-proportion z-test p = 0.119.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Filgrastim (Neupogen) and Plerixafor and the control. The interval gives the range of effects that remain compatible with the data.

The posted secondary outcomes span different parts of the mobilization and transplant pathway: collection thresholds, readmission, engraftment and safety. Their directions do not form a single uniform pattern. The selected collection results include one comparison that numerically favors the Granix combination and another that is nearly alike between groups, while the readmission estimate is numerically lower in the Neupogen combination. These descriptive directions do not establish a consistent advantage across outcomes.

These endpoints complement the primary Day 5 mean yield, but they do not replace it. Threshold outcomes classify participants according to whether a collection target was reached, whereas the primary outcome compares average yield on Day 5. A threshold may summarize a clinically recognizable target, but it discards variation among participants on the same side of that cutoff. Total collection through Day 8 and collection during one Day 5 procedure also measure different aspects of collection. Readmission addresses a later outcome rather than the primary mobilization measure.

The secondary estimates are unadjusted, and the registry record does not report a multiplicity procedure. In general, examining many outcomes increases the chance that some estimates will appear notable by chance, even when there is no underlying difference. This is one reason unadjusted secondary results carry less weight than a prespecified primary analysis. Their confidence intervals also include zero, so the posted estimates are uncertain and do not independently confirm a difference.

The registry lists 8 secondary outcomes, but numerical results are posted for only a subset. Results for the other listed secondary outcomes are not reported in the registry record. The available secondary pattern should therefore be treated as partial and exploratory, not as a comprehensive account of all outcomes specified for the study.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms
• Comparison of the Time to Neutrophil Engraftment Between the Two Arms
• Comparison of the Time to Platelet Engraftment Between the Two Arms
• Comparison of the Readmission Rate Between the Two Arms
• Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms
• Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms
• Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms
• Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms

9. Statistical Interpretation

10. Statistical Methods Explained

The primary outcome is continuous, and the registry reports an arm mean and standard deviation for each group. The comparison is expressed as a mean difference, with the Neupogen-plus-plerixafor group compared with the Granix-plus-plerixafor group. With this direction, a negative estimate means the observed mean was lower in the Neupogen group, while a positive estimate would mean it was higher. The estimate is -1.6 cells/kg.

What does the standard deviation describe?

In general, a standard deviation describes how dispersed participant-level values are around an arm mean. It is not the uncertainty interval for the estimated mean or the difference between means. The registry lists standard deviations of 6.7 for the Granix arm and 6.8 for the Neupogen arm. These summaries indicate variability among the observed yields, while the confidence interval for the mean difference describes uncertainty in the comparison.

What does the confidence interval add?

In general, a confidence interval gives a range of values compatible with the observed data under the statistical method used. It communicates both the estimated direction and the precision of the estimate. The primary 95% confidence interval runs from -4.289 to 1.089 cells/kg. Because it includes zero, it is compatible with no mean difference as well as with differences in either direction. Its width also signals that the study does not pin down the size of the mean difference narrowly. A confidence interval is not a guarantee that the true value lies in the particular interval, and it does not answer whether a difference is clinically important.

How should the p-value be understood?

The unadjusted p-value for the primary comparison is 0.244. In general, a p-value measures how unusual data at least as incompatible with a specified null hypothesis would be if that hypothesis and the model assumptions were correct. It is not the probability that the null hypothesis is true, nor a measure of the probability that the result will replicate. The registry does not report an adjustment for multiple comparisons. The p-value should therefore be read as an unadjusted result, not as a multiplicity-controlled finding.

Why does the non-inferiority label need care?

In general, a non-inferiority analysis asks whether a new approach is not worse than a comparator by more than a prespecified margin. The margin determines which losses are considered acceptable for the study question. The trial title uses non-inferiority language, but the margin, its justification and the corresponding analysis are not reported in the registry record. The posted mean difference and its confidence interval cannot substitute for those missing details. In particular, a confidence interval that includes zero does not by itself show non-inferiority.

How do the binary secondary outcomes differ?

The posted secondary outcomes include participant counts or percentages meeting collection thresholds and a count of participants readmitted up to Day 100. For binary outcomes, the comparison is expressed as a risk difference, the difference between the proportions with the outcome in the two groups. In general, a risk difference is an absolute contrast, while a risk ratio compares the proportions multiplicatively. The registry also lists risk-ratio and odds-ratio summaries for these posted outcomes. Each measure answers a different descriptive question, and none removes uncertainty due to limited outcome counts or incomplete reporting.

