Home Based Daratumumab Administration for Patients With Multiple Myeloma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Home Based Daratumumab Administration for Patients With Multiple Myeloma (NCT05511428), a na phase 4 study. The primary outcome is Treatment Satisfaction Will be Measured Using the SWT Score From the Cancer Treatment Satisfaction Questionnaire (CTSQ) - Cycle 1.

2. Clinical Question

3. Trial Design

This was a Phase 4, open-label, single-group study at Thomas Jefferson University. The registry lists 20 participants as enrolled and the study status as completed. The recorded study start date is 2022-11-08, and the primary completion date is 2024-05-22. No masking was used, and allocation is listed as not applicable.

Participants received daratumumab and hyaluronidase-fihj subcutaneously. Treatment was scheduled in the infusion center on day 1 of cycles 1, 2, 7 and 8, and at home on day 1 of cycles 3, 4, 5 and 6. Each cycle was scheduled to repeat every 28 days for 8 cycles in the absence of disease progression or unacceptable toxicity. The listed interventions also include questionnaire administration, quality-of-life assessment and interview.

The design is suited to describing experiences and outcomes within a group receiving the planned mixture of clinic and home administration. Because there is no separate control group and treatment was not masked, changes over time cannot be attributed specifically to the location of administration using this design alone. The registry record does not report a randomized comparison, a control-group result or a protocol-defined comparison between home and clinic administration.

The record lists 43 outcome measures, including medication adherence during home-treatment cycles and global health status or quality-of-life scores at specified cycles. The primary outcome is treatment satisfaction at baseline. Although the study is completed, numerical results for that primary outcome are not reported in the registry record. Further details about recruitment, participant characteristics and treatment completion 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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Although 20 participants were enrolled in the study, not all participants completed the CTSQ at each time point. As a result, only the 19 participants who provided complete and analyzable responses were included in the analysis for this outcome.

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 Treatment Satisfaction Will be Measured Using the SWT Score From the Cancer Treatment Satisfaction Questionnaire (CTSQ) - Cycle 1, measured over At Visit 1,Baseline.

The primary outcome is treatment satisfaction measured using the Satisfaction with Therapy (SWT) subscale of the Cancer Treatment Satisfaction Questionnaire (CTSQ). The registry describes the CTSQ as measuring satisfaction with cancer treatment across several domains. The SWT score ranges from 0 to 100, with a higher score representing greater satisfaction or fewer issues. The planned summary is the mean score and standard deviation at the specified time point.

The recorded timeframe is Visit 1, baseline, and the outcome title identifies Cycle 1. A baseline satisfaction measure can describe participants’ reported experience at the start of the scheduled treatment assessment. It does not, on its own, measure a change caused by home administration, particularly because the study has no separate comparison group. The registry record does not report a later SWT result as the primary endpoint or a change-from-baseline estimate.

Although 20 participants were enrolled, only 19 participants with complete and analyzable responses were included in the primary outcome analysis. The registry record does not report the numerical mean or standard deviation, individual score distribution, or a confidence interval for this outcome. It also does not report why a participant’s response was unavailable or how missing responses were handled beyond identifying the number analyzed.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry specifies a continuous primary outcome summarized by its mean and standard deviation. It does not report the numerical outcome values or a detailed statistical analysis plan. The explanations below describe the general meaning of the named statistical summaries and the limits that follow from the recorded design.

What does a mean represent?

In general, a mean is an average of the observed scores in the analysis group. For the SWT outcome, the registry specifies that the average score is to be reported on a scale from 0 to 100. A mean provides a compact description of the group’s scores, but it does not show whether individual participants had similar or very different experiences. Without the actual score distribution or mean, the registry record does not show the observed level of satisfaction.

What does the standard deviation describe?

In general, a standard deviation describes how spread out observed measurements are around their mean. A relatively larger standard deviation indicates more variability among the recorded scores; a smaller one indicates scores are more concentrated around the mean. It is a description of participant-to-participant variation, not a measure of how precisely the mean has been estimated. The registry names the standard deviation as the dispersion measure but does not report its value for the primary endpoint.

How is a confidence interval different?

In general, a confidence interval expresses uncertainty around an estimated quantity, such as a group mean. It helps readers judge the range of values compatible with the data and the analysis method. A confidence interval is not the same as the standard deviation: the latter describes variation among observations, while the former describes uncertainty in an estimate. No confidence interval for the primary endpoint is reported in the registry record.

What does a single-group design permit?

