Efficacy and Safety Study of I10E in Treatment of Patients With CIDP: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy and Safety Study of I10E in Treatment of Patients With CIDP (NCT02293460), a na phase 3 study. The primary outcome is Efficacy Endpoint: Responder Rate at End of Study.

2. Clinical Question

The study’s stated primary objective was to assess whether I10E improved disability in patients with CIDP. Its secondary objective was to assess safety. The primary endpoint therefore focuses on a change in disability score, while the safety summary counts participants with adverse events. These are different kinds of evidence: the endpoint definition specifies how a response would be classified, but the posted record does not give the number of participants who met that definition.

Because this is a single-group study, the posted results cannot provide a randomized comparison of I10E with a control treatment. The record also does not report an estimated difference against a comparator. The clinical question represented by the study is thus whether a defined disability response was observed among participants treated with I10E, alongside a description of adverse events—not whether I10E performed better than another treatment.

3. Trial Design

This was a Phase 3, completed, international, multicentre study of I10E in patients with chronic inflammatory demyelinating polyradiculoneuropathy. The study title describes both initial and maintenance treatment. The registry record lists a single-group model, allocation as NA, and masking as none. It lists 44 participants enrolled and identifies I10E as the intervention.

The registry lists the study start as 2015-05 and primary completion as 2017-09-29. These dates describe the record’s study timeline; they do not establish when individual participants started or finished treatment. The primary endpoint timeframe is 24 weeks after first treatment injection. The registry does not report further details about treatment schedules, dose, duration beyond that endpoint timeframe, or participant follow-up.

The single-group structure matters for interpretation. There is no control arm in the posted design against which to compare the responder outcome or adverse events. Changes from baseline within a treated group can describe what was observed, but by themselves they do not separate a treatment effect from other possible explanations for change over time. This is a general limitation of uncontrolled studies, not a finding that any particular alternative explanation occurred.

The record identifies a primary efficacy objective and a secondary safety objective. It lists one outcome measure and one analysis, but does not report the statistical model, test, confidence interval method, or other analysis details. The registry record also does not report the planned sample-size justification, any interim analyses, or further protocol details. These gaps limit how fully the analysis and design can be evaluated from the posted information.

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: 59 subjects signed an informed consent in the pre-assigment period. Among them, 18 subjects were considered as a screening failure including 3 subjects re-screened and finally enrolled. Of 44 enrolled subjects, on subject was discontinued (consent withdrawn by subject) from the study before the first administration of study drug.

Overall Study

Reasons for not completing this 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.

6. Primary Endpoint

The registry lists the primary outcome as Efficacy Endpoint: Responder Rate at End of Study, measured over 24 weeks after first treament injection.

The primary endpoint is titled “Efficacy Endpoint: Responder Rate at End of Study.” Responders are defined as participants with a decrease of at least 1 point in adjusted INCAT disability score compared with baseline. The adjusted score can range from 0, described as normal, to 9, described as maximal disability. The endpoint timeframe is 24 weeks after first treatment injection.

This endpoint converts a score change into a binary classification: each participant is counted as either meeting the responder definition or not meeting it. A responder rate can be useful because it describes how many participants cross a prespecified threshold, rather than only describing an average score change. It also makes the endpoint’s meaning depend on the chosen threshold and the rules for handling missing or unusable observations. These are general features of binary responder outcomes.

The registry record specifies two handling rules. Scores measured after a participant received a not-allowed treatment were censored. If the score at the end-of-study visit was missing, the Last Observation Carried Forward (LOCF) approach was applied. The record does not report how many participants were affected by either rule, nor the responder count or rate. Consequently, the definition is available, but the posted results do not show how many participants met it or how precisely the responder rate was estimated.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The statistical information available in the registry record is limited. It gives the study structure, the responder definition, rules for handling certain observations, and summary safety counts. It does not report the statistical test or model used for the efficacy endpoint, the confidence interval method, or a detailed analysis plan. The explanations below distinguish those posted details from general statistical concepts.

What does a single-group design allow?

The study is listed as single-group, with allocation NA and masking none. There is no comparator group described in the record. In general, a single-group study can summarize outcomes among participants who received the intervention. A change from baseline can be described, but it does not by itself establish what would have happened to the same participants over the same period in the absence of treatment. Without a control group, differences attributable to treatment cannot be isolated from other influences using a randomized between-group comparison.

Masking is also listed as none. In general, masking can reduce the influence of expectations on aspects of study conduct or outcome assessment, depending on what is masked. The record does not describe further procedures for outcome assessment, so the implications of the masking status cannot be elaborated for this study.

How does a binary responder endpoint work?

The endpoint labels a participant a responder if adjusted INCAT disability score decreases by at least 1 point compared with baseline. This transforms a score into a yes-or-no outcome for each participant. A rate then summarizes the proportion meeting that rule in the analyzed group. In general, this can make a result easier to interpret against a specified threshold, but it also discards information about the size of score changes beyond the classification boundary. The registry record does not provide the responder count or rate, so those summaries cannot be evaluated.

