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 Romiplostim in Increasing Low Platelet Counts in Patients With Multiple Myeloma Receiving Chemotherapy (NCT01676961), a na phase 2 study. The primary outcome is Percentage of Patients Who Have Responded.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01676961 |
| Phase | Phase 2 |
| Condition | Refractory Multiple Myeloma, Stage I Multiple Myeloma, Stage II Multiple Myeloma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | n/a |
| Lead sponsor | NYU Langone Health |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned patients with multiple myeloma receiving chemotherapy and low platelet counts. The registry lists refractory and stage I, stage II, and stage III multiple myeloma, as well as thrombocytopenia, among the conditions. It does not report further eligibility criteria or a detailed description of the analyzed population. The planned enrollment was 5, and the registry records enrollment of 5.
Intervention
The intervention was romiplostim, given by subcutaneous injection once weekly for up to 6 weeks. The arm description says that patients reaching a platelet count > 50 x 10^9 could then receive weekly romiplostim during one course of chemotherapy, with treatment potentially continuing as long as benefit was seen. The registry does not report analyzed response data.
Comparator
There was no concurrent control group in this single-group design. Allocation is listed as NA, and masking as none. Supportive care (romiplostim) is the name of the registered arm, not a separate comparator. Without a control group, the record cannot provide a randomized estimate of how outcomes would have differed under another treatment or under no romiplostim.
Primary question
The primary endpoint asked what percentage of patients responded by 8 weeks. Response was defined as a platelet increase to > 50 x 10^9/L for more than 2 weeks. The registry states that the data were not analyzed because Dr. Mazumder left the institution. Thus, the record specifies the question and its definition but does not report an observed response percentage or an estimate of uncertainty.
3. Trial Design
This was a phase II, single-group trial registered as NCT01676961, sponsored by NYU Langone Health. Its title describes romiplostim for increasing low platelet counts in patients with multiple myeloma receiving chemotherapy. The registry identifies romiplostim as the intervention and lists one arm, Supportive Care (Romiplostim). Allocation is NA and masking is none. There is no registered comparison arm, so the design is not a randomized comparison of romiplostim with another treatment.
The arm description specifies weekly subcutaneous romiplostim for up to 6 weeks. Patients who achieved a platelet count > 50 x 10^9 could then receive weekly romiplostim during one course of chemotherapy and might continue for as long as benefit was seen. This describes the intended treatment pathway; it does not establish how many patients followed each part of it or how long anyone received treatment.
The registry records enrollment of 5 and gives the study status as terminated. The recorded start is 2013-01 and primary completion is 2015-11. The reason for termination is not reported in the registry record. The primary outcome entry says data were not analyzed because Dr. Mazumder left the institution. That statement explains the recorded absence of primary outcome analysis, but the record does not say whether other circumstances also contributed to termination.
These design details set limits on interpretation. A single-group study can describe outcomes observed among treated participants if those outcomes are analyzed and reported, but it cannot by itself separate treatment effects from other explanations for change. In this trial, the primary outcome was not analyzed, so the registry does not provide even a descriptive response estimate.
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 | Supportive Care (Romiplostim) |
|---|---|
| STARTED | 5 |
| COMPLETED | 0 |
| NOT COMPLETED | 5 |
Reasons for not completing this period:
| Reason | Supportive Care (Romiplostim) |
|---|---|
| Death | 3 |
| Lost to Follow-up | 1 |
| Withdrawal by Subject | 1 |
Analysis population for the primary outcome, as defined in the registry: Data was not analyzed. Dr. Mazumder left institution and data was not analyzed.
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.
Population: Confirmed diagnosis of multiple myeloma.
| Characteristic | Unit | Supportive Care (Romiplostim) |
|---|---|---|
| Participants analysed | 5 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 5 |
| Region of Enrollment: United States | Participants | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Patients Who Have Responded, measured over 8 weeks.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the percentage of patients who responded by 8 weeks. Response was defined as a platelet increase to > 50 x 10^9/L for more than 2 weeks. The definition combines a threshold with a duration requirement: a platelet count above the stated threshold alone would not meet the recorded definition unless the increase also lasted for the specified period. This makes the endpoint more specific than a measurement at a single time point.
In general, a defined endpoint helps make the clinical question measurable and clarifies which observations qualify as an outcome. A platelet response was relevant to this trial because it concerned treatment of chemotherapy-induced thrombocytopenia. The registry description says romiplostim may cause the body to make platelets after chemotherapy; that is the rationale stated in the brief summary, not a result of the trial.