For the posted risk differences, the confidence intervals describe uncertainty in the absolute contrasts. The interval for total collection above 5.0x10^6 CD34+Cells/kg extends from -0.075 to 0.084. The interval for collection above 2.0x10^6 CD34+Cells/kg in one Day 5 apheresis procedure extends from -0.168 to 0.015. The readmission interval extends from -0.236 to 0.097. Each includes zero, so none of these unadjusted comparisons precisely establishes a difference in either direction.

Why can outcome definitions and denominators matter?

Binary outcome percentages depend on both the event definition and the denominator used. For example, a total-collection threshold through Day 8 is not the same endpoint as reaching a threshold in one Day 5 apheresis procedure. The registry reports varying denominators across the primary and secondary summaries. Reasons for the denominators, handling of participants without an observed outcome, and analysis-population definitions are not reported in the registry record. Without those details, the numerical summaries should be interpreted as posted rather than assumed to represent a particular intention-to-treat or per-protocol analysis.

General statistical interpretation also depends on the assumptions and analysis plan. The registry record does not report enough detail to establish whether the primary analysis adjusted for covariates, whether any interim analyses were conducted, or how missing outcome data were handled. Those features should not be inferred from the summary estimates alone.

11. Confidence Intervals and Precision

The precision of the primary estimate is best assessed from its confidence interval, not from the point estimate alone. The mean difference is -1.6 cells/kg, and the 95% confidence interval runs from -4.289 to 1.089 cells/kg. The interval includes zero and extends in both directions. The data are therefore compatible with a range of possible mean differences, including no difference and values favoring either arm under the reported comparison direction.

In general, narrower confidence intervals indicate more precise estimates, while wider intervals indicate greater uncertainty. Precision depends on factors such as sample size, outcome variability and the analysis method. The primary outcome summaries include 46 participants in the Granix arm and 51 in the Neupogen arm; the registry does not report why those analysis denominators differ from one another or from the total enrollment. The standard deviations are 6.7 and 6.8, respectively, indicating that participant-level yields vary around the arm means.

The secondary confidence intervals also include zero for each of the posted risk-difference comparisons. Those intervals are broad relative to their point estimates, so the registry results do not precisely determine the direction or magnitude of those contrasts. In general, a confidence interval containing zero is not proof of no effect. It indicates that the data and method do not rule out zero at the corresponding confidence level, while also leaving a range of nonzero values compatible with the estimate.

Precision and clinical interpretation are distinct. Even a precise estimate would need to be considered against a clinically meaningful threshold, and the non-inferiority margin is not reported in the registry record. The confidence intervals describe statistical uncertainty in the posted comparisons; they do not determine whether a difference matters clinically or satisfy a non-inferiority criterion.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are illustrations based on the observed effect, not the trial’s original design assumptions. The planning method uses a two-sided significance level of 0.05, equal allocation between the two arms and a normal approximation. It considers the full observed effect and smaller assumed effects, expressed as 100%, 75% and 50% of the observed effect. The original sample-size justification and its assumptions are not reported in the registry record.

The figures for the continuous primary outcome and the posted binary outcomes are participants per arm. They are not total sample sizes. The registry notes that time-to-event planning displays, if used, give the total number of events; the posted planning illustrations in this record concern continuous and binary outcomes. The registry’s assumption description distinguishes per-arm participant counts from total-event counts for time-to-event outcomes.

The 80% power column means the illustrated number of participants per arm for a design with an 80% chance of detecting the assumed effect under the stated planning assumptions, if that effect and the assumptions were correct. The 90% power column has the same interpretation at a 90% chance. These are prospective planning quantities, not evidence about the probability that the completed trial detected an effect.

As the assumed effect becomes smaller, more participants are needed to detect it with the same target power and significance level. This relationship is general: smaller contrasts are harder to distinguish from random variation. The planning figures are not observed power, and they do not show that the trial was or was not adequately designed. Because the calculations use observed effects for illustration rather than the trial’s own design assumptions, they should not be read as a reconstruction of the original protocol’s sample-size rationale.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, Δ is the difference in means to detect and σ is the common standard deviation.