The score’s stated range is 0 to 9, with 0 described as normal and 9 as maximal disability. That range gives context for the endpoint but does not reveal the participants’ baseline scores or their changes. Those details are not reported in the registry record.

What do censoring and LOCF mean?

The record says that adjusted INCAT disability scores measured after a participant received a not-allowed treatment were censored. In general, censoring means that some observations are not used as if they were valid measurements for the analysis after a defined point or event. The record gives the rule but does not report how many participants were affected, which treatments qualified, or the detailed analysis consequences of censoring those scores.

For a missing end-of-study visit score, the record says LOCF was applied: the last observed score was carried forward to replace the missing value. This is an imputation rule, meaning a missing value is filled in using an observed value. As a general statistical matter, the resulting estimate depends on the assumption implicit in carrying the earlier observation forward. The registry record does not report the number of missing end-of-study scores, the timing of last observations, or any sensitivity analyses assessing this choice.

How should uncertainty and significance be understood?

General explanation: a confidence interval describes uncertainty around an estimate under a specified statistical method and its assumptions. A narrower interval indicates greater precision than a wider interval, but interpretation also depends on the outcome and analysis. A p-value, when reported, summarizes how compatible data are with a specified null hypothesis under the analysis assumptions; it is not a measure of the size or clinical importance of an effect. Neither a confidence interval nor a p-value for the primary endpoint is reported in the registry record.

The study summary lists one analysis, but does not identify its method or result. It would therefore be inappropriate to assume a particular statistical test, adjustment, or model. The analysis population used for the efficacy endpoint is also not reported in the registry record. The Total Treated Set appears in the safety section, but that label alone does not establish which participants were included in the efficacy analysis.

What do planning calculations represent?

General explanation: sample-size planning asks how many participants might be needed under stated assumptions to detect a chosen effect with a chosen probability, at a chosen significance level. Such calculations are planning illustrations, not observed evidence and not “observed power.” The sample-size assumptions described for this page use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with effects specified as fractions of the observed effect. Those are not descriptions of this single-group study’s reported design. The registry record does not report its own sample-size justification, and no planning illustration can replace a missing efficacy result or establish the study’s actual precision.

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("Full Analysis Set")
n      <- c(42)
events <- c(32)
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  Full Analysis Set  42  32  76.2  60.5  87.9  61.5  86.5  61.4  86.5

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one outcome measure and one analysis, but does not give the analysis details or identify statistical tests. It also states a primary efficacy objective and a secondary safety objective. The record does not report a multiplicity procedure or explain how any multiple comparisons or outcomes were handled. No adjustment or lack of adjustment should be inferred from the count of listed outcomes alone.

The endpoint rules address two situations: scores after receipt of a not-allowed treatment were censored, and a missing end-of-study score was replaced using LOCF. The registry does not report the number of participants with censored scores or missing endpoint scores, and it does not describe sensitivity analyses. General explanation: missing-data handling can affect an estimate when the missingness or replacement rule is related to participant outcomes. Sensitivity analyses can examine whether conclusions change under alternative assumptions, but their use is not reported in the registry record.

The registry does not identify the efficacy analysis population or explain how participants who discontinued, had protocol deviations, or lacked usable measurements were handled, beyond the stated rules. Enrollment is 44 and the safety set contains 43; why these counts differ is not reported in the registry record. The safety set should not be assumed to be the efficacy analysis population.

These omissions matter because a result is easier to assess when the analysis population, missing-data approach, and any multiplicity handling are stated alongside the endpoint estimate and its uncertainty. Here, the endpoint definition is reported, but the responder result and those supporting analysis details are not reported in the registry record.

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: femur fracture (2.3%), synovial rupture (2.3%), fibrin d dimer increased (2.3%), colorectal adenocarcinoma (2.3%), neutropenia (2.3%).

The safety summary is for the Total Treated Set, with 43 participants at risk. It reports 7 participants affected by serious adverse events and 39 affected by other adverse events. It also reports 0 deaths. These are participant counts in the posted safety summary; they do not show whether events were judged related to I10E, how long participants were observed, or how event severity was assessed beyond the serious-event classification.

The listed serious adverse-event terms are femur fracture, synovial rupture, fibrin D dimer increased, colorectal adenocarcinoma, neutropenia, and anaphylactic reaction. Each term is reported as 2.3%. The registry record does not give further term-level counts or details about timing, recurrence, or individual clinical circumstances. The list should therefore be read as a summary of reported serious-event terms, not as a full clinical account.

General explanation: adverse-event counts describe what was recorded in a study population. Without a comparator group, they cannot establish whether the frequency is higher or lower than it would have been under another treatment or no treatment. The record does not report a comparative safety analysis.

