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 Testing the Addition of Ixazomib/Placebo to Lenalidomide in Patients With Evidence of Residual Multiple Myeloma, OPTIMUM Trial (NCT03941860), a randomized phase 3 study. The primary outcome is Overall Survival (OS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03941860 |
| Phase | Phase 3 |
| Condition | Multiple Myeloma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Patients with evidence of residual multiple myeloma after stem cell transplantation were the intended study population. The registry record reports that only one patient was enrolled. It does not provide individual characteristics, so the enrolled patient's clinical profile and how well it represents the intended population are not reported in the registry record.
Intervention
The active-treatment arm was described as lenalidomide taken by mouth daily on days 1-28, together with ixazomib citrate taken by mouth on days 1, 8, and 15. Cycles were described as repeating every 28 days while there was no disease progression or unacceptable toxicity.
Comparator
The comparator arm was described as lenalidomide taken by mouth daily on days 1-28, together with a placebo taken by mouth on days 1, 8, and 15. The record describes both arms as receiving lenalidomide, making the addition of ixazomib the planned difference between them. It does not report which arm the one enrolled patient received.
Primary question
The primary endpoint was overall survival, defined as time from randomization to death or the date last known alive. The intended comparison concerned survival with the ixazomib combination versus lenalidomide with placebo. Because individual outcome data are withheld for confidentiality and no analyses are listed, the registry record does not establish a comparative survival result.
3. Trial Design
The registry describes OPTIMUM as a phase III, randomized, double-blind, parallel-group trial sponsored by the National Cancer Institute. Its stated focus was patients with evidence of residual multiple myeloma after stem cell transplantation. The planned comparison was lenalidomide plus ixazomib against lenalidomide plus placebo. The record reports enrollment of one patient, but does not identify the assigned arm or describe the patient's treatment exposure.
The intervention descriptions specify oral lenalidomide once daily on days 1-28 in each arm. Ixazomib or placebo was scheduled for days 1, 8, and 15, with cycles repeating every 28 days in the absence of disease progression or unacceptable toxicity. The arm descriptions also mention bone marrow aspirate and/or biopsy, PET, and CT at screening and during the study. These descriptions do not establish which assessments the enrolled patient actually completed.
The registry lists a start date of 2022-02-16, primary completion on 2023-08-06, and status as completed. The reason for completion, whether enrollment ended earlier than planned, and any changes to the planned study are not reported in the registry record. The planned enrollment target and the rationale for the enrollment observed are also not reported in the registry record.
Randomization is intended to allocate participants among treatment groups so that treatment comparisons are less vulnerable to systematic differences between groups. Double masking is intended to limit the influence of treatment knowledge on participant experience, clinical decisions, and outcome assessment. These are general design purposes; with one participant enrolled and no arm assignment or comparative outcome data reported, the registry record does not permit an assessment of how these protections operated in practice.
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 | Arm A (Lenalidomide, Ixazomib Citrate) | Arm B (Lenalidomide, Placebo) |
|---|---|---|
| STARTED | 0 | 1 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 0 | 1 |
Reasons for not completing this period:
| Reason | Arm A (Lenalidomide, Ixazomib Citrate) | Arm B (Lenalidomide, Placebo) |
|---|---|---|
| Withdrawal by Subject | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Only one patient was enrolled on this study. Due to the concern on confidentiality, individual data is not reported.
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: Only one patient was enrolled on this study. Due to the concern on confidentiality, individual data is not reported.
| Characteristic | Unit | Arm A (Lenalidomide, Ixazomib Citrate) | Arm B (Lenalidomide, Placebo) | Total |
|---|---|---|---|---|
| Participants analysed | 0 | 0 | 0 | |
| Sex: Female, Male: Female | — | — | — | |
| Sex: Female, Male: Male | — | — | — | |
| Ethnicity (NIH/OMB): Hispanic or Latino | — | — | — | |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | — | — | — | |
| Ethnicity (NIH/OMB): Unknown or Not Reported | — | — | — | |
| Race (NIH/OMB): American Indian or Alaska Native | — | — | — | |
| Race (NIH/OMB): Asian | — | — | — | |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | — | — | — | |
| Race (NIH/OMB): Black or African American | — | — | — | |
| Race (NIH/OMB): White | — | — | — | |
| Race (NIH/OMB): More than one race | — | — | — | |
| Race (NIH/OMB): Unknown or Not Reported | — | — | — |
6. Primary Endpoint
The registry lists the primary outcome as Overall Survival (OS), measured over Assessed every 3 months if patient is <2 years from randomization, every 6 months if patient is 2-5 years from randomization, annually if patient is 5 years or more from randomization..
