A Study to Evaluate the Safety, Tolerability, PK, PD, and Clinical Activity of EQ001 in Subjects With aGVHD: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study to Evaluate the Safety, Tolerability, PK, PD, and Clinical Activity of EQ001 in Subjects With aGVHD (NCT03763318), a randomized phase 1, phase 2 study. The primary outcome is Number of Treatment Emergent Adverse Events.

2. Clinical Question

3. Trial Design

The registry identifies NCT03763318 as a Phase 1b/2 study of EQ001 in subjects with newly diagnosed acute graft versus host disease. It classifies the study as randomized, quadruple masked, and sequential. The study is marked completed, and the registry enrollment figure is 30. The study start date is 2019-07-15, and the primary completion date is 2022-11-21.

The design description separates treatment into two parts. Part A was an open-label dose-escalation component. Its EQ001 groups received 0.4mg/kg, 0.8mg/kg, or 1.6mg/kg by intravenous infusion every two weeks for a total of 5 doses. Part B was blinded: participants received EQ001 at the optimal dose selected from Part A or EQ001 placebo, also by intravenous infusion every two weeks for a total of 5 doses. The registry does not report the method used to select that dose or the details of allocation within either part.

The primary outcome is recorded at Study Day 85 and is described as a count of participants with treatment-related adverse events assessed using CTCAE v5.0. The posted outcome values are for the Part A dose-escalation groups. The record also lists secondary outcomes covering EQ001 pharmacokinetics, including serum concentration and exposure measures, as well as inflammatory markers. Numeric results for those secondary outcomes are not reported in the registry record as.

The registry record does not report the sample-size justification, the detailed protocol, or the statistical analysis plan. Although the overall study is classified as randomized and quadruple masked, those labels do not mean that the Part A dose-escalation groups were masked: Part A is explicitly described as open label.

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: Part B of the study was not conducted.

Overall Study

5. Baseline Characteristics

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

6. Primary Endpoint

The registry lists the primary outcome as Number of Treatment Emergent Adverse Events, measured over Study Day 85.

The primary outcome is titled “Number of Treatment Emergent Adverse Events.” Its description specifies the number of participants with treatment-related adverse events assessed according to CTCAE v5.0, with a timeframe of Study Day 85. This is a participant count, rather than a measure of the number of separate adverse-event episodes or the duration of an event.

Safety outcomes are used to describe adverse events observed during a study and can help characterize the types of events reported during treatment. The participant-count approach indicates how many people in a group were recorded as experiencing the specified event, according to the endpoint definition. Interpreting such a count requires knowing the event definition, assessment period, population included, and how missing observations were handled. Those analysis details are not reported in the registry record.

There is a wording difference between the endpoint title, which says treatment-emergent, and the description, which says treatment-related. These terms do not necessarily mean the same thing: an event occurring after treatment begins is not automatically attributed to treatment. The registry record does not explain whether the posted counts use the title or the description as the operational definition. That ambiguity matters when reading the values of 4, 17, and 9 for the three Part A dose groups.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a participant count represent?

A binary endpoint classifies each participant according to whether the defined event occurred during the stated assessment period. Summing those classifications produces a count of participants with the event in each group. This is different from counting every episode: one participant with repeated events may still contribute only once if the endpoint is defined as the number of participants affected. The registry describes its primary outcome as a participant count, but does not report the detailed coding rules used to produce the posted values.

For this study, the recorded timeframe is Study Day 85. The description refers to treatment-related adverse events assessed using CTCAE v5.0. The title instead says treatment-emergent adverse events. In general, “treatment-emergent” describes an event occurring during or after treatment begins, whereas “treatment-related” implies an attribution judgment. The distinction can change which events are counted. The registry does not explain how this difference was resolved.

Why is a comparison different from separate group counts?

