A Study of LY3053102 in Healthy Participants: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study of LY3053102 in Healthy Participants (NCT01736241), a randomized phase 1 study. The primary outcome is Number of Participants With One or More Adverse Events (AEs) or Any Serious AEs.

2. Clinical Question

This was a safety, tolerability, and pharmacokinetic study, rather than a trial of clinical benefit in a patient population. Its stated purpose was to evaluate safety and tolerability, and to investigate how much study drug entered the bloodstream and how long the body took to dispose of it. Information about side effects was also collected. The registered secondary outcomes include area under the concentration-time curve, observed maximum drug concentration, and development of anti-LY3053102 antibodies.

The primary endpoint is a participant-level count: a participant is counted if at least one qualifying event occurred. The record gives the number with an event in each dose group and placebo, but does not identify the specific events within this endpoint summary. Detailed event descriptions are directed to the Reported Adverse Events module. The principal reading is therefore descriptive and limited to the posted counts and follow-up timeframe.

3. Trial Design

The registry describes a Phase 1, randomized, double-masked, parallel-group study. Its official title identifies it as a single-dose, dose-escalation study of LY3053102 in healthy subjects. The study was sponsored by Eli Lilly and Company and is recorded as completed. The registry lists the start as 2012-12 and primary completion as 2013-07.

Participants were assigned to six LY3053102 dose-level cohorts or placebo. A single dose was administered subcutaneously. The LY3053102 doses were 2 mg, 7 mg, 20 mg, 50 mg, 150 mg, and 405 mg. The record states that dose escalation was based on safety results over at least a 7-day evaluation period after dosing. The text describing the escalation procedure is incomplete, so further details about the sequence or decision rules are not reported in the registry record.

The posted endpoint groups contain 6 participants for 2 mg, 6 for 7 mg, 6 for 20 mg, 5 for 50 mg, 6 for 150 mg, 6 for 405 mg, and 6 for placebo. These are the group sizes attached to the primary outcome results. The record reports 41 enrolled overall. The arm descriptions indicate a placebo participant in each dose-level cohort; they do not establish the allocation process beyond what is explicitly stated in the record.

The primary safety timeframe was baseline through Day 31. The brief summary says each participant was expected to be in the study for approximately 8 weeks, including up to 4 weeks from screening to study-drug administration and an additional 4 weeks of follow-up. That expected participation period is distinct from the stated primary endpoint timeframe.

Secondary outcomes concerned pharmacokinetic measures and anti-LY3053102 antibodies. Three secondary outcomes are listed, but results for them are not included in the registry record. The registry record does not report a detailed statistical analysis plan, a sample-size justification, or further design information about the masking procedure.

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.

Only the first 6 of 7 groups are shown.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Participants who received at least one dose of LY3053102 or placebo.

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: All enrolled participants.

Only the first 6 of 8 groups are shown.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With One or More Adverse Events (AEs) or Any Serious AEs, measured over Baseline through Day 31.

The primary endpoint is the number of participants with one or more adverse events or any serious adverse events, regardless of causality, from baseline through Day 31. Its unit is participants. It is a safety endpoint: it summarizes whether a participant experienced at least one qualifying event during the specified period, rather than counting the total number of events or measuring their severity.

A participant-level endpoint is useful for presenting how broadly events occurred across a group. It makes a direct descriptive summary possible even when participants may experience different event types or more than one event. However, this summary does not show how many events each participant experienced, what those events were, when they occurred, or whether investigators considered them related to study drug. The endpoint description expressly includes events regardless of causality.

The posted primary outcome counts are 5 of 6 participants for 2 mg LY3053102, 6 of 6 for 7 mg, 4 of 6 for 20 mg, 4 of 5 for 50 mg, 6 of 6 for 150 mg, 5 of 6 for 405 mg, and 6 of 6 for placebo. These are descriptive group results, not estimates accompanied by uncertainty intervals or a reported hypothesis test.

The registry points readers to the Reported Adverse Events module for a summary of serious and other non-serious events. That distinction matters: the primary endpoint combines participants with one or more adverse events or any serious adverse events, whereas the safety summary also presents serious and other non-serious events separately. The endpoint does not by itself establish a causal relationship or a comparative safety conclusion.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The posted primary outcome is a count of participants with at least one adverse event or any serious adverse event. This is a binary outcome at the participant level: each participant either meets the endpoint definition during the timeframe or does not. The registry presents the resulting number of participants for each dose group and placebo. A count is easy to read descriptively, but it does not show the uncertainty around the underlying event frequency or provide a comparison by itself.

What does a confidence interval communicate?