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
Overall survival is the time from randomization until death or the date a participant was last known to be alive. It is a time-to-event endpoint: the event is death, while a participant who has not had the event by the end of available follow-up can contribute information up to the last known alive date. The registry states that survival was to be assessed every 3 months if the patient was less than 2 years from randomization, every 6 months if the patient was 2-5 years from randomization, and annually if the patient was 5 years or more from randomization.
General statistical context: overall survival is used in clinical trials because it captures time until death from any cause, rather than relying on a disease-specific measurement or a treatment response category. It can be meaningful to patients and researchers, but interpretation depends on follow-up duration, completeness of vital-status information, and the number of events. A time-to-event comparison ordinarily needs outcome and follow-up information for participants in both groups. The registry reports that only one patient was enrolled and that individual data are not reported due to confidentiality concerns. It therefore does not provide a usable comparative estimate of overall survival or its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central limitation for interpretation is the amount of outcome information made available. The registry reports one enrolled patient and says individual data are not reported because of confidentiality concerns. It lists zero analyses. As a result, no arm-specific survival estimate, treatment contrast, confidence interval, or significance test is available in the registry record. There is no basis in the posted results for estimating whether survival differed between the planned regimens or how precisely such a difference might be estimated.
The registry lists overall survival as the primary outcome and progression-free survival and best response on treatment as secondary outcomes. It reports 20 outcome measures, but does not provide a comparative result for those outcomes in the registry record for this analysis. A count of outcome measures is not itself evidence about the direction or size of a treatment effect. Nor does the absence of a posted estimate mean that the treatments had identical outcomes.
The adverse-event record lists both treatment groups with zero participants at risk, zero participants affected by serious events, zero participants at risk for other events, zero participants affected by other events, and zero deaths. These entries do not establish that no adverse events occurred. With no patient reported in the arm-level risk counts and no individual data, the entries cannot be interpreted as a comparison of safety or as a complete account of the enrolled patient's experience.
General statistical context: an estimate describes the observed treatment contrast, while its uncertainty describes how much that estimate could vary because of limited information. A confidence interval communicates this uncertainty; a wide interval generally indicates that many effect sizes remain compatible with the data. No confidence interval or underlying comparative estimate is reported, so precision cannot be characterized. A p-value, if reported, would address compatibility with a specified null model, not the clinical importance of an effect. No p-value is reported.
The trial's phase, randomization, and masking describe planned design features; they do not substitute for outcome data. The registry record does not state why enrollment was one, whether follow-up was completed, or why outcome data were withheld beyond the stated confidentiality concern. Those unknowns further limit interpretation. The appropriate reading is that the record documents a planned randomized comparison but does not support a statistical conclusion about comparative effectiveness or safety.
9. Statistical Methods Explained
The registry record identifies overall survival as the primary endpoint but lists no analyses. It does not report a statistical analysis plan, model, treatment-effect estimate, confidence interval, p-value, or analysis population. The methods below explain general statistical concepts relevant to the stated endpoint and design; they are not claims about analyses conducted in OPTIMUM.
What does a time-to-event analysis use?
General explanation: a time-to-event analysis uses both the time until an event and the information that a participant remained under observation without the event until a particular point. For overall survival, the event is death. If a participant is last known alive before a death is observed, their follow-up is usually treated as censored at that last known date. Censoring is not the same as a confirmed absence of the event after that date; it means later status is unavailable to the analysis. The registry defines the endpoint using death or date last known alive, but does not report the enrolled patient's follow-up or vital status.
When enough observations are available, a survival curve can describe the estimated probability of remaining alive over follow-up. A comparison between treatment groups can then summarize differences in survival experience. Some analyses instead estimate a relative event rate under model assumptions. The method selected, its assumptions, and any diagnostics are not reported in the registry record. With individual outcomes withheld and no analyses listed, no such curve or comparison can be assessed from the record.
Why does follow-up matter?
General explanation: the amount and distribution of follow-up influence what a survival analysis can learn. Participants who are observed for longer may contribute information about later outcomes, while those with shorter observation contribute less about long-term survival. The registry gives an intended schedule of assessments: every 3 months if the patient was less than 2 years from randomization, every 6 months if the patient was 2-5 years from randomization, and annually if the patient was 5 years or more from randomization. That schedule does not report the actual visits, duration of follow-up, or completeness of vital-status ascertainment.