Counts within groups describe what was recorded in each group. A comparative analysis estimates how outcomes differ between groups, while accounting for the design and the uncertainty arising from the observed data. The registry record does not give a usable EQ001-versus-placebo comparison for this primary outcome; it marks the values as missing or not estimable for a two-arm comparison. The listed Part A counts alone cannot fill that gap. They refer to dose-escalation groups, not to a posted Part B EQ001-versus-placebo result.

A confidence interval, in general, describes the range of effect estimates compatible with the data and the analysis assumptions. Its width conveys precision: wider intervals indicate greater uncertainty. A p-value, in general, measures how compatible data are with a specified null model under the test assumptions; it is not a measure of clinical importance. Neither a confidence interval nor a p-value for a primary two-arm comparison is reported in the registry record, so neither can be interpreted for this outcome.

How should the count values be checked?

The posted values are 4, 17, and 9, and the listed group sizes are also 4, 17, and 9. A count of participants with an event that equals the listed group size could mean all members of each group were counted as affected, but the registry record does not clarify that interpretation. The matching figures could also reflect an issue in how the outcome was entered or described. No correction or explanation is reported in the registry record. It is therefore more appropriate to report the values as posted and flag their ambiguity than to infer an event pattern from them.

The terms used for outcomes also matter. Adverse events, treatment-emergent events, treatment-related events, and serious adverse events are not interchangeable categories. Seriousness is a classification separate from whether an event is considered related to treatment. The record reports a separate serious adverse-event summary, but does not provide enough detail to reconcile it with the primary endpoint count. A count in one category should not be substituted for a count in another.

What analysis details are absent?

The registry record does not report the statistical analysis plan, the analysis population for the primary outcome, the handling of missing observations, or the method used to estimate uncertainty. It also does not report whether any adjustment or model was used. These omissions prevent a reader from determining whether the posted values are raw observed counts, whether participants with incomplete follow-up were included, or how the study team intended to address differing exposure or follow-up.

In general, a statistical model must reflect the endpoint and design. A binary participant outcome is often analyzed using methods for event proportions or binary responses. If observations are clustered or outcomes are more variable than the model assumes, standard errors and confidence intervals can be unreliable; this is one reason to assess over-dispersion or other model assumptions when relevant. This is general statistical context, not a claim that such a model or diagnostic was used in this study. The registry record does not report a model, an over-dispersion assessment, or a diagnostic procedure.

Likewise, sample-size calculations depend on assumptions about the effect to detect, outcome variability, allocation, and the analysis method. The record describes planning illustrations based on a fraction of the observed effect and specifies a two-sided significance level of 0.05, equal allocation, and a normal approximation. However, no numerical planning results are reported in the registry information used for this interpretation, and the primary outcome does not have an estimable two-arm effect in the posted results. Such planning illustrations, when available, are not observed power and do not establish what the trial was designed to detect. The actual protocol rationale and sample-size justification are not reported in the registry record.

Finally, the registry lists 0 analyses for the posted outcomes. That does not itself explain the statistical process used by the investigators; it means that the registry does not supply a formal analysis record for the outcome. The distinction between descriptive counts and inferential analyses is essential: the former can be stated as recorded, while the latter require estimates, uncertainty measures, and a defined comparison that are not reported.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("EQ001 Dose Escalation (Part A) 0.4mg/kg", "EQ001 Dose Escalation (Part A) 0.8mg/kg", "EQ001 Dose Escalation (Part A) 1.6mg/kg")
n      <- c(4, 17, 9)
events <- c(4, 17, 9)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  EQ001 Dose Escalation (Part A) 0.4mg/kg  4  4  100.0  39.8  100.0  51.0  100.0  47.8  99.5
ARM  EQ001 Dose Escalation (Part A) 0.8mg/kg  17  17  100.0  80.5  100.0  81.6  100.0  81.5  99.9
ARM  EQ001 Dose Escalation (Part A) 1.6mg/kg  9  9  100.0  66.4  100.0  70.1  100.0  69.2  99.7

11. Multiplicity, Missing Data and Analysis Populations

The study lists several secondary outcomes, including EQ001 serum concentration measures, exposure, half-life, volume of distribution, clearance, and inflammatory markers. When a study examines multiple outcomes or multiple comparisons, the chance of observing an apparently notable result can increase unless the analysis plan addresses multiplicity. In general, multiplicity methods define how confirmatory claims are controlled across a set of tests. The registry record does not report whether such a method was planned or used, or how the primary and secondary outcomes were prioritized in analysis.