A single-group study can summarize participants’ observed scores and events under the study schedule. It does not include a concurrent control group to distinguish the effect of administration at home from other influences, such as participants’ prior experience, changes over time or other aspects of care. Open-label conduct also means the record does not describe masking of participants or investigators. These are design features, not evidence that a reported outcome is biased; they define what causal conclusions the design can support.

How should the schedule be considered?

Participants were scheduled to receive treatment in the infusion center during cycles 1, 2, 7 and 8, and at home during cycles 3, 4, 5 and 6. The registry lists adherence outcomes during the home-treatment cycles and quality-of-life outcomes at specified cycles. Repeated measurements can describe how outcomes vary across scheduled assessments, but interpreting differences over time requires knowing the measurements, the number assessed at each time point and the analysis approach. Those numerical results and details are not reported in the registry record.

The registry describes the primary outcome as a mean with standard deviation, but does not report whether any additional model or adjustment was planned. The statistical analysis plan, handling of repeated measurements, and any covariate adjustments are not reported in the registry record. Therefore, no particular unreported model or inferential procedure should be assumed.

What do the planning assumptions mean?

General sample-size planning uses assumptions about the effect to detect, the significance level, allocation and statistical approximation. Any planning illustrations associated with this record are planning scenarios, not observed power and not evidence about the study’s actual outcomes. The stated illustration assumptions use a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the effect set as a stated fraction of the observed effect. This hypothetical two-arm setup does not match the trial’s single-group design. The registry record does not report that these assumptions were used to design the trial, nor does it report a sample-size justification.

Over-dispersion is a general concern when count data vary more than a model assumes; if present, it can make uncertainty estimates from a simpler model too narrow. The primary endpoint is a continuous score, and the registry record does not describe a count-data model or an over-dispersion analysis. No specific model should be inferred from that general concept.

Finally, the record lists no analyses for the outcome data and provides no reported primary or secondary estimates. Without outcome values and analysis details, readers cannot evaluate precision, statistical significance, model fit or robustness. The outcome definitions identify what the study intended to measure; they do not substitute for the results or a complete statistical analysis plan.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Treatment (daratumumab and hyaluronidase-fihj)")
n   <- c(19)
m   <- c(87.2)
sp  <- c(10.5)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Treatment (daratumumab and hyaluronidase-fihj)  19  87.200  10.500  82.139  92.261

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 43 outcome measures, including a primary satisfaction measure, adherence outcomes across home-treatment cycles and quality-of-life assessments at several cycles. When many outcomes or time points are examined, general statistical practice recognizes the possibility of increased false-positive findings if multiple tests are conducted. Whether that concern applies to any particular reported result depends on which analyses were planned and how they were interpreted.

A multiplicity adjustment, hierarchy of outcomes or other strategy for handling multiple comparisons is not reported in the registry record. The record also lists no analyses, and numerical results and p-values are not reported. It is therefore not possible to determine whether any adjustment was used or whether any result was selected from a larger set of analyses.

For the primary outcome, 19 participants with complete and analyzable responses were included, although 20 participants were enrolled. This establishes that the primary analysis did not include every enrolled participant. The reason for incomplete responses, the pattern of missingness, any imputation, and any sensitivity analysis are not reported in the registry record. The analysis population for each secondary outcome and the number contributing data at each time point are also not reported.

In general, missing observations can affect interpretation if the participants with available data differ from those without data. The registry record does not provide information to assess that possibility. It also does not report a prespecified analysis population beyond the complete and analyzable responses identified for the primary outcome. These omissions limit evaluation of how missing data and multiple outcomes were handled.

12. Safety Results

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

Deaths

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: lung infection (5.0%), sepsis (5.0%), stomach pain (5.0%).

The registry’s adverse-event summary lists 20 participants at risk in the treatment group. It reports 3 participants affected by serious adverse events, 18 affected by other adverse events and 0 deaths. These are group-level counts and should be read as recorded safety summaries, not as a comparison with an untreated or clinic-only control group.

The listed serious adverse-event terms include lung infection, sepsis and stomach pain, each reported at 5.0%. The registry record does not provide event-level details such as timing, duration, severity grade, outcome or the number of events represented by each term. It also does not state in the information reported whether participants could contribute to more than one category. The serious and other adverse-event counts should not be added or treated as mutually exclusive categories.

These figures describe what the registry reports for this study group. Without a comparator and fuller event details, they do not establish whether an event was caused by treatment or whether the frequency differs from another setting. Additional safety details are not reported in the registry record.

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

The study asks an implementation-focused question: treatment was scheduled partly at home, and the registry lists treatment satisfaction, adherence and quality-of-life assessments as outcomes. The design and outcome definitions show what was studied and how the primary satisfaction measure was defined. They do not provide the numerical findings needed to determine the observed satisfaction scores or the reported adherence and quality-of-life results.

