Clinical Trial on Ladarixin Adjunctive Therapy to Improve Glycemic Control in Type 1 Diabetes.: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Clinical Trial on Ladarixin Adjunctive Therapy to Improve Glycemic Control in Type 1 Diabetes. (NCT05368402), a randomized phase 2 study. The primary outcome is Number of Participants With an HbA1c Reduction From Baseline of ≥0.50% (Absolute Difference) Without Episodes of Severe Hypoglycemia..

2. Clinical Question

The clinical question was whether adding ladarixin, compared with matching placebo, could improve glycemic control in the intended population. The registry also lists glycemic variability, based on continuous glucose monitoring parameters, and safety as secondary objectives. These objectives describe what the trial set out to study; they do not establish that the corresponding outcomes could be evaluated. With only 1 participant in each arm, the record explicitly states that no efficacy evaluation was conducted.

3. Trial Design

This was a randomized, placebo-controlled, double-blinded, parallel-arm phase 2 trial of ladarixin as adjunctive therapy in overweight, insulin-resistant adults with type 1 diabetes. The registry describes masking as quadruple. Participants were assigned to ladarixin or a matching placebo, with the placebo following the same schedule as the study drug.

The planned ladarixin regimen was 400 mg twice daily for 7 cycles of 14 days, with 14 days off between cycles, for a total duration of 26 weeks. The primary outcome was scheduled for Visit 4 at week 27/28. The record does not report further details about the timing or conduct of other assessments beyond the listed outcomes and objectives.

The study began on 2022-09-14, and the recorded primary completion date is 2023-09-18. Its status is terminated. The primary outcome narrative says that 24 participants were screened, but only 2 were enrolled and randomized, with 1 assigned to each arm. It states that the sponsor decided to close enrollment on 04th August 2023 because of the low recruitment rate and to terminate the study early. The registry enrollment field lists 3; the record does not explain how that figure relates to the narrative count of 2 randomized participants.

The central design limitation is not simply that the study was smaller than intended: the registry says that the recruitment rate made a consistent sample size for formal statistical analyses impossible and that no efficacy evaluation was conducted on the randomized participants’ data. The registry record does not report a target sample size, a sample-size justification, or additional details about the planned statistical analysis. The posted arm counts and outcome values therefore offer only a very limited description of the participants represented in the 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

Analysis population for the primary outcome, as defined in the registry: There were only two participants recruited in the study e.g., 1 in the Ladarixin group and 1 in the placebo group. Due to the low recruitment rate, the Sponsor decided to close enrollment on 04th August 2023 and thus to early terminate the study. Accordingly, Population consists of the two randomized participants only.

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 Participants With an HbA1c Reduction From Baseline of ≥0.50% (Absolute Difference) Without Episodes of Severe Hypoglycemia., measured over At Visit 4 (week 27/28).

The primary endpoint was a count of participants who had both an HbA1c reduction from baseline of at least 0.50% in absolute terms and no episode of severe hypoglycemia. The registry lists the unit as participants and the assessment time as Visit 4, at week 27/28. Requiring the HbA1c reduction and absence of severe hypoglycemia makes this a combined outcome: a participant would need to satisfy both conditions to be counted.

HbA1c is used to summarize glycemic exposure over time, while the additional condition makes clear that the endpoint was not defined by HbA1c change alone. In general, a threshold-based endpoint can make a result easier to interpret as a count of participants meeting a specified criterion. It also discards information about how far an individual’s measurement changed beyond the threshold. That general trade-off matters particularly when the available group counts are very small.

The posted count is 0 for ladarixin and 0 for placebo, with 1 participant in each arm. These are descriptive values only. The registry specifically states that no efficacy evaluation was conducted, so the counts should not be treated as a formal comparison or as evidence of a treatment effect. The record does not report additional details about how baseline values, severe hypoglycemia episodes, or missing measurements were handled in the outcome calculation.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a binary participant-level outcome, reported as a count. Each participant either meets the combined criterion or does not: the HbA1c reduction must reach at least 0.50% in absolute terms, and the participant must have no episode of severe hypoglycemia. The registry describes the intended output as a count and percentage of participants. The posted arm values are counts, with 1 participant in each arm.