In survival analysis, censoring assumptions matter. A common general assumption is that the process ending observation does not, after accounting for relevant information, systematically select participants with different future event risks. If that assumption is not reasonable, estimates may be biased. The record does not describe censoring patterns or methods for addressing them. It would therefore be inappropriate to infer that follow-up was complete or that a particular censoring assumption was checked.
What does randomization contribute?
General explanation: random allocation helps make treatment groups comparable on average with respect to measured and unmeasured characteristics, supporting a fair treatment comparison. It does not guarantee that small groups will be balanced, and it cannot create information when very few participants are enrolled. The record identifies a randomized, parallel-group, double-blind design, but reports enrollment of one patient and does not give the allocation. No estimate of baseline balance or implementation of masking can be made from the available record.
An analysis population is also important. For example, a trial might define its primary analysis according to assigned treatment, treatment received, or another prespecified rule. Each choice answers a somewhat different question and may handle departures from assigned treatment differently. The registry record does not report the analysis population or any rules for handling treatment discontinuation, crossover, or deviations from the protocol. None should be assumed.
What can be said about sample size and power?
The record includes a general description of a planning illustration based on the observed effect: a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect, and that time-to-event tables give total events while other tables give participants per arm. However, no actual observed effect or sample-size illustration values are reported in the registry record for this analysis. The planning description therefore does not supply a trial-specific target or a justification for the enrollment of one patient.
General explanation: a sample-size calculation is a planning exercise based on assumptions about the effect to detect, variability or event rate, significance threshold, and desired power. It is not evidence that an effect occurred. Power calculations based on an observed effect are especially dependent on that estimate and are not a substitute for interpreting the uncertainty in the trial results. The record does not report an original protocol calculation, its assumptions, or whether the planned enrollment changed. No claim about observed power can be made.
General explanation: confidence intervals are useful because they show a range of effect values compatible with the data and model, subject to their assumptions. Statistical significance alone would not establish the magnitude or practical importance of a difference. In OPTIMUM, there is no posted treatment-effect estimate or confidence interval to interpret. The absence of these statistics reflects the lack of reportable comparative data, not evidence that the comparison met or failed a statistical threshold.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists overall survival as the primary outcome and progression-free survival and best response on treatment as secondary outcomes. It also reports 20 outcome measures. Multiple outcomes can raise a multiplicity question: when many hypotheses are tested, the chance of at least one apparently unusual result can increase. General methods for addressing multiplicity include defining a hierarchy of outcomes, adjusting a significance threshold, or treating secondary analyses as descriptive. The registry record does not report a multiplicity strategy, the planned ordering of tests, or whether any adjustment was used. Since no analyses are listed, there are no reported hypothesis tests to interpret.
The record does not provide individual outcomes or a description of missing-data handling. For a time-to-event endpoint, a last known alive date can mark the end of observed follow-up, but the record does not say whether that information was available for the enrolled patient. It also does not report follow-up completeness, reasons for loss to follow-up, or any imputation or sensitivity analysis. These details are not reported in the registry record and cannot be inferred from the scheduled assessment intervals.
Analysis populations are likewise not specified. The record does not say whether analyses were intended to include all randomized participants, participants who received treatment, or another defined group. The allocation of the one enrolled patient is not reported. Consequently, there is no basis for identifying an intention-to-treat or per-protocol result, or for assessing departures from assigned treatment. General statistical practice requires clear definitions of the population and missing-data assumptions because both can affect interpretation; no trial-specific choices can be attributed to OPTIMUM from this record.
11. What the Result Does, and Does Not, Mean
The record establishes that OPTIMUM was registered as a phase III randomized, double-blind, parallel-group comparison of lenalidomide plus ixazomib against lenalidomide plus placebo for patients with residual multiple myeloma after stem cell transplantation. It identifies overall survival as the primary endpoint and describes a schedule for its assessment. It also lists progression-free survival and best response on treatment as secondary outcomes.
It does not establish a comparative treatment effect. Only one patient was enrolled, and individual outcome data are not reported because of confidentiality concerns. No analyses are listed, and the record does not identify the enrolled patient's arm, follow-up, survival status, or treatment experience. Therefore, no conclusion about the magnitude, direction, or precision of a difference between regimens can be drawn from the posted results.
