Evaluation of Efficacy and Safety of Roxadustat for the Treatment of Chemotherapy Induced Anemia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Evaluation of Efficacy and Safety of Roxadustat for the Treatment of Chemotherapy Induced Anemia (NCT04076943), a na phase 2 study. The primary outcome is Maximum Change in Hb Within 16 Weeks From Baseline Without Red Blood Cell (RBC) Transfusion.

2. Clinical Question

The study asked whether roxadustat could be used to investigate anemia in participants receiving chemotherapy for non-myeloid malignancies. Its primary question concerned the mean maximum change in hemoglobin (Hb) from baseline during up to 16 weeks, excluding Hb values within 4 weeks after a red blood cell (RBC) transfusion. The study also recorded secondary measures of Hb change and response, along with transfusion and safety outcomes.

3. Trial Design

This was a phase 2, open-label, single-group study sponsored by Kyntra Bio. The registry lists an enrollment of 92 and a study status of completed. The study began on 2019-08-20, and primary completion was recorded as 2021-03-26. Participants received roxadustat as an oral tablet three times per week, for up to a maximum of 16 weeks.

Allocation is listed as not applicable, and masking is listed as none. These features describe a study without randomized assignment or blinded treatment. The design therefore focuses on observations among participants who received roxadustat; it does not include a concurrent control group against which to compare changes in Hb or safety outcomes. Changes over time in a single group can be described, but they cannot by themselves distinguish a treatment-associated change from other influences or the course of illness.

The registry describes the population as participants with anemia receiving chemotherapy treatment for non-myeloid malignancies. The primary analysis population was more specifically defined: participants had to be enrolled, receive at least one dose, have a baseline Hb value, and have at least one central-laboratory Hb assessment during the treatment period. The number meeting all of these criteria is not reported in the registry record.

The primary endpoint concerned maximum Hb change from baseline within 16 weeks, without RBC transfusion. The registry also lists secondary outcomes involving Hb change at specified visits, increases from baseline, hematopoietic response, time to an increase, and RBC transfusion. Although the study is marked completed, completion status alone does not establish that every planned outcome was reported. The numerical result for the primary endpoint is 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: Full analysis set included all enrolled participants who received at least 1 dose of roxadustat, had a baseline Hb and at least 1 central lab Hb assessment during the treatment period.

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: Intent-to-treat (ITT) population included all participants enrolled in the roxadustat treatment period and received at least 1 dose of study medication.

6. Primary Endpoint

The registry lists the primary outcome as Maximum Change in Hb Within 16 Weeks From Baseline Without Red Blood Cell (RBC) Transfusion, measured over Baseline, up to Week 16.

The primary endpoint was the maximum change in Hb from baseline within 16 weeks, without RBC transfusion. The registry identifies Hb as a continuous measure in g/dL and says the endpoint was summarized as a mean with a standard deviation. In practical terms, the endpoint is intended to capture the largest observed Hb change during the stated assessment period for each participant, then describe those changes across the analysis population.

Baseline Hb was defined as the mean of central-laboratory assessments before the first dose. The definition allowed up to 2 latest screening values before Day 1 and a value on Day 1. Central-laboratory assessments from Day 1 through end of treatment or early termination were included. Hb values within 4 weeks after an RBC transfusion were excluded from this endpoint.

As a general statistical matter, a mean summarizes the average observed change, while a standard deviation describes how spread out individual changes are around that mean. A maximum-within-period measure can capture a participant’s best observed value during follow-up, but it does not describe the timing or durability of that value on its own. The registry record does not report the primary endpoint’s numerical mean or standard deviation, so the size and variability of the observed change cannot be assessed from the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a continuous primary endpoint summarized by a mean and standard deviation. It does not report the numerical estimate or a detailed statistical analysis plan. The concepts below explain how these measures are generally interpreted; they do not imply that any unreported analysis was performed.

What does the mean describe?

As a general statistical concept, a mean is the arithmetic average of the values included in an analysis. For this endpoint, the intended measure is the average of participants’ maximum Hb changes from baseline within the assessment window, after applying the transfusion-related exclusion rule. A mean gives a compact description of the group’s central tendency, but it does not show whether individual participants had similar or widely varying changes. It can also be influenced by unusually high or low values.

The word maximum matters. This endpoint is not simply the change at a single common visit: it refers to the maximum change observed within the stated period. Participants may reach their maximum at different times. As a general matter, a maximum can be useful for describing the best observed value during follow-up, but it does not by itself show when that value occurred, how long it persisted, or what Hb values were at other visits. The registry record does not report participant-level trajectories or the numerical result of this endpoint.

What does the standard deviation describe?

In general, a standard deviation describes the spread of observations around their mean. A smaller standard deviation indicates that observations are more clustered around the mean; a larger one indicates more dispersion. It is not the same as a confidence interval. A standard deviation describes variation among observations, whereas a confidence interval is a range intended to express uncertainty about an estimated quantity under a specified statistical method. No standard deviation or confidence interval for the primary result is reported in the registry record.