Worked calculation for this trial

1. Observed difference Δ = -1.600; pooled standard deviation σ = 6.753.
2. n = 2 × (1.96 + 0.842)² × 6.753² / -1.600² = 279.6, rounded up to 280 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
delta <- -1.6                      # observed difference in means
sigma <- 6.75281617666211                     # pooled standard deviation
n_per_arm <- function(power, frac = 1) {
  ceiling(round(2 * (z_a + qnorm(power))^2 * sigma^2 / (delta * frac)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (t-based, so very slightly larger)
cat("power.t.test:", round(power.t.test(delta = delta, sd = sigma, power = 0.80)$n, 1), "\n")

# Simulation: exact two-sample t-test using simulated means and variances
set.seed(2098109)
n    <- n_per_arm(0.80)
reps <- 20000
m_diff <- rnorm(reps, delta, sigma * sqrt(2 / n))             # difference in sample means
s2     <- sigma^2 * rchisq(reps, 2 * (n - 1)) / (2 * (n - 1))   # pooled sample variance
tstat  <- m_diff / sqrt(s2 * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(tstat) > qt(0.975, 2 * (n - 1))), reps))

Output from running the code

100% of observed effect: n per arm = 280 (80% power), 375 (90% power)
 75% of observed effect: n per arm = 498 (80% power), 666 (90% power)
 50% of observed effect: n per arm = 1,119 (80% power), 1,498 (90% power)
power.t.test: 280.6 
Simulated power at n = 280 per arm: 0.798 (20000 replicates)

The simulation draws many sets of group means and variances at the planned sample size and counts how often the two-sample t-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Multiplicity, Missing Data and Analysis Populations

The registry lists 9 outcome measures, including the primary Day 5 mean yield and secondary outcomes covering safety, engraftment, readmission and collection thresholds. Numerical results are posted for the primary outcome and selected secondary outcomes. Results for the remaining listed outcomes are not reported in the registry record. The record also lists no reported analyses. It does not state how many comparisons were planned for formal testing or whether a hierarchy, family-wise error procedure or other multiplicity strategy was used.

In general, multiplicity matters when several hypotheses are tested. The more comparisons examined, the more opportunities there are for chance findings. An unadjusted p-value addresses a single comparison under its assumptions; it does not control the overall chance of a false-positive result across a collection of endpoints. Because the registry does not report a multiplicity plan, the secondary estimates should not be treated as confirmatory evidence simply because they are posted.

The record does not report how missing data were handled, why the outcome denominators vary, or how participants were included in each analysis. It does not identify an intention-to-treat, modified intention-to-treat or per-protocol population. The primary summaries list 46 and 51 participants in the two arms, while some secondary summaries use other denominators. These differences are reported, but their causes are not reported in the registry record. No assumptions about exclusions, incomplete follow-up or outcome availability should be made from the counts alone.

The title describes a non-inferiority study, but the registry record does not report the non-inferiority margin, the margin’s justification, or an analysis population for a formal non-inferiority test. These omissions are important because non-inferiority conclusions depend on a prespecified margin and appropriate analysis choices. The record also does not report interim analyses or the original statistical plan. The registry therefore supports description of the posted estimates, but not a reconstruction of unreported analysis decisions.

15. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: atrial fibrillation (2.2%), panic attack (2.2%), acute kidney injury (2.2%), edema - limbs (2.0%), pain (2.0%).

The registry lists serious adverse events for both arms: 2 of 46 participants in the XM02 filgrastim (Granix) plus plerixafor group and 3 of 51 participants in the filgrastim (Neupogen) plus plerixafor group. These counts describe participants with serious adverse events as recorded in the registry. They do not by themselves establish whether the regimens differ in safety, particularly because the summary does not provide a comparative uncertainty estimate or details about event attribution and follow-up.

The record also lists commonly reported serious-event terms with percentages: atrial fibrillation, panic attack and acute kidney injury at 2.2% each, and edema - limbs, pain and central line site cellulitis at 2.0% each. The registry does not clarify the denominators or whether these terms are specific to one arm in the displayed list. Death counts are not reported in the registry record.

In general, safety summaries are descriptive unless the study reports a comparative analysis and its uncertainty. The registry also reports counts of participants with other, non-serious adverse events, but those figures do not replace review of event definitions, severity, timing and attribution. Such details are not reported in the registry record.

16. What the Result Does, and Does Not, Mean

The posted primary result estimates the difference in mean Day 5 CD34+ cell yield between two combination regimens. The point estimate is -1.6 cells/kg for Neupogen plus plerixafor relative to Granix plus plerixafor, with a 95% confidence interval from -4.289 to 1.089. This interval includes zero and leaves substantial uncertainty about the direction and size of the difference. The result is a summary of the observed comparison, not proof that the regimens are equal.

The title’s non-inferiority wording should not be taken as a reported non-inferiority conclusion. The registry does not report a non-inferiority margin or the analysis needed to evaluate such a margin. A non-significant p-value does not establish equivalence or non-inferiority, and the confidence interval cannot be interpreted against a threshold that is absent from the record.