The registry does not report an analyzed response percentage, the number meeting the definition, or an interval describing uncertainty. It states that the data were not analyzed because Dr. Mazumder left the institution. Therefore, the endpoint definition can be described, but the record does not establish how often response occurred or how precisely it might have been estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the registered primary endpoint has no analyzed result. The outcome was defined as a platelet increase to > 50 x 10^9/L for more than 2 weeks, assessed by 8 weeks, but the registry says the data were not analyzed because Dr. Mazumder left the institution. No response percentage, effect estimate, confidence interval, or test result is reported. Consequently, the trial record does not quantify the primary outcome.
Enrollment was 5 in a single-group study. That enrollment figure describes the registered trial, not the number who responded, completed treatment, or contributed observations to every outcome. The analysis population and outcome-specific denominators are not reported in the registry record. It would therefore be inappropriate to infer response counts or to treat enrollment as a denominator for an outcome estimate.
In general, a confidence interval expresses uncertainty around an estimate, with wider intervals indicating less precision under the method used. There is no confidence interval for the primary endpoint in the registry record, so its precision cannot be assessed from the posted results. Likewise, a p-value would address a specified statistical test, but no primary analysis or p-value is reported. The absence of those statistics should not be mistaken for evidence that an effect was either present or absent.
The design also limits what an outcome could establish even if a response estimate were available. Because the trial had no control group, a change among treated patients could not be directly contrasted with a concurrent alternative group. A before-and-after description, if reported, would not by itself isolate the contribution of romiplostim from other influences. Here, even that descriptive primary result is unavailable because the data were not analyzed.
The record lists a secondary outcome concerning the percentage of patients who experienced thrombosis or marrow fibrosis. It reports two outcome measures in total, but it does not give an analyzed result for that secondary outcome. The safety summary separately records 0 serious adverse events affecting participants and 0 other adverse events affecting participants, with 4 deaths recorded in the arm. These entries should be read as registry figures, not as a complete account of event timing, causes, or relationships to treatment.
Any sample-size values associated with the posted analysis are planning illustrations, not observed power. The planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. Those assumptions do not describe the actual single-group design, and the registry does not report an analyzed primary effect on which to base a clinical conclusion. The illustrations cannot substitute for observed results or repair the missing analysis.
9. Statistical Methods Explained
The registry identifies the design as single-group, with allocation listed as NA and masking as none. It reports phase II and an enrollment of 5. These are design and conduct descriptors, not statistical evidence of an outcome. No primary analysis is reported, and the registry lists zero analyses. In particular, there is no reported method for estimating the response percentage or its uncertainty.
What does a single-group design estimate?
In general, a single-group study can estimate an outcome among the participants observed under the study conditions, provided the outcome data are collected and analyzed. Such an estimate is descriptive: it reports what was observed in that group. Without a concurrent comparison group, it does not directly estimate the difference between romiplostim and another treatment. This distinction matters because clinical outcomes can be affected by more than the intervention being studied.
Here, the primary endpoint is not reported as analyzed, so there is no descriptive response estimate to interpret. The record also does not report an analysis population, missing-outcome handling, or the number with outcome data. The enrollment figure cannot fill those gaps. A valid endpoint denominator depends on who had evaluable data under the endpoint definition, and the record does not state that denominator.
Why does endpoint definition matter?
The response definition requires a platelet increase to > 50 x 10^9/L lasting more than 2 weeks, with the endpoint assessed by 8 weeks. In general, defining both a threshold and a duration makes the outcome rule explicit and limits ambiguity about what qualifies as a response. It also makes the observation schedule and completeness of follow-up relevant: an assessment must be capable of establishing whether the duration condition was met. The registry does not describe assessment schedules, how borderline or incomplete observations were handled, or whether any participants met the definition.
What would an uncertainty estimate add?
In general, an estimate alone does not show how much uncertainty surrounds it. A confidence interval gives a range of values compatible with the data under the chosen method and assumptions. Its width reflects precision, among other things. A statistical test and p-value instead address a specified null hypothesis under a particular analysis. These tools answer related but different questions, and neither can be interpreted without knowing the analysis and data used. No such primary estimate, interval, or test result is reported in the registry record.
Small datasets can yield imprecise estimates, but no numerical precision calculation should be inferred from enrollment alone. The actual number with evaluable endpoint information is not reported, and the observed outcome count is unavailable. The record therefore does not support a quantitative statement about the likely range of response rates.