What does a count show?

A count records how many participants meet a defined outcome. A proportion expresses that count relative to the number assessed. In general, a proportion can be useful for comparing groups when each group contains enough observations to make the estimate informative. With only 1 participant in each arm here, the observed count is highly dependent on the experience of that individual. The registry accordingly says that no efficacy evaluation was conducted.

The endpoint combines an HbA1c threshold with a safety condition. This means that the count cannot be interpreted as a count of participants meeting the HbA1c threshold alone. A participant who had the specified HbA1c reduction but also experienced severe hypoglycemia would not meet the complete endpoint as written. The registry does not provide separate posted counts for the HbA1c component and the severe-hypoglycemia component, so the joint endpoint is the only primary outcome result.

Why is a formal comparison absent?

A comparison between randomized groups ordinarily aims to estimate how outcomes differ under the assigned interventions, while accounting for uncertainty. The registry states that low recruitment meant there was no consistent sample size for formal statistical analyses. It also says directly that no efficacy evaluation was conducted on the randomized subjects’ data. The arm counts should therefore not be recast as a statistical test or as a precise estimate of the difference between treatments.

In general, confidence intervals communicate uncertainty around an estimated effect, and p-values assess how compatible data are with a specified null hypothesis under a statistical model. Neither is reported for this primary outcome. Without a reported inferential analysis, there is no interval or test result to interpret. The absence of those statistics is not evidence that the groups were equivalent; it reflects the record’s statement that efficacy was not evaluated.

What can a planning illustration tell us?

Sample-size illustrations are planning exercises, not results observed in a trial. In general, such calculations depend on assumptions about the effect to detect, the significance level, allocation, and statistical method. Any planning values displayed alongside the results should be understood as hypothetical requirements under their stated assumptions, not as observed power and not as evidence that the terminated trial had those numbers of participants. The registry’s account of recruitment and the absence of a formal efficacy evaluation remain the relevant context for the posted outcome.

For a binary outcome, planning calculations also depend on how often the outcome is expected in each group. When the underlying event information is sparse, a planning result may be unstable or unavailable. The record does not report a formal sample-size justification for this trial. No target enrollment or protocol-based calculation is reported in the registry record, and the posted primary outcome counts do not supply a reliable basis for inferring what the original design assumptions were.

How should the posted values be read?

The displayed outcome values are 0 for ladarixin and 0 for placebo, with 1 participant in each arm. These are the counts recorded for the primary endpoint. They do not show the distribution of individual HbA1c changes, the timing or details of any measurements, or the results of a model-adjusted analysis. The record does not report such details. The registry describes the outcome as a count of participants, so interpretation should stay at that level.

The trial’s randomized, placebo-controlled design describes how treatment assignment was intended to work. Randomization can, in general, help balance known and unknown participant characteristics across groups when evaluated in a sufficiently informative study. It does not make a two-participant study capable of supporting a precise group comparison. In this record, the small number randomized and the absence of an efficacy evaluation limit what can be concluded from the design and observed counts.

The posted results include no analysis for the primary endpoint, and the record does not report an adjusted or unadjusted effect estimate. It also does not report methods for handling repeated measures, missing observations, or protocol deviations. Those methods should not be inferred from the fact that the trial was randomized or from the outcome definition. The plain interpretation is that the registry posts descriptive counts and explicitly states that it did not conduct an efficacy evaluation.