The registry record describes a group of 20 enrolled participants and identifies 19 complete, analyzable CTSQ responses for the primary outcome. It also reports selected adverse-event counts. These observations can describe the participants included in the study, but without numerical results for the primary and secondary outcomes, the record does not show the magnitude or precision of satisfaction, adherence or quality-of-life estimates.

The single-group design is important to interpretation. Participants experienced a planned sequence of clinic and home treatment, not a randomized assignment to home versus clinic administration. Any observed experience could reflect multiple aspects of the schedule and care. The design does not isolate the effect of administration location or support a causal comparison with standard clinic administration.

The record does not establish whether home administration reduced costs or care burden. Those potential benefits appear in the study background, but comparative cost or burden results are not reported in the registry record. Nor does the record establish a treatment effect on cancer outcomes: the primary endpoint is satisfaction, and the clinical outcome results are not reported.

Thus, the appropriate reading is limited to the documented design, outcome definitions, analysis count and safety summary. The completed status does not itself supply outcome findings. Missing numerical outcomes and the absence of a control group constrain both descriptive assessment and causal interpretation.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between a study question and the evidence needed to answer it. The registry describes an intervention schedule that includes both clinic and home administration, and it names outcomes relevant to participant experience and implementation. But without outcome estimates, the record cannot show the observed level of satisfaction or the pattern of adherence and quality-of-life scores.

The design also highlights the role of a comparison group. In a single-group study, reported outcomes can characterize the enrolled participants, while separating the effects of administration location from other influences is not possible through randomized comparison. This matters when a study’s motivation includes possible changes in cost or burden: those claims require appropriate outcomes and comparative information, neither of which is reported in the record.

Missing outcome data are another central statistical consideration. The primary analysis includes 19 complete and analyzable responses among 20 enrolled participants. That difference is documented, but the reasons for missing responses and their implications cannot be assessed from the registry record. Reporting these details alongside estimates would help readers judge how representative the analysis may be.

Finally, the record lists many outcome measures but no analyses. Multiple outcomes and time points can require a clear analysis strategy so readers can distinguish planned primary findings from exploratory results. The registry does not report such a strategy or numerical findings. These gaps demonstrate why transparent outcome reporting and analysis details are necessary for interpreting a completed clinical study.

15. 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 primary limitation is that the registry record does not report the numerical result for the primary treatment-satisfaction outcome. It provides the scale, direction and planned summary, but not the mean or standard deviation. Numerical results for the listed secondary outcomes are also not reported. Consequently, the record does not permit assessment of the size or precision of the study’s outcome estimates.

The design is open-label and single-group, with no separate control group. Participants received treatment in both the infusion center and at home according to the scheduled cycles. Thus, the study cannot isolate an effect of home administration from other influences or provide a randomized comparison with clinic administration.

For the primary endpoint, 19 participants with complete and analyzable responses were included from 20 enrolled. The reasons for incomplete data and the methods used to address missingness are not reported in the registry record. Participant characteristics, the number assessed at each secondary-outcome time point and treatment completion details are also not reported.

The record lists 43 outcome measures but does not report analyses, a statistical analysis plan, multiplicity handling, or estimates with confidence intervals. It therefore does not permit appraisal of the planned inferential approach or the consequences of examining multiple outcomes. The registry’s adverse-event summary is limited to counts and selected terms; event timing, severity, attribution and overlap are not reported.

These limitations concern what can be interpreted from the registry record and do not imply that unreported analyses were or were not conducted elsewhere.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Home Based Daratumumab Administration for Patients With Multiple Myeloma — NCT05511428.
• Registry record last updated 2025-11-21; status last verified 2025-11.
• Results first posted 2025-11-21.
• 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.

19. Record Summary

NCT05511428 is a completed Phase 4, open-label, single-group study of home-based daratumumab and hyaluronidase-fihj administration in patients with multiple myeloma. Thomas Jefferson University is listed as sponsor. The study enrolled 20 participants and recorded its start date as 2022-11-08 and primary completion date as 2024-05-22.

The primary outcome is the CTSQ Satisfaction with Therapy score at baseline, summarized as a mean and standard deviation on a 0 to 100 scale. Nineteen participants with complete and analyzable responses were included for this outcome. Numerical primary and secondary outcome results, along with analyses, are not reported in the registry record. The safety summary lists 3 participants affected by serious adverse events, 18 affected by other adverse events and 0 deaths.