For clinical data, observations may vary for many reasons, including differences among participants and measurement variability. Describing the mean without a measure of spread can therefore give an incomplete account of the group. Conversely, a standard deviation alone does not establish how precisely the mean has been estimated. Assessing precision usually also requires information such as the analysis population size and a confidence interval, which are not reported for the primary endpoint in the registry record.

How was baseline and follow-up defined?

The registry defines baseline Hb using central-laboratory assessments before the first study-treatment dose: up to 2 latest screening values before Day 1 and a value on Day 1. Assessments from Day 1 through end of treatment or early termination were eligible for the endpoint. Hb values within 4 weeks after an RBC transfusion were excluded. These rules identify which measurements could contribute; they do not describe how missing measurements were handled or how a participant’s maximum was selected when measurements were unavailable.

As a general principle, a baseline definition can affect the calculated change because every change is measured relative to that starting value. Using more than one pre-treatment assessment can characterize the starting level differently from using a single measurement. The registry record specifies the allowed baseline assessments, but does not report the actual baseline values or the number of participants with each pattern of measurements.

Why do the analysis population criteria matter?

The primary population included enrolled participants who received at least one dose, had a baseline Hb value, and had at least one central-laboratory Hb assessment during treatment. This definition sets eligibility for the primary analysis. It does not tell readers how many participants met all criteria; that number is not reported in the registry record. The enrollment figure and the analysis-population count should not be assumed to be identical, because the population definition contains additional requirements.

In general, analysis-population rules can affect which observations contribute to an estimate. Excluding participants without a follow-up assessment may change the composition of the analyzed group if missingness is related to health status or outcomes. The registry record does not report the reasons for missing measurements, the frequency of missing data, or any imputation or sensitivity analyses. It is therefore not possible to assess from the record how missing Hb assessments may have influenced the primary result.

What can a single-group analysis establish?

In general, a single-group study can describe outcomes observed among participants receiving the study intervention. It cannot provide the same direct treatment-versus-control comparison as a randomized study. Changes from baseline may be affected by time, underlying illness, chemotherapy, other care, or measurement patterns. Without a concurrent control group, those influences cannot be separated from the treatment-associated pattern using a randomized contrast.

The registry record lists no masking and no randomized allocation. It does not report a model for the primary endpoint, an adjustment strategy, confidence intervals, p-values, or a prespecified statistical testing procedure. Those details are not reported in the registry record and should not be inferred from the endpoint’s mean-and-standard-deviation description.

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("Roxadustat")
n   <- c(89)
m   <- c(2.47)
sp  <- c(1.51)
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  Roxadustat  89  2.470  1.510  2.152  2.788

11. Multiplicity, Missing Data and Analysis Populations

The registry lists the primary Hb endpoint and secondary outcomes covering Hb changes at Weeks 9, 13, and 16, increases from baseline, hematopoietic response, time to an increase, and RBC transfusion. It records 9 outcome measures overall. These outcomes involve multiple measures and time points, so interpretation of any collection of statistical tests would depend on how the analyses were planned and whether multiplicity was addressed.

As a general statistical concept, testing several outcomes or time points can increase the chance of obtaining at least one apparently notable result by chance. Methods for addressing this can include prespecifying a hierarchy of outcomes, adjusting significance thresholds, or treating secondary results as descriptive. The registry record does not report a multiplicity strategy, a testing hierarchy, or which outcomes, if any, were intended to support confirmatory claims. No outcome-specific statistical analysis results are reported in the record.

The primary population definition requires enrollment, at least one dose, a baseline Hb value, and at least one central-laboratory Hb assessment during treatment. The number of participants in that population is not reported in the registry record. The record also does not report the amount or pattern of missing outcome data, reasons for missingness, imputation methods, or sensitivity analyses. These omissions limit assessment of whether participants with observed outcomes differed from those without them.

As a general matter, missing-data assumptions can influence an estimate when observations are unavailable, especially if absence of measurement is related to the outcome. The transfusion exclusion is a separate endpoint rule, not a full missing-data strategy. The registry record does not describe how other unavailable Hb assessments or early discontinuations were handled in the analysis. Without these details and the numerical endpoint results, the robustness of the primary estimate cannot be evaluated.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: pancreatic carcinoma metastatic (4.3%), pulmonary embolism (4.3%), febrile neutropenia (3.3%), asthenia (3.3%), disease progression (3.3%).

The registry reports safety figures for the roxadustat group: 40 participants affected by serious adverse events among 92 at risk, 71 affected by other adverse events among 92 at risk, and 17 deaths. These are descriptive figures from the study record. They do not, on their own, show that roxadustat caused an event or death, and the single-group design provides no randomized control comparison for estimating an excess risk.

The registry lists pancreatic carcinoma metastatic and pulmonary embolism among the leading serious-event terms, each reported as 4.3%. Febrile neutropenia, asthenia, disease progression, and sepsis are each reported as 3.3%. These percentages are reported as listed; the registry record does not provide further detail about event-level counting, timing, attribution, or whether a participant could contribute to more than one term.

Serious adverse events, other adverse events, and deaths are distinct safety summaries and may overlap. They should not be added together to create a total, and the registry record does not state that deaths were caused by treatment. The available safety figures describe what was recorded in this group, not a comparative measure of treatment-related harm.