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("Ladarixin", "Placebo")
n      <- c(1, 1)
events <- c(0, 0)
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  Ladarixin  1  0  0.0  0.0  97.5  0.0  79.3  1.3  84.2
ARM  Placebo  1  0  0.0  0.0  97.5  0.0  79.3  1.3  84.2

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary objective of assessing glycemic control and secondary objectives concerning continuous-glucose-monitoring-derived glycemic variability and safety. It lists 2 outcome measures and 1 secondary outcome: the number of participants with treatment-emergent adverse events of any kind from the beginning of study treatment through the end of participation. The record reports no statistical analyses. It does not describe a multiplicity strategy, such as how multiple outcomes or tests would be handled in a formal analysis.

In general, multiplicity matters when several outcomes or comparisons are tested because examining many results can increase the chance of a misleading positive finding. That general issue does not imply that a particular adjustment was planned. No adjustment method or testing hierarchy is reported in the registry record. Given that no efficacy evaluation was conducted, the posted results do not allow a reader to assess how multiplicity would have affected formal efficacy conclusions.

The record does not report how missing data were handled, whether any outcome measurements were missing, or whether participants who discontinued would be included in a particular analysis. It also does not describe a detailed analysis population beyond the primary outcome narrative’s reference to the 2 randomized participants. The narrative says there was 1 participant in each arm and that the randomized data were not used for an efficacy evaluation.

These omissions should not be filled in by assuming common trial practices. The registry does not report an intention-to-treat, per-protocol, or other named analysis population, nor does it describe imputation or sensitivity analyses. The stated limitation is that recruitment did not permit a consistent sample size for formal statistical analyses. Consequently, the posted counts should be read as registry summaries, not as the output of an analysis population or missing-data procedure that the record does not document.

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 0% reporting threshold.

The posted adverse-event summary lists 1 participant at risk in each arm. It reports 0 participants with serious adverse events in the ladarixin arm and 0 in the placebo arm. It also reports 0 participants with other adverse events in each arm and 0 deaths in each arm. These figures are counts, not estimates of comparative risk.

The secondary outcome description concerns treatment-emergent adverse events of any kind, from the beginning of study treatment through the end of study participation. The adverse-event summary distinguishes serious events from other events. The record does not provide further event-level descriptions; no serious adverse event terms are listed.

These zero counts should be interpreted in light of the very small number of participants. With only 1 participant at risk per arm, the summary describes the events recorded for those participants but cannot characterize the range or frequency of potential events in a larger population. A zero count in this study does not establish that an event cannot occur or that the groups have comparable safety profiles. It is a limited description of the posted record, not a broad safety conclusion.

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

The primary result is a pair of descriptive counts: 0 participants met the combined endpoint in the ladarixin arm, and 0 met it in the placebo arm. There was 1 participant in each arm. The endpoint required an HbA1c reduction from baseline of at least 0.50% in absolute terms without episodes of severe hypoglycemia, and it was assessed at Visit 4, at week 27/28.

This result does not tell us whether ladarixin changes glycemic control compared with placebo. The registry explicitly says that no efficacy evaluation was conducted because only 2 participants were randomized and the recruitment rate did not permit a consistent sample size for formal statistical analyses. There is no reported effect estimate, confidence interval, or p-value for the primary comparison. The counts should not be interpreted as showing equivalence, benefit, or lack of benefit.

The trial’s intended population was overweight, insulin-resistant adults with type 1 diabetes, but the posted data describe only the 2 randomized participants. The record states that 24 people were screened and that enrollment was closed on 04th August 2023, followed by early termination. These circumstances limit how much the posted values can say about the question the trial set out to address.

The safety summary likewise records zero participants with serious adverse events, zero with other adverse events, and zero deaths in each arm, with 1 participant at risk per arm. These figures describe the registry summary; they do not establish the safety of treatment more generally. The record does not report detailed event terms or a broader safety analysis.

In short, the page presents what the registry recorded and the constraints it states. It does not provide evidence for a reliable treatment comparison. Conclusions about a larger or different population, or about outcomes beyond those posted, are not supported by this record.