General statistical explanation: a confidence interval is a range of values compatible with the observed data under a specified statistical procedure. It expresses uncertainty in an estimate; it is not a guarantee that the true value lies in that particular interval. The registry results do not include confidence intervals for the primary endpoint. Without them, readers cannot use the posted results to assess the precision of a group-level event estimate through an interval.

What is missing from the two-arm comparison?

The registry record indicates that values are missing or not estimable for a two-arm comparison of this binary outcome. No comparative estimate, confidence interval, or p-value is available in the results. The group counts can be read separately, but a comparison requires a defined contrast between groups and an analysis that estimates it. The record does not report such an analysis for the primary outcome. For that reason, the counts should not be treated as evidence of a statistically established difference or lack of difference.

General statistical explanation: a p-value, when reported, summarizes how incompatible observed data are with a specified null hypothesis under a statistical model. It is not the probability that the null hypothesis is true, and it does not measure the size or clinical importance of an effect. No p-value for the primary comparison is reported in the registry record, so no such interpretation is available.

Why does the small group size matter?

The posted outcome groups contain 5 or 6 participants each. General statistical explanation: in a small group, each observed participant has a relatively large influence on the group count. This makes estimates imprecise and can make comparisons unstable. A difference in the observed counts, if one is visible in a display, is not automatically evidence of a reliable underlying difference. Inferential methods and uncertainty estimates are needed to evaluate how compatible the observations are with a range of possible effects.

The record also does not provide an adjusted analysis or a prespecified statistical model for these counts. The statistical analysis plan is not reported in the registry record. It is therefore not possible to describe the exact test, model assumptions, covariate adjustments, or multiplicity strategy used for the primary endpoint. No assumptions beyond those explicitly shown in the record should be attributed to the trial.

How should the safety summaries be distinguished?

The primary endpoint is a combined participant-level measure: one or more adverse events or any serious adverse events. The separate safety fields report serious events and other non-serious events by group. These summaries answer related but not identical descriptive questions. A participant can contribute to the primary endpoint because of a non-serious event even when the serious-event count is zero. The record's endpoint description says events are included regardless of causality, so these counts should not be interpreted as events judged to have been caused by treatment.

General statistical explanation: over-dispersion is a feature of some count data in which variability exceeds what a simple model assumes. It matters when modelling numbers of events, because underestimated variability can make uncertainty appear too small. Here, however, the posted primary measure is the number of participants with an event, not the total event count. The results do not report a count model or an over-dispersion assessment. It would not be appropriate to claim that a particular model was used or that over-dispersion was present.

What do the planning assumptions mean?

The record describes planning illustrations based on an observed effect, a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect. Such illustrations are planning calculations, not observed evidence and not a description of the trial's original design. The record explicitly distinguishes them from the trial's own design. Since the two-arm comparison is not estimable for this binary outcome, no such planning values should be interpreted as a reliable comparative result.

General statistical explanation: a sample-size calculation asks how many participants would be needed under particular assumptions to detect a specified effect with a chosen error rate and power. It depends on the assumed effect, variability or event frequency, allocation, and analysis method. It does not reveal the probability that the completed trial's findings are correct, and it is not observed power. A sample-size justification for the original study is not reported in the registry record. The posted group sizes alone do not establish that the study was adequately powered for a comparison.

The endpoint has a stated timeframe, from baseline through Day 31, and a stated population of participants who received at least one dose. Those definitions clarify which participants and observations belong in the outcome summary. The detailed handling of missing outcome data, protocol deviations, and analysis populations beyond the stated treated population is not reported in the registry record. Consequently, further analysis details cannot be inferred from the posted counts.

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("2 mg LY3053102", "7 mg LY3053102", "20 mg LY3053102", "50 mg LY3053102", "150 mg LY3053102", "405 mg LY3053102")
n      <- c(6, 6, 6, 5, 6, 6)
events <- c(5, 6, 4, 4, 6, 5)
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  2 mg LY3053102  6  5  83.3  35.9  99.6  43.6  97.0  42.1  96.3
ARM  7 mg LY3053102  6  6  100.0  54.1  100.0  61.0  100.0  59.0  99.6
ARM  20 mg LY3053102  6  4  66.7  22.3  95.7  30.0  90.3  29.0  90.1
ARM  50 mg LY3053102  5  4  80.0  28.4  99.5  37.6  96.4  35.9  95.7
ARM  150 mg LY3053102  6  6  100.0  54.1  100.0  61.0  100.0  59.0  99.6
ARM  405 mg LY3053102  6  5  83.3  35.9  99.6  43.6  97.0  42.1  96.3

11. Multiplicity, Missing Data and Analysis Populations

The registry lists three secondary outcomes: area under the concentration-time curve for LY3053102, observed maximum drug concentration, and the number of participants who developed anti-LY3053102 antibodies. The registry entries do not include numerical results for those outcomes. They also do not state whether the trial applied a multiplicity adjustment, used a hierarchy of hypotheses, or treated the secondary outcomes as exploratory. Those details are not reported in the registry record.

