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 Ixazomib With Cyclophosphamide and Dexamethasone in Patients With Previously Untreated Symptomatic Multiple Myeloma or Light Chain Amyloidosis (NCT01864018), a na phase 1, phase 2 study. The primary outcome is Maximum Tolerated (MTD) Dose of Cyclophosphamide With Ixazomib and Dexamethasone (Phase I).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01864018 |
| Phase | Phase 1, Phase 2 |
| Condition | Amyloidosis, Plasma Cell Myeloma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 87 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Mayo Clinic |
| Results posted | Yes |
2. Clinical Question
Population
People with previously untreated symptomatic multiple myeloma or light chain amyloidosis. The registry record reports enrollment of 87 and describes 10 patients accrued to the Phase I portion for the maximum tolerated dose assessment.
Intervention
Ixazomib citrate, cyclophosphamide, and dexamethasone. The induction description specifies oral ixazomib on days 1, 8, and 15, and oral cyclophosphamide and dexamethasone on days 1, 8, 15, and 22, repeating every 28 days for up to 12 courses if disease progression or unacceptable toxicity does not occur.
Comparator
There was no concurrent control group. The registry record classifies the study as single-group, with allocation listed as NA and masking as none. The cohorts therefore should not be read as randomized treatment arms or as a like-for-like control comparison.
Primary question
For the Phase I portion, what is the maximum tolerated dose of cyclophosphamide when given with ixazomib and dexamethasone? The registry defines the endpoint using dose-limiting toxicity assessed at 28 days. It does not report the resulting dose.
The study also lists safety and time-to-event outcomes, including grade 3 or greater adverse events at least possibly related to treatment, progression-free survival, and survival time. Those outcome labels indicate what was tracked, but the registry record does not provide numerical results for these outcomes in the registry record for this analysis. Consequently, the main statistical question that can be explained from the record is the design and meaning of the dose-finding endpoint, alongside the limits of the posted safety counts.
3. Trial Design
This was a Phase 1/2 study with a single-group model. The registry record lists allocation as NA and masking as none. It reports enrollment of 87 and classifies the study as completed. The listed condition categories are amyloidosis and plasma cell myeloma; the stated population is previously untreated symptomatic multiple myeloma or light chain amyloidosis.
The treatment combined ixazomib citrate, cyclophosphamide, and dexamethasone. The induction regimen describes oral ixazomib on days 1, 8, and 15, with oral cyclophosphamide and dexamethasone on days 1, 8, 15, and 22. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. The arm description begins a maintenance therapy description but is incomplete in the registry record, so its full details are not reported in the registry record.
The primary outcome is a Phase I maximum tolerated dose measure for cyclophosphamide with ixazomib and dexamethasone. The record says the 10 patients accrued to the Phase I portion were analyzed for this dose determination. It defines the maximum tolerated dose in relation to the dose level below the lowest level that induces dose-limiting toxicity in at least 1/3 of patients, assessed using National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. The timeframe is at 28 days. The registry record does not report the dose selected under this rule.
Listed secondary outcomes include grade 3 or greater adverse events at least possibly related to treatment, progression-free survival, and survival time. Laboratory biomarker analysis, pharmacological study, quality-of-life assessment, and questionnaire administration are also listed. No randomized contrast is described, so differences between the named cohorts cannot be interpreted as a randomized treatment effect.
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.
Before assignment: The phase I participants are grouped with the phase II, cohort A participants because they were grouped in the published manuscript
Overall Study
| Stage | Phase I/II, Cohort A | Phase II, Cohort B |
|---|---|---|
| STARTED | 51 | 36 |
| Accrued During Phase I | 10 | 0 |
| Accrued During Phase II | 41 | 36 |
| COMPLETED | 48 | 35 |
| NOT COMPLETED | 3 | 1 |
Reasons for not completing this period:
| Reason | Phase I/II, Cohort A | Phase II, Cohort B |
|---|---|---|
| Ineligible | 3 | 1 |
Analysis population for the primary outcome, as defined in the registry: The 10 patients accrued to the phase I portion of the study were analyzed to determine the maximum tolerated dose of ixazomib with cyclophosphamide and dexamethasone
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Phase I/II, Cohort A | Phase II, Cohort B | Total |
|---|---|---|---|---|
| Participants analysed | 48 | 35 | 83 | |
| Age, Continuous | years | 64.5 (41 to 88) | 67 (38 to 78) | 65 (38 to 88) |
| Sex: Female, Male: Female | Participants | 23 | 11 | 34 |
| Sex: Female, Male: Male | Participants | 25 | 24 | 49 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): White | Participants | 46 | 35 | 81 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 1 |
| Region of Enrollment: United States | participants | 48 | 35 | 83 |
| ECOG Performance Status: 0 | Participants | 25 | 16 | 41 |
| ECOG Performance Status: 1 | Participants | 19 | 17 | 36 |
| ECOG Performance Status: 2 | Participants | 4 | 2 | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated (MTD) Dose of Cyclophosphamide With Ixazomib and Dexamethasone (Phase I), measured over At 28 days.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg weekly |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the maximum tolerated dose of cyclophosphamide when given with ixazomib and dexamethasone, expressed in mg weekly. It is a dose-finding outcome rather than a measure of comparative efficacy. The registry defines the maximum tolerated dose as the dose level below the lowest dose that induces dose-limiting toxicity in at least 1/3 of patients. Toxicity assessment is specified at 28 days and uses National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0.