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

The study addresses a relevant statistical question: how Hb values changed during treatment with roxadustat among participants receiving chemotherapy for non-myeloid malignancies. Its primary endpoint was defined in detail, including the baseline period, follow-up window, and exclusion of Hb values within 4 weeks after an RBC transfusion. These definitions help clarify what the intended measure represents.

However, the registry record does not report the numerical primary-endpoint result. Without a mean, measure of variability, or confidence interval, readers cannot determine the observed size of the maximum Hb change or how precisely it was estimated. The absence of a reported result is not evidence that the change was zero or that the endpoint was not measured; it means the result is not reported in the registry record.

The design also determines what conclusions the observations can support. Because this was an open-label, single-group study without a randomized comparator, a within-group change cannot be interpreted as a controlled treatment effect. Other influences on Hb during chemotherapy and follow-up cannot be separated from the intervention using a randomized contrast in this study.

The safety record lists adverse-event and death counts, as well as selected serious-event terms and percentages. Those figures are useful descriptive information about events recorded in the roxadustat group. They do not establish causation, and without a comparison group they cannot quantify how event frequency differs from what would have occurred under another treatment or care approach.

Overall, the registry record documents the study’s question, design, endpoint definitions, analysis-population criteria, and descriptive safety figures. It does not provide the primary outcome estimate or a statistical analysis plan sufficient to assess precision, missing-data handling, or multiplicity. The record therefore supports a description of the intended evaluation and reported safety observations, but not a conclusion about a comparative treatment effect.

14. Why This Trial Matters Statistically

This trial matters statistically because it illustrates the difference between a clearly defined endpoint and a reported endpoint result. The primary measure specifies how baseline Hb was constructed, which follow-up assessments were eligible, and how measurements after RBC transfusion were treated. Such details are essential for understanding what an estimate would represent. But without the numerical estimate and its variability, readers cannot assess the magnitude or precision of the observed outcome.

The study also demonstrates why design and interpretation must be considered together. A single-group study can describe the pattern of outcomes among participants who received roxadustat. It cannot supply the randomized contrast needed to distinguish treatment-associated change from other changes over time. This is particularly important when the endpoint is measured repeatedly and the participants are receiving chemotherapy, because several influences may operate during the observation period.

The analysis-population definition provides important context: participants had to receive at least one dose and have both baseline and on-treatment central-laboratory Hb assessments. Yet the number meeting those criteria is not reported in the registry record, and missing-data methods are not described. These omissions matter because the set of observed participants and measurements determines the estimate being summarized.

Finally, the record contains safety counts and selected serious-event terms, but these remain descriptive without a comparison group or causal attribution. Together, these features make the study useful for understanding how endpoint definitions, population rules, reporting completeness, and design constrain statistical interpretation. The record’s detailed endpoint specification is informative, while its missing primary result and analysis details limit evaluation of the study’s main statistical question.

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 main limitation for interpreting efficacy is that the registry record does not report a numerical result for the primary endpoint. It also does not report the primary estimate’s standard deviation or confidence interval. Consequently, neither the observed magnitude nor its precision can be evaluated from the record.

The single-group, open-label design has no randomized comparator. Any observed change from baseline would therefore be descriptive and could not isolate the effect of roxadustat from other influences over time. The registry record does not report a statistical model, adjustment plan, or formal testing procedure for the primary outcome.

The primary analysis population is defined by treatment exposure and availability of baseline and follow-up central-laboratory Hb assessments, but the number meeting those criteria is not reported. The record does not describe the extent or reasons for missing assessments, how early termination affected the endpoint, or whether imputation or sensitivity analyses were used. The transfusion-related exclusion is specified, but other missing-data handling is not reported.

The registry lists multiple secondary outcomes, but does not report a multiplicity strategy or an outcome hierarchy. It also does not report numerical results for those outcomes in the information summarized by the record. Safety counts are descriptive, without a control group or reported causal attribution. These limitations are specific to what the registry record documents; additional study reports or analyses are not described in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Evaluation of Efficacy and Safety of Roxadustat for the Treatment of Chemotherapy Induced Anemia — NCT04076943.
• Registry record last updated 2022-06-03; status last verified 2022-06.
• Results first posted 2022-06-03.
• 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

NCT04076943 is a completed phase 2, open-label, single-group study sponsored by Kyntra Bio. It evaluated roxadustat for anemia in participants receiving chemotherapy for non-myeloid malignancies. The registry lists an enrollment of 92, a start date of 2019-08-20, and primary completion on 2021-03-26. Participants received oral roxadustat three times per week for up to a maximum of 16 weeks.

The primary endpoint was maximum Hb change from baseline within 16 weeks, without RBC transfusion, summarized as a mean and standard deviation. The registry record defines the endpoint and analysis population but does not report the numerical primary result or its precision. Safety figures include 40 participants affected by serious adverse events, 71 affected by other adverse events, and 17 deaths among the 92 at risk. The study has no randomized comparator, and the record does not establish that reported events were caused by roxadustat.