The secondary summaries provide additional descriptive information about collection thresholds and readmission, but they are unadjusted, incomplete across the outcomes listed, and uncertain. Their differing endpoint definitions mean they should not be treated as interchangeable measures of the primary question. The safety counts likewise describe recorded events and do not establish a comparative safety conclusion.

In general, trial results apply most directly to the study population and the procedures actually studied. The registry identifies patients with multiple myeloma or non-Hodgkin lymphoma and describes the regimens as including plerixafor in both arms. The available record does not provide enough detail about participant characteristics, analysis populations or missing data to determine how broadly the estimates apply. No treatment recommendation follows from these registry summaries alone.

The most defensible reading is that the registry reports an uncertain mean difference and selected secondary and safety summaries. It does not establish that either combination is superior, equivalent, or non-inferior. The absence of key protocol and analysis details limits stronger conclusions.

17. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between a study label and the evidence needed to support a particular claim. Although the registry title describes a non-inferiority study, the posted primary result is a mean difference with a confidence interval, and the record does not give the non-inferiority margin. Without that margin, readers cannot determine whether the interval excludes an unacceptable loss according to the study’s intended criterion.

The primary result also illustrates why an estimate should be read together with its precision. The point estimate alone gives one direction and magnitude, but its confidence interval includes zero and extends to values in either direction. The unadjusted p-value is not a substitute for assessing that uncertainty, and failure to meet a conventional significance threshold would not demonstrate that the groups are equivalent.

The study also contains multiple related outcomes. Mean yield, threshold collection outcomes and readmission address different questions, and the registry posts numerical results for only selected secondary outcomes. When multiple outcomes are examined without a reported multiplicity strategy, isolated results deserve caution. This is a general issue in clinical research, not a judgment about the importance of any specific endpoint.

Finally, the record shows how incomplete reporting can constrain statistical interpretation. The original sample-size rationale, analysis-population definitions, missing-data handling and full analysis plan are not reported. The planning illustrations based on observed effects cannot fill those gaps or reconstruct the original design. Careful interpretation therefore separates the numerical results that are posted from the methodological details that are absent.

18. 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.

Several trial-specific limitations constrain interpretation. The primary result is a mean comparison, but the study title identifies a non-inferiority objective and the registry does not report the non-inferiority margin or a formal analysis against it. Consequently, the posted result cannot establish whether any observed difference falls within a prespecified acceptable range.

The registry reports a total enrollment of 100, while the primary outcome summaries list 46 participants in one arm and 51 in the other. It also uses other denominators for secondary outcomes. The reasons for these denominators, participant flow, analysis-population definitions and handling of missing outcomes are not reported in the registry record. The available summaries cannot show whether the analyses followed an intention-to-treat or per-protocol approach.

The record lists 8 secondary outcomes but posts numerical results for only a subset, and it lists no reported analyses. It does not report a multiplicity strategy, interim analyses, the original statistical plan or the sample-size justification. The planning illustrations are based on observed effects rather than the original design assumptions and cannot resolve these omissions.

The study is open-label, with masking listed as none. The registry’s arm-description text is truncated, so complete procedural details are not available. These reporting limits make it difficult to assess the full context of outcome measurement and analysis. The posted data remain useful as descriptive estimates, but they do not support more definitive claims about non-inferiority, equivalence or comparative safety.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: Non-inferiority Study of XM02 Filgrastim (Granix) and Filgrastim (Neupogen) in Combination With Plerixafor for Autologous Stem Cell Mobilization in Patients With Multiple Myeloma or Non-Hodgkin Lymphoma — NCT02098109.
• Registry record last updated 2017-07-18; status last verified 2017-07.
• Results first posted 2017-07-18.
• 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.

22. Record Summary

NCT02098109 is a completed, randomized, open-label, parallel-group Phase 2 study sponsored by Washington University School of Medicine. It enrolled 100 participants with multiple myeloma or non-Hodgkin lymphoma and compared XM02 filgrastim (Granix) plus plerixafor with filgrastim (Neupogen) plus plerixafor for autologous stem cell mobilization.

The primary outcome was mean Day 5 CD34+ cell yield. The registry reports an estimated mean difference of -1.6 cells/kg for Neupogen relative to Granix, with a 95% confidence interval from -4.289 to 1.089 and an unadjusted p-value of 0.244. The interval includes zero, and the registry does not report the non-inferiority margin needed to assess the title’s stated objective formally.

Numerical results are also posted for selected secondary outcomes and safety. The original design assumptions, analysis populations, missing-data methods and multiplicity plan are not reported in the registry record.