Missing-data handling is also not reported in the registry record. The posted values include a group size and an outcome count for each Part A dose group, but the record does not specify the analysis population, follow-up completeness, or whether any participants were excluded from the endpoint analysis. Counts alone do not reveal how missing assessments were handled. There is no reported sensitivity analysis to show whether conclusions would change under alternative assumptions about missing observations.

The registry record does not specify whether the primary analysis was intention-to-treat, per-protocol, or based on another population. It also does not report rules for treatment discontinuation, protocol deviations, or the timing of adverse-event attribution. These details are relevant because analysis populations and event definitions can affect the count attributed to each group.

Although a sample-size planning approach is described, the record does not report the original sample-size justification or numerical planning results for the primary endpoint. It also does not report an interim-analysis plan or whether any interim analyses were conducted. Those items should not be inferred from the sequential study label or from the posted counts.

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: adenovirus infection (88.9%), sepsis (52.9%), skin infection (25.0%), graft versus host disease in gastrointestinal tract (25.0%), pyrexia (25.0%).

The separate serious adverse-event summary lists the number at risk and the number affected in each Part A dose group. The record gives 2 of 4 for the 0.4mg/kg group, 9 of 17 for the 0.8mg/kg group, and 8 of 9 for the 1.6mg/kg group. It also lists deaths of 1, 7, and 6, respectively. These are descriptive counts in the registry record; they are not comparative estimates and do not establish that EQ001 caused the events or deaths.

For other adverse events, the record lists 4 of 4, 17 of 17, and 9 of 9 affected. These counts are separate from the serious-event counts and from the primary endpoint as worded. Seriousness, treatment emergence, and attribution to treatment describe different aspects of an event. The registry does not provide enough detail to reconcile these categories or to determine whether the primary outcome values refer to the same event set.

The registry lists top serious-event terms with percentages: Adenovirus infection, 88.9%; Sepsis, 52.9%; Skin infection, 25.0%; Graft versus host disease in gastrointestinal tract, 25.0%; Pyrexia, 25.0%; and Graft versus host disease, 17.6%. The denominator and the group allocation for these percentages are not reported in the registry record, so they should not be treated as group-specific risks.

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

The registry results show posted participant counts in the three EQ001 Part A dose-escalation groups and a separate set of serious adverse-event counts. These observations describe what the registry records for those groups. They do not provide an estimable comparison of EQ001 with placebo for the primary outcome. The record explicitly marks the values as missing or not estimable for a two-arm comparison, and it does not report an effect estimate, confidence interval, or p-value for that contrast.

The values for the primary endpoint equal the listed group sizes: 4 in the 0.4mg/kg group, 17 in the 0.8mg/kg group, and 9 in the 1.6mg/kg group. Since the endpoint description says the values count participants with treatment-related adverse events, this match raises an interpretation question. The registry does not say whether these figures mean that every participant experienced the endpoint or whether the values reflect a recording or definition issue. The difference between “treatment-emergent” in the title and “treatment-related” in the description adds uncertainty about the event definition.

These findings do not establish whether EQ001 is safer or less safe than placebo, nor do they establish a causal relationship between treatment and any listed adverse event. The Part A dose groups were open label, and the posted primary outcome results do not include a placebo comparison. The serious-event counts and the list of serious-event terms are descriptive; their denominators and analysis details are incomplete.