In general, a maximum tolerated dose rule is used in early-phase studies to guide dose selection by relating dose levels to observed toxicity. It is important to distinguish this endpoint from a claim about clinical benefit: it describes a dose-selection criterion and does not by itself establish whether a regimen improves survival or disease control. The study also lists progression-free survival, survival time, and treatment-related grade 3 or greater adverse events as secondary outcomes, but the numerical results for these outcomes are not reported in the registry record.
The record states that the 10 patients accrued to the Phase I portion were analyzed to determine the maximum tolerated dose. However, the endpoint result itself is not reported in the registry record. Without that result, the dose selected by the prespecified definition cannot be identified from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes an early-phase, single-group investigation rather than a randomized comparison. Its primary question concerns dose selection: the maximum tolerated dose of cyclophosphamide in combination with ixazomib and dexamethasone. This kind of endpoint is different from a direct estimate of treatment benefit. The registry supplies the dose-limiting toxicity rule and the assessment timeframe, but it does not report the resulting dose or the toxicity observations used to reach it.
The study is listed as completed, with enrollment of 87. That overall enrollment figure does not replace the specifically described Phase I population of 10 patients used for the maximum tolerated dose assessment. Nor does it identify the number contributing to each secondary outcome. Those outcome-specific analysis populations are not reported in the registry record.
The safety information lists two cohorts. Phase I/II, Cohort A reports 48 at risk for serious adverse events, with 16 affected; its other-adverse-event figures report 48 at risk and 48 affected, and the death count is 2. Phase II, Cohort B reports 35 at risk for serious adverse events, with 15 affected; its other-adverse-event figures report 35 at risk and 35 affected, and the death count is 7. These are descriptive counts. The record does not establish that the cohorts were randomized or comparable, and the groups should not be treated as a controlled contrast.
The listed serious terms include lymphocyte count decreased at 10.4%, atrial fibrillation at 8.6%, heart failure at 8.6%, lung infection at 8.6%, platelet count decreased at 5.7%, and syncope at 5.7%. These terms describe reported events, not a causal estimate attributable to treatment. The registry also lists a separate outcome for grade 3 or greater adverse events at least possibly related to treatment, but its results are not reported in the registry record. Serious adverse event counts and grade-based, treatment-related adverse event outcomes are not interchangeable.
Progression-free survival and survival time are listed as secondary outcomes, but their results are not reported in the registry record. With no posted effect estimates, uncertainty intervals, or analysis details for those outcomes, the record cannot support a quantitative interpretation of disease control or survival. The registry reports nine outcome measures and no analysis records. The absence of analysis records means statistical details such as model specification, handling of censoring, and uncertainty estimates are not reported in the registry record.
9. Statistical Methods Explained
What does a single-group design tell the reader?
The registry classifies the study as single-group, with allocation listed as NA and masking as none. In general, a single-group study can describe outcomes observed among people receiving the study regimen, but it does not provide a concurrent randomized control for estimating what would have happened under another treatment. Any comparison between Cohort A and Cohort B would therefore be descriptive unless the record posted a design and analysis establishing otherwise. It does not.
This matters because differences in observed outcomes across groups can reflect differences in who entered each cohort, how follow-up occurred, or other factors, as well as treatment exposure. Without random allocation and relevant analysis details, the cohort labels alone do not support a causal comparison. The registry itself flags the study as not a randomized comparison and says the arms are not a like-for-like control comparison.
How should the dose-finding endpoint be understood?