What do the planning assumptions mean?
The planning description refers to a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effect sizes expressed as fractions of an observed effect. In general, sample-size planning uses assumptions about the effect to be detected, the variability of the outcome, the error rate, and the analysis design. A planning calculation estimates the number of participants that a proposed design would need under those assumptions. It is not a result from the trial.
The registered trial is single-group, whereas the planning description specifies equal allocation between two arms. The planning assumptions should therefore be understood as illustrations rather than a description of the trial's actual design. Sample-size illustrations are not observed power: observed power is not a substitute for reporting the estimate and its uncertainty. The primary outcome is not analyzed in the registry record, so no clinical effect estimate is available to interpret. The protocol's own sample-size justification and statistical plan are not reported in the registry record.
Why does over-dispersion matter in general?
For count outcomes, some statistical models assume a particular relationship between the average count and its variability. Over-dispersion means that observed variability exceeds what that model assumes. If it is ignored, uncertainty estimates can be too small and tests can appear more decisive than warranted. This is a general consideration for count data, not a reported feature of this trial. The registry does not provide count outcome analyses, a model specification, or evidence that over-dispersion was assessed.
Overall, the available methodological information establishes a phase II, single-group treatment study and a prespecified response definition, but it does not document how the primary outcome was analyzed. Details such as adjustment, model choice, confidence-interval construction, testing strategy, and assumptions for the primary analysis are not reported in the registry record. No particular method should be attributed to the investigators without that information.
10. Multiplicity, Missing Data and Analysis Populations
The registry names a primary outcome, the percentage of patients who responded, and lists a secondary outcome concerning the percentage who experienced thrombosis or marrow fibrosis. It also records two outcome measures in total. However, it does not report an analysis of the primary outcome, and no analyzed result for the secondary outcome is presented. The protocol's approach to multiplicity—how interpretation would account for examining more than one outcome—is not reported in the registry record.
In general, multiplicity arises when several outcomes or statistical tests are examined. Without a prespecified strategy, repeated testing can increase the chance of apparently notable findings occurring by chance. A multiplicity adjustment is not automatically needed for every descriptive report, but the intended inferential framework and outcome hierarchy should be clear. Here, no inferential results are reported, so there is no set of p-values or estimates whose multiplicity can be evaluated.
Missing-data methods are also not reported in the registry record. The primary outcome entry says that data were not analyzed because Dr. Mazumder left the institution. It does not specify whether endpoint observations were incomplete, how many participants had outcome data, or whether any missing observations were anticipated or handled. Those points cannot be inferred from the enrollment figure.
The analysis populations are not reported in the registry record. It is therefore unclear which participants would have been included in an intended primary analysis and whether the analysis would have followed a particular population definition. No assumptions about intention-to-treat, per-protocol, or evaluable populations should be assigned to this trial based on the information recorded.
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 |
|---|---|---|---|
| Supportive Care (Romiplostim) | 0 | 5 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Supportive Care (Romiplostim) | 4 of 5 |
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 |
|---|---|
| Supportive Care (Romiplostim) | 0 of 5 |
The registry's safety summary for Supportive Care (Romiplostim) records 5 participants at risk for serious adverse events and 0 affected. It also records 5 at risk for other adverse events and 0 affected. The summary separately lists 4 deaths. These figures are presented as recorded; the registry does not provide event terms for serious adverse events, as the list of top serious terms is empty.
These entries do not explain whether the deaths occurred during treatment or follow-up, their causes, or whether they were considered related to romiplostim. The record also does not clarify how the deaths relate to the adverse-event categories or provide individual event narratives. The absence of reported affected participants in the adverse-event counts should not be read as a complete account of safety when timing, definitions, ascertainment details, and relationships are not available.
In general, an adverse-event count is most informative when its observation period, event definitions, and participant denominator are clear. The registry gives at-risk counts but does not report the relevant observation period or further details about event collection. These figures are therefore limited to the specific counts entered in the record and do not support broader conclusions about safety.
12. What the Result Does, and Does Not, Mean
The registry describes a phase II study of weekly romiplostim in patients with multiple myeloma receiving chemotherapy. It defines response as a platelet increase to > 50 x 10^9/L for more than 2 weeks by 8 weeks. It also records enrollment of 5 and a single-group design. These registry entries identify the clinical question and the setting in which it was intended to be studied.