14. Why This Trial Matters Statistically

This trial illustrates why a registered randomized design and a clinically defined endpoint do not by themselves guarantee an interpretable efficacy comparison. Randomization, placebo control, and masking are important design features in general, but their value for estimating a group difference depends on having enough outcome information. Here, the registry reports only 2 randomized participants and states that no efficacy evaluation was conducted.

The primary endpoint is clearly framed as a participant count combining an HbA1c threshold with the absence of severe hypoglycemia. Yet the posted count is 0 in each arm, and each arm contains 1 participant. That makes the result easy to state but not a dependable estimate of what might occur in the intended population. A precise endpoint definition cannot compensate for the limited information in the observed data.

The record also makes an important distinction between an outcome being registered and an outcome being formally analyzed. It lists primary and secondary objectives and posts outcome values, but it reports no analyses. Readers should not mistake registry values for a complete analysis or infer that an unreported analysis was performed. The statement that no efficacy evaluation was conducted is central to the statistical interpretation.

Finally, the enrollment figures highlight the importance of transparent accounting. The outcome narrative describes 2 participants enrolled and randomized, while the registry enrollment field lists 3. The record does not reconcile these figures. Noting this difference helps keep the evidence description faithful without guessing which number should be treated as definitive for every purpose.

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 most direct limitation is the very small randomized group: the primary outcome narrative describes 2 participants, with 1 in each arm. The registry says this low recruitment prevented a consistent sample size for formal statistical analyses and that no efficacy evaluation was conducted. Consequently, the posted primary endpoint counts cannot support a dependable treatment comparison.

The study was terminated early after recruitment was closed on 04th August 2023. The record attributes this decision to the low recruitment rate. It reports 24 participants screened and 2 enrolled in the primary outcome narrative, while the enrollment field lists 3. The record does not reconcile the difference, so the figures should be presented with their contexts rather than silently combined.

The available primary outcome data are limited to the counts for the combined HbA1c and severe-hypoglycemia endpoint. The record does not report separate component results, individual HbA1c changes, an effect estimate, a confidence interval, or a p-value. It also does not report methods for missing observations or a named analysis population. These details should not be inferred.

The safety summary has the same small-participant limitation: each arm has 1 participant at risk. The record reports zero serious adverse events, zero other adverse events, and zero deaths in each arm, but does not provide further event-level detail. These counts cannot establish the frequency of events beyond the participants represented in the registry.

Finally, the registry record does not report a sample-size justification, a detailed statistical plan, or a multiplicity strategy. The absence of this information limits assessment of the intended inferential approach. The explicit statement that no efficacy evaluation was conducted remains the key interpretive constraint.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Clinical Trial on Ladarixin Adjunctive Therapy to Improve Glycemic Control in Type 1 Diabetes. — NCT05368402.
• Registry record last updated 2025-02-03; status last verified 2025-01.
• Results first posted 2025-02-03.
• 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

This phase 2 randomized, placebo-controlled, double-blinded, parallel-arm trial studied oral ladarixin as adjunctive therapy for overweight, insulin-resistant adults with type 1 diabetes. The planned regimen was 400 mg twice daily across 7 cycles of 14 days, with 14 days off between cycles, for a total of 26 weeks. The comparator was matching placebo on the same schedule.

The primary outcome counted participants with an HbA1c reduction from baseline of at least 0.50% in absolute terms without episodes of severe hypoglycemia, assessed at Visit 4, at week 27/28. The registry posts 0 participants meeting this endpoint in each arm, with 1 participant per arm. It states that low recruitment prevented formal statistical analyses and that no efficacy evaluation was conducted.

The study status is terminated. The record reports low recruitment, early closure of enrollment, and early termination. Its enrollment field lists 3, while the primary outcome narrative describes 2 randomized participants; the difference is not explained. The adverse-event summary reports no serious adverse events, other adverse events, or deaths in either arm. These small counts are descriptive and do not establish a comparative efficacy or safety conclusion.