The safety entries also should not be read as evidence that no events occurred. They list zero participants at risk and zero affected in both arms, but those counts do not provide an interpretable comparison when the record reports one enrollee and withholds individual data. A zero in these fields is not a substitute for an account of events and exposure.
General statistical context: a study's design describes how a comparison was intended to be made; results require observed data and an analysis that accounts for follow-up and uncertainty. A completed registry status does not, by itself, mean that a treatment comparison is estimable or that a particular hypothesis was confirmed. For this record, the defensible conclusion is about reporting and available information: it documents the trial plan and a very limited enrollment, but does not provide reportable comparative outcome evidence. This is not evidence that the two regimens are equivalent, nor that one is superior.
12. Why This Trial Matters Statistically
This record illustrates the distinction between a trial's design and the evidence available for estimating a treatment effect. OPTIMUM was registered as a phase III randomized, double-blind trial, with a clinically defined comparison and overall survival as its primary endpoint. Yet the record reports enrollment of one patient and withholds individual outcome data for confidentiality. Those details sharply limit what statistical inference can be drawn, regardless of the planned design.
For biostatisticians and clinical researchers, the case underscores why enrollment and outcome availability are central to interpretation. Randomization can support causal comparisons when enough usable information is collected, but it does not overcome an absence of reportable outcomes. Likewise, a stated primary endpoint and an assessment schedule do not reveal the number of events, duration of follow-up, or precision of an estimate. Those elements are needed to understand a time-to-event result.
The record also highlights the need to distinguish administrative status from analytical conclusions. The study is marked completed, but the reason for completion and any departure from the intended plan are not reported in the registry record. The record lists outcome measures but no analyses. Neither the status nor the listed endpoints can stand in for results.
Finally, sparse reporting requires restraint. The appropriate statistical account is not to infer a null effect from missing estimates, or to treat zero-valued safety counts as proof of no events. It is to identify what the record documents, distinguish general methodological principles from trial-specific methods, and state clearly where comparative evidence is unavailable. That discipline preserves the difference between no demonstrated effect and no reportable estimate.
13. 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 direct limitation is that the registry reports enrollment of one patient and does not report individual outcome data because of confidentiality concerns. It does not identify the patient's treatment assignment, baseline characteristics, treatment received, or follow-up. Without these details, there is no arm-level comparison of overall survival, progression-free survival, or best response that can be interpreted.
The registry lists zero participants at risk and zero affected for both arms in the adverse-event entries, as well as zero deaths. These entries cannot be treated as evidence of no adverse events or as complete safety results, particularly because individual data are withheld and the arm receiving the enrolled patient is not identified. The record does not describe the safety observation period or the basis for the counts.
Although the trial status is completed, the reason for completion and the relationship between the single enrollee and the intended enrollment are not reported in the registry record. The planned enrollment target, sample-size justification, statistical analysis plan, and any changes to the protocol are also not reported. The registry provides a generic planning-illustration description, but no trial-specific planning values or observed treatment-effect estimate are available in the registry record used for this analysis.
Other limitations include the absence of a reported analysis population, missing-data strategy, multiplicity plan, and actual follow-up details. The registry lists 20 outcome measures and zero analyses, leaving no results with which to assess selective reporting or consistency across outcomes. These gaps prevent a detailed evaluation of protocol adherence, statistical assumptions, and the precision or robustness of any treatment comparison.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Testing the Addition of Ixazomib/Placebo to Lenalidomide in Patients With Evidence of Residual Multiple Myeloma, OPTIMUM Trial — NCT03941860.
- Registry record last updated 2025-05-23; status last verified 2025-05.
- Results first posted 2024-08-01.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
17. Record Summary
OPTIMUM is registered as a phase III, randomized, double-blind, parallel-group trial sponsored by the National Cancer Institute. It was designed to compare lenalidomide plus ixazomib with lenalidomide plus placebo in patients with residual multiple myeloma after stem cell transplantation. The registry lists overall survival as the primary outcome, with progression-free survival and best response on treatment as secondary outcomes.
The record reports enrollment of one patient, a start date of 2022-02-16, primary completion on 2023-08-06, and completed status. Individual outcome data are not reported because of confidentiality concerns, and the record lists no analyses. The patient's arm, follow-up, and outcome are not identified. Consequently, the registry documents the planned comparison but does not provide an interpretable estimate of comparative efficacy or safety.