The primary endpoint is a maximum tolerated dose, not a response rate or survival measure. The registry defines it as the dose level below the lowest dose that induces dose-limiting toxicity in at least 1/3 of patients, based on adverse-event assessment at 28 days. In general, this rule uses observed toxicity across dose levels to guide selection of a dose for further study. Its interpretation depends on which dose levels were studied, how many patients received each dose, which events met the dose-limiting definition, and whether the planned assessment period was completed. Those details and the selected dose are not reported in the registry record.
Early-phase dose-finding estimates can be imprecise because they are based on limited observations at each dose level. This is a general statistical consideration, not a statement about the specific precision in this study: the record does not provide the dose-level data or an uncertainty interval for the selected dose. The primary endpoint description does not provide enough information to reconstruct the dose-finding analysis.
What do adverse-event counts describe?
Counts of participants affected describe the number recorded with an event under the stated cohort and denominator. They do not, by themselves, measure how likely an event would be in a comparable untreated population. General safety interpretation also depends on event definitions, observation time, severity, relationship assessment, and completeness of reporting. The registry provides counts for serious and other adverse events and lists selected serious terms with percentages, but the corresponding detailed event-level data and full analysis methods are not reported in the registry record.
The listed secondary outcome of grade 3 or greater adverse events at least possibly related to treatment has a severity threshold and a relationship qualifier. That is a distinct outcome from the general serious adverse event counts. Seriousness and severity are separate concepts in adverse-event reporting; an event can be classified by its seriousness and also receive a severity grade. The registry record does not provide the numerical results for the grade-based, possibly treatment-related outcome.
How should time-to-event outcomes be assessed?
Progression-free survival and survival time are time-to-event outcomes. In general, their analysis must account for follow-up duration and participants whose event has not been observed by the end of observation. Such observations are often described as censored. The choice of time origin, event definition, censoring rules, estimator or model, and uncertainty interval affects interpretation. These analysis details and the numerical results are not reported in the registry record, so no survival estimate or comparison can be inferred from the listed outcome names.
What do the planning assumptions mean?
The sample-size planning illustration is described as a calculation based on the observed effect, not as the trial's original design. Its stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. For time-to-event outcomes, the illustration gives total events, while other outcomes use participants per arm. These are hypothetical planning assumptions, not features of the single-group trial design and not observed power. Equal allocation between two arms in a planning illustration does not convert this study into a randomized two-arm trial.
In general, a confidence interval communicates the range of effect values compatible with the data and analysis under its assumptions. A narrow interval indicates greater precision than a wide interval, but neither guarantees that an estimate is unbiased or clinically important. The registry record does not report confidence intervals or effect estimates for the listed secondary outcomes. Over-dispersion, in general, occurs when variation in count data exceeds what a simple model assumes; accounting for it can affect uncertainty estimates. The record does not state whether count models or over-dispersion adjustments were used.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists nine outcome measures, including the primary maximum tolerated dose endpoint and secondary outcomes for adverse events, progression-free survival, and survival time. It does not report a plan for controlling multiplicity across outcomes or across any subgroup, cohort, or time-based analyses. A multiplicity plan is important when many statistical questions are assessed, because repeated testing can increase the chance of at least one apparently notable result by chance. This is a general issue; no multiplicity adjustment should be assumed for this study.
The registry reports no analysis records. It does not provide outcome-specific analysis populations, statistical models, rules for censoring time-to-event outcomes, or methods for handling missing observations. Missing data can affect interpretation when the probability of an observation being missing is related to a participant's health or outcome. General methods include describing the amount and pattern of missingness and testing how assumptions about missing outcomes affect estimates. The methods actually used in this trial are not reported in the registry record.
The primary endpoint description specifies that 10 patients accrued to the Phase I portion were analyzed to determine the maximum tolerated dose, but the record does not report the dose-level allocations or whether all relevant toxicity assessments were complete. For the secondary outcomes, the registry does not identify which enrolled participants contributed data or how follow-up ended. These gaps limit assessment of completeness and the population represented by any estimate.
Analysis-population definitions also matter in general. An intention-to-treat population, a safety population, and a population with evaluable endpoint data can contain different participants, depending on the study design and definitions. The registry record does not state such definitions for the outcomes. No population should therefore be inferred beyond the stated Phase I dose-finding population and the cohort denominators reported for adverse events.