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

The record establishes that a Phase 3, single-group study of I10E was completed, and it describes a primary disability endpoint based on a decrease of at least 1 point in adjusted INCAT disability score. It also reports selected safety counts for the Total Treated Set. These registry entries allow readers to understand the endpoint definition and some of the observed safety reporting.

However, the primary responder count and rate are not reported in the registry record. There is no posted efficacy estimate, confidence interval, or p-value to interpret. The definition of a responder is not itself evidence that participants did or did not meet the definition. The record therefore does not support a conclusion about the size or precision of the primary efficacy result.

The single-group design also limits what can be concluded causally. A result among treated participants, even if reported, would not on its own show how outcomes compare with a control treatment. The registry record does not describe a control group or randomized allocation. It likewise does not report a comparative safety assessment. These limitations concern the information available for interpretation; they are not claims that the intervention had a particular effect.

General explanation: statistical significance and clinical importance are distinct. A statistical test, when reported, addresses compatibility with a null hypothesis under specified assumptions. Clinical importance concerns whether the magnitude and meaning of an outcome matter in its context. Neither can be judged for this primary endpoint from the posted registry information because the result and its uncertainty are absent.

The safety summary reports serious events, other adverse events, and zero deaths, but it does not supply the detail or comparison needed to attribute events to treatment or quantify comparative risk. The most defensible reading is therefore descriptive: the registry records the study’s design and endpoint rules and provides limited safety counts, while leaving the central efficacy result and important analysis details unreported.

14. Why This Trial Matters Statistically

This trial is statistically instructive because it pairs a clearly stated responder definition with a registry record that does not report the resulting responder count or rate. It shows why an endpoint’s operational definition and its observed result are separate pieces of information. The definition tells readers what would count as a response; the result is needed to establish how often that response was observed.

The single-group design also illustrates the difference between describing outcomes and estimating a comparative treatment effect. A treated group can be summarized, but no randomized control comparison is available in the posted design. Statistical methods cannot recover a randomized contrast that the design does not contain. This is a general point about causal interpretation, rather than a claim about what the unreported study results would show.

The record’s LOCF rule and censoring rule are useful reminders that endpoint values depend not only on the measurement scale but also on how post-treatment events and missing observations are handled. Reporting the rules makes the intended approach more transparent, while counts affected and sensitivity analyses would help readers assess its influence. Those details are not reported in the registry record.

Finally, the safety information is presented as participant counts and event terms without a comparator. This underscores why descriptive safety figures should not be mistaken for comparative risks. The trial matters statistically as an example of the questions a registry record can answer—design, endpoint definition, and selected safety summaries—and the questions it leaves open when outcome estimates, uncertainty, and analysis details are unavailable.

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 limitation for interpreting this study from the registry record is that its primary efficacy result is not reported. The responder count and rate, uncertainty interval, and statistical significance result are unavailable. Without these, readers cannot assess the observed endpoint or its precision.

The design is single-group, with allocation listed as NA and masking as none. No comparator is described, so the registry record does not support a randomized comparative estimate. The record does not report further details about recruitment, eligibility criteria, treatment dose or schedule, duration of treatment, or follow-up beyond the stated endpoint timeframe. These details should not be inferred from the study title.

The endpoint uses a responder threshold and specified handling rules: scores after not-allowed treatment were censored, and LOCF was used for a missing end-of-study score. The number of affected participants, the identity of not-allowed treatments, and sensitivity analyses are not reported in the registry record. The analysis population, statistical model or test, confidence interval method, multiplicity handling, and sample-size justification are also not reported.

The safety results use a Total Treated Set of 43, compared with 44 enrolled; the reason for this difference is not reported in the registry record. The adverse-event summary does not provide comparative data or further event-level context. These limitations restrict causal interpretation and assessment of precision, but they do not establish what the unreported efficacy findings would have been.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Efficacy and Safety Study of I10E in Treatment of Patients With CIDP — NCT02293460.
• Registry record last updated 2021-01-27; status last verified 2021-01.
• Results first posted 2020-03-09.
• 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

This completed Phase 3 study evaluated I10E in patients with chronic inflammatory demyelinating polyradiculoneuropathy. It is described as an international, multicentre, single-group study, with allocation NA and masking none. The registry lists 44 participants enrolled and a primary endpoint timeframe of 24 weeks after first treatment injection.

The primary endpoint defines responders by a decrease of at least 1 point in adjusted INCAT disability score compared with baseline. The registry record does not report the responder count or rate, or the statistical analysis details needed to assess the primary result. It reports a Total Treated Set of 43 for safety, with 7 participants affected by serious adverse events, 39 by other adverse events, and 0 deaths. The posted record supports a description of the study and selected safety data, but not a conclusion about the primary efficacy result or a comparison with another treatment.