They do not establish the frequency of response. The primary outcome entry explicitly states that the data were not analyzed because Dr. Mazumder left the institution. No response percentage, estimate of uncertainty, or statistical test is reported. The absence of an analyzed result is not evidence that nobody responded, and it is not evidence that everyone responded. It means the registry does not provide an analyzed answer to the primary question.
Even with a response estimate, the lack of a control group would limit causal interpretation. In general, observing an outcome after treatment does not alone show what would have happened to the same patients under a different treatment or without the intervention. A concurrent comparator helps address that question, although its own design and analysis must also be considered. This trial's single-group structure offers no randomized contrast.
The safety summary records 0 affected participants for serious adverse events and 0 for other adverse events, alongside 4 deaths. The record does not explain the deaths' causes or relationship to treatment, or provide event terms. Those entries should not be expanded into claims about treatment-related harm or absence of harm.
Accordingly, the most defensible conclusion is about reporting rather than efficacy: the registry records the intervention, design, endpoint definition, enrollment, status, and limited safety counts, but it does not report an analyzed primary outcome. It cannot establish whether romiplostim changed platelet counts or characterize the precision of any such change.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a well-defined research question and an answered research question. The registry gives a specific response definition and a timeframe, which make the intended endpoint understandable. But an endpoint definition is not an outcome estimate. Without analyzed data, readers cannot determine the observed response percentage or its uncertainty.
The single-group design makes the distinction between description and comparison especially important. In general, a descriptive outcome among treated participants does not provide a direct estimate of the difference between treatment strategies. A comparator is needed for that contrast, and randomized allocation can help make groups comparable in expectation. This registry lists allocation as NA and has no control arm, so it does not support a randomized comparative interpretation.
The record also shows why sample-size calculations must be kept separate from observed evidence. Planning illustrations rest on assumptions about design and effect size. They can help plan a future study, but they do not demonstrate that the current study had a particular level of power or that an effect was observed. The registry does not report its own sample-size justification or statistical plan, and its primary data were not analyzed.
Finally, the safety counts and the primary efficacy endpoint answer different questions and have different limitations. Safety entries need context about event definitions, timing, and attribution; efficacy needs an analyzed outcome and clear denominator. The registry's sparse reporting means neither domain can be fully characterized. For clinical researchers, the case underscores the importance of analysis plans, complete outcome reporting, and transparent explanations when planned analyses are not completed.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most consequential trial-specific limitation is that the primary outcome data were not analyzed. The registry attributes this to Dr. Mazumder leaving the institution. It does not report a response estimate, the number meeting the response definition, or an uncertainty interval. The status is terminated, but the reason for termination is not reported in the registry record.
The enrollment was 5, and the design was single-group with no comparator. The registry does not report detailed eligibility criteria, participant characteristics, treatment adherence, follow-up completeness, or the analysis population. It also does not state how endpoint data would have been handled if incomplete. These omissions prevent assessment of who contributed to outcome data and how representative the enrolled participants were of the intended population.
The endpoint definition is explicit, but assessment procedures are not described. The record does not state when platelet counts were measured, how sustained increases were established, or how observations that could not establish the duration condition would have been classified. The registered secondary outcome about thrombosis or marrow fibrosis has no analyzed result reported.
Safety information is limited to at-risk and affected counts and a death count. The registry does not provide causes, timing, event narratives, or attribution. The protocol's own statistical plan, sample-size justification, multiplicity strategy, and analysis-population definitions are not reported in the registry record. These gaps limit interpretation independently of the absence of the primary analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Romiplostim in Increasing Low Platelet Counts in Patients With Multiple Myeloma Receiving Chemotherapy — NCT01676961.
- Registry record last updated 2018-10-15; status last verified 2018-09.
- Results first posted 2017-09-05.
- 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
NCT01676961 was a phase II, single-group trial sponsored by NYU Langone Health, titled Romiplostim in Increasing Low Platelet Counts in Patients With Multiple Myeloma Receiving Chemotherapy. The intervention was weekly subcutaneous romiplostim, initially for up to 6 weeks, with possible continuation during a course of chemotherapy for patients reaching the stated platelet threshold. The registry records enrollment of 5, status terminated, a start of 2013-01, and primary completion in 2015-11.
The primary endpoint was the percentage responding by 8 weeks, with response defined as a platelet increase to > 50 x 10^9/L for more than 2 weeks. The registry says the data were not analyzed because Dr. Mazumder left the institution. It reports no primary result or analysis. Safety entries record 0 serious adverse events, 0 other adverse events, and 4 deaths; further context about the deaths is not reported in the registry record.