The study also assessed pharmacokinetic, pharmacodynamic, and clinical activity outcomes, but the numeric results for those outcomes are not reported in the registry record as. The overall study status and enrollment figure do not replace outcome-level analysis. A completed study can still have incomplete or non-comparative results in its registry entry. The most defensible reading is therefore limited to the recorded group counts and the limitations of their definition and comparison.

14. Why This Trial Matters Statistically

This trial illustrates why a registry outcome needs to be read together with its definition, group structure, and analysis status. The primary endpoint title refers to treatment-emergent adverse events, while the description specifies treatment-related events. Those labels imply different event-classification rules, and the registry does not resolve which rule generated the posted counts.

It also demonstrates the difference between a study-level design label and the design of a specific treatment part. The registry classifies the study as randomized and quadruple masked, yet describes Part A as open label and Part B as blinded. The primary outcome values listed are for Part A, whereas the placebo group appears in the Part B description. A randomized label by itself cannot turn the Part A counts into a placebo-controlled estimate.

For statistical interpretation, counts alone are not enough to establish comparative effects. Readers also need a defined comparison, an analysis population, uncertainty estimates, and clear handling of missing data. Those details are not reported for this primary outcome, and the registry marks the values as missing or not estimable for a two-arm comparison. This prevents a valid inferential reading of the posted counts as an EQ001-versus-placebo result.

The serious adverse-event summaries show another important distinction: event counts, seriousness, and treatment attribution are different concepts. Keeping those categories separate avoids overstating what descriptive registry entries can establish. The record is useful for identifying what was registered and what values were posted, while its omissions and ambiguous endpoint wording limit the conclusions that can be drawn.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The primary outcome values are difficult to interpret because the count in each dose group matches its listed group size. The registry record does not explain whether every participant was counted as having the endpoint, whether another coding convention was used, or whether the values were entered incorrectly. The endpoint title and description also differ in their use of treatment-emergent and treatment-related. No clarification is reported in the registry record.

The posted primary outcome entries are for the open-label Part A dose groups, while the placebo comparator is described in Part B. The registry does not provide a usable two-arm comparison for this outcome, and no comparative effect estimate or uncertainty measure is reported. The study-level randomized and masking labels do not resolve this limitation for the posted Part A results.

The record does not report the analysis population, missing-data approach, statistical model, confidence intervals, p-values, or a detailed analysis plan. It does not give enough information to assess whether follow-up or exposure differed across groups, or how such differences were handled. The serious adverse-event percentages are listed without denominators or group assignment, limiting their interpretation as group-specific risks.

The registry gives a total enrollment figure of 30, but the relationship between that figure, the outcome analysis population, and the posted group counts is not described. Numeric results for the listed secondary outcomes are not reported in the registry record. The sample-size justification, interim-analysis details, and protocol-level rationale are also not reported. These limitations restrict both precision assessment and the strength of any comparative conclusions.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study to Evaluate the Safety, Tolerability, PK, PD, and Clinical Activity of EQ001 in Subjects With aGVHD — NCT03763318.
• Registry record last updated 2026-06-11; status last verified 2026-05.
• Results first posted 2025-04-18.
• 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

NCT03763318 is a completed Phase 1b/2 study of EQ001 in newly diagnosed acute graft versus host disease. The registry lists a randomized, quadruple-masked, sequential design, with open-label dose escalation in Part A and blinded EQ001 and placebo groups in Part B. The enrollment figure is 30.

The primary outcome is a participant count assessed through Study Day 85, but its title and description differ on whether events are treatment-emergent or treatment-related. The posted values for the Part A dose groups are 4, 17, and 9, matching the listed group sizes. The registry marks the values as missing or not estimable for a two-arm comparison. Serious adverse-event counts are also posted, but they do not establish treatment causation or replace a comparative analysis. Key analysis details, including the analysis population, missing-data handling, and statistical plan, are not reported in the registry record.