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 |
|---|---|---|---|
| Phase I/II, Cohort A | 16 | 48 | 33.3% |
| Phase II, Cohort B | 15 | 35 | 42.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Phase I/II, Cohort A | 2 of 48 |
| Phase II, Cohort B | 7 of 35 |
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 |
|---|---|
| Phase I/II, Cohort A | 48 of 48 |
| Phase II, Cohort B | 35 of 35 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase I/II, Cohort A | Phase II, Cohort B |
|---|---|---|---|
| Lymphocyte count decreased | Investigations | 5 of 48 | 0 of 35 |
| Atrial fibrillation | Cardiac disorders | 1 of 48 | 3 of 35 |
| Heart failure | Cardiac disorders | 0 of 48 | 3 of 35 |
| Lung infection | Infections and infestations | 0 of 48 | 3 of 35 |
| Platelet count decreased | Investigations | 1 of 48 | 2 of 35 |
| White blood cell decreased | Investigations | 2 of 48 | 1 of 35 |
| Hyperglycemia | Metabolism and nutrition disorders | 2 of 48 | 1 of 35 |
| Urinary tract infection | Infections and infestations | 1 of 48 | 1 of 35 |
| Neutrophil count decreased | Investigations | 2 of 48 | 0 of 35 |
| Peripheral sensory neuropathy | Nervous system disorders | 1 of 48 | 1 of 35 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase I/II, Cohort A | Phase II, Cohort B |
|---|---|---|---|
| Fatigue | General disorders | 47 of 48 | 32 of 35 |
| Constipation | Gastrointestinal disorders | 36 of 48 | 25 of 35 |
| Peripheral sensory neuropathy | Nervous system disorders | 35 of 48 | 19 of 35 |
| Nausea | Gastrointestinal disorders | 29 of 48 | 22 of 35 |
| Lymphocyte count decreased | Investigations | 42 of 48 | 9 of 35 |
| White blood cell decreased | Investigations | 42 of 48 | 6 of 35 |
| Diarrhea | Gastrointestinal disorders | 29 of 48 | 15 of 35 |
| Platelet count decreased | Investigations | 31 of 48 | 11 of 35 |
| Neutrophil count decreased | Investigations | 29 of 48 | 2 of 35 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 19 of 48 | 11 of 35 |
Serious events with the highest arm-level rate: lymphocyte count decreased (10.4%), atrial fibrillation (8.6%), heart failure (8.6%), lung infection (8.6%), platelet count decreased (5.7%).
The registry reports serious adverse event figures separately for Phase I/II, Cohort A and Phase II, Cohort B. Cohort A lists 48 at risk and 16 affected for serious adverse events; Cohort B lists 35 at risk and 15 affected. The corresponding other-adverse-event figures are 48 at risk and 48 affected in Cohort A, and 35 at risk and 35 affected in Cohort B. The death counts are 2 and 7, respectively. These are descriptive registry figures, not a randomized comparison.
The record lists lymphocyte count decreased at 10.4%, atrial fibrillation at 8.6%, heart failure at 8.6%, lung infection at 8.6%, platelet count decreased at 5.7%, and syncope at 5.7% among the top serious terms. The registry does not report the detailed event-level data or all definitions needed to independently interpret each percentage. These entries do not establish that treatment caused the events.
Serious adverse events should not be conflated with the separately listed outcome of grade 3 or greater adverse events at least possibly related to treatment. Seriousness concerns the event's classification, while grade refers to severity. The numerical results for that grade-based outcome are not reported in the registry record. The displayed counts also do not reveal event timing, repeated events in the same participant, follow-up duration, or whether all participants had the same observation period.
12. What the Result Does, and Does Not, Mean
The record identifies a Phase 1/2 single-group study of ixazomib, cyclophosphamide, and dexamethasone in previously untreated symptomatic multiple myeloma or light chain amyloidosis. Its primary endpoint is a dose-finding measure: the maximum tolerated dose of cyclophosphamide in the combination, defined through a dose-limiting toxicity rule assessed at 28 days. This identifies the study's main Phase I question, but the result itself is not reported in the registry record.
The registry also provides descriptive adverse-event counts for two cohorts and percentages for selected serious event terms. These figures can be read as reported safety observations within the named cohorts, with the listed denominators. They do not establish comparative safety, because the study is not a randomized comparison and the cohorts are not a like-for-like control group. Nor do the counts alone establish that the regimen caused an event.
The study's listed secondary outcomes include progression-free survival, survival time, and grade 3 or greater adverse events at least possibly related to treatment. Their numerical estimates are not reported in the registry record. It is therefore not possible from the record to characterize the magnitude or precision of survival outcomes, assess a treatment effect, or determine the frequency of the grade-based, possibly related adverse-event outcome.
In general, an observed outcome is not itself evidence of what would have occurred under a different treatment or without treatment. A controlled comparison, clearly defined analysis population, appropriate methods, and uncertainty estimates are needed to assess comparative effects. The single-group design supports descriptive interpretation but does not supply that counterfactual comparison. The registry record also lacks the dose-level observations and statistical analysis details needed to reproduce the dose-finding conclusion.
Thus, the available record supports a description of the trial's population, regimen, primary dose-finding question, and selected safety counts. It does not support a numerical conclusion about the maximum tolerated dose or a quantitative claim about treatment benefit. The posted data should be interpreted within those boundaries.
13. Why This Trial Matters Statistically
This trial raises a useful statistical distinction between dose finding and efficacy evaluation. Its primary endpoint is a maximum tolerated dose determined using a toxicity threshold, while progression-free survival and survival time appear among the secondary outcomes. These outcomes answer different questions. Dose finding concerns a dose-selection rule; time-to-event outcomes concern how long participants remain free of a defined event or survive. Treating the dose endpoint as evidence of efficacy would confuse the purpose of the measure.
The design also illustrates why the presence of multiple cohorts does not automatically create a comparative trial. The registry identifies a single-group model, allocation as NA, and no masking. It specifically indicates that this is not a randomized comparison. Cohort-level counts can describe what was recorded, but they cannot by themselves isolate treatment effects from differences in participants or follow-up.
Safety reporting likewise calls for careful reading. Serious adverse event counts, selected event-term percentages, and a listed grade 3 or greater possibly treatment-related outcome are not interchangeable. Separating seriousness, severity, and relationship to treatment is a general requirement for clear interpretation. The record lists each kind of information differently, and the result for the grade-based outcome is not reported.
Finally, the record demonstrates the importance of complete statistical reporting. A stated endpoint definition is not the same as a reported endpoint result. Without the selected dose, dose-level toxicity observations, analysis populations, and uncertainty estimates, readers cannot assess how stable the dose-finding conclusion is. Likewise, listed survival outcomes without estimates or methods cannot establish magnitude or precision. These distinctions are relevant when researchers use registry records to understand what a study asked and what its posted results can support.
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 principal limitation for statistical interpretation is that the registry record defines the primary dose-finding endpoint but does not report its result. Dose-level enrollment, observed dose-limiting toxicities, and an uncertainty assessment are not reported in the registry record. The stated Phase I population is 10 patients, but that description alone is insufficient to reconstruct the dose-finding process.
The study is single-group, with allocation listed as NA and masking as none. It has no randomized control group, so the cohort labels do not support a like-for-like comparison. The registry lists adverse-event counts by cohort, but does not report all details needed to compare observation time, participant characteristics, event recurrence, or completeness of follow-up. The cohorts should therefore be treated descriptively.
The registry lists progression-free survival, survival time, and grade 3 or greater adverse events at least possibly related to treatment, but their numerical results and analysis methods are not reported in the registry record. No statistical analysis records are listed. The record does not describe missing-data methods, time-to-event censoring rules, outcome-specific populations, multiplicity handling, or uncertainty estimates.
The treatment description includes an induction regimen and says treatment may continue for up to 12 courses, but the maintenance description is incomplete in the registry record. The listed safety figures cannot fill in those treatment details or establish exposure duration. Finally, registry outcome labels and adverse-event summaries may not provide the detail available in a full statistical report, but no such report is identified in the record details analyzed. These limitations restrict interpretation to the design, endpoint definition, and descriptive safety figures that are explicitly reported.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Ixazomib With Cyclophosphamide and Dexamethasone in Patients With Previously Untreated Symptomatic Multiple Myeloma or Light Chain Amyloidosis — NCT01864018.
- Registry record last updated 2025-07-24; status last verified 2025-07.
- Results first posted 2022-09-21.
- 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.
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.
18. Record Summary
The registry record describes a completed Phase 1/2, single-group study with enrollment of 87. It evaluated ixazomib citrate with cyclophosphamide and dexamethasone in previously untreated symptomatic multiple myeloma or light chain amyloidosis. The primary outcome is the Phase I maximum tolerated dose of cyclophosphamide with the combination, defined using a dose-limiting toxicity rule assessed at 28 days.
The record states that 10 patients accrued to the Phase I portion were analyzed for the dose assessment, but the selected dose is not reported in the registry record. Listed secondary outcomes include grade 3 or greater adverse events at least possibly related to treatment, progression-free survival, and survival time. Cohort-specific adverse-event counts and selected serious event terms are reported, but these do not constitute a randomized comparison. The registry reports nine outcome measures and no analysis records; analysis methods and results for the listed secondary outcomes are not reported in the registry record.