Multiplicity is the issue of interpreting several statistical tests or outcome comparisons together. General statistical context: when many hypotheses are tested, the chance of at least one apparently notable result can increase if no adjustment or prespecified testing strategy is used. Whether that concern applies to a particular analysis depends on the protocol and analysis plan. Since that plan is not reported, no claim can be made about how multiplicity was addressed.

The primary outcome population is described as participants who received at least one dose of LY3053102 or placebo. This identifies a treated population for the endpoint, but the registry record does not report a fuller analysis-population definition, such as how exclusions or protocol deviations were handled. It also does not report the number of participants with missing endpoint information or a method for handling missing data. The posted group counts are therefore best read as the results shown, without assuming an imputation method or a broader intention-to-treat analysis.

The registry lists zero analyses for the recorded outcomes and flags the binary two-arm comparison as missing or not estimable. The reason for that status, beyond the information that values are missing or not estimable, is not reported in the registry record. No interim analysis plan or stopping rule is reported. These absences limit what can be said about the analysis process, but they do not change the descriptive meaning of the posted counts.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent non-serious adverse event terms

The serious adverse event summary reports zero participants with serious adverse events in each of the six LY3053102 dose groups and in the placebo group. The number at risk was 6 for the 2 mg, 7 mg, 20 mg, 150 mg, 405 mg, and placebo groups, and 5 for the 50 mg group. These are the posted serious-event figures for the recorded groups.

This finding is limited to the serious-event category and the reporting represented in the registry record. It does not mean that no adverse events of any kind occurred. The primary endpoint counts participants with one or more adverse events or any serious adverse events, and the other non-serious event counts are 5, 6, 4, 4, 6, 5, and 6 across the groups in dose order and placebo. The record says the primary endpoint includes events regardless of causality.

Zero observed serious events in a small group is a descriptive result, not proof that a serious event cannot occur or that groups have identical risk. General statistical context: when the number of observed participants is limited, the data may be compatible with a range of underlying event frequencies. No confidence intervals for these safety counts are reported. Death counts are not reported in the registry record, and no serious-event terms are listed in the registry record.

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

This trial's posted primary results describe how many participants had one or more adverse events or any serious adverse events from baseline through Day 31. The counts are available for six single-dose LY3053102 groups and placebo. The safety summary separately reports zero participants with serious adverse events in each group. Taken together, these are descriptive findings from the registry record for the stated population and timeframe.

They do not establish a comparative effect of LY3053102 versus placebo. The record does not provide an estimable two-arm comparison for the binary endpoint, nor a confidence interval or p-value for such a comparison. The observed counts cannot on their own establish whether any difference is due to chance, nor do they quantify the precision of a comparative estimate. The absence of a reported comparison is not evidence that groups are equivalent.

The results also do not show which specific adverse events occurred, their severity or duration, or whether investigators judged them related to study drug. The endpoint description says events were counted regardless of causality. Further event-level detail is referred to the Reported Adverse Events module, and the additional event details are not reported in the registry record.

The study was conducted in healthy participants and was designed to evaluate safety, tolerability, and pharmacokinetics after single doses. The primary counts do not answer questions about clinical benefit in a disease population, repeated dosing, or outcomes beyond the stated follow-up. Although pharmacokinetic and antibody outcomes are listed, their results are not included.

In short, the data show the recorded event counts across the listed groups, and no serious adverse events are recorded for those groups. They do not support a conclusion about comparative risk, causality, or clinical effectiveness. Those questions require information and analyses that are not reported in the registry record.

14. Why This Trial Matters Statistically

Statistically, this trial illustrates the difference between a descriptive safety summary and an inferential comparison. The registry provides participant counts for each dose level and placebo, which can show what was observed in the enrolled groups. It does not provide an estimable two-arm analysis for the primary binary endpoint. A visible pattern across group counts is therefore not the same as a tested dose-response relationship or a treatment-placebo effect.

The dose-escalation structure is also relevant to interpretation. The record states that dose escalation was based on safety results over at least a 7-day evaluation period after dosing. This places safety review within the study design, but the available information does not give the detailed rules or decision process. The record's truncated description of escalation leaves further procedural details unreported.

The study also makes clear why endpoint definition matters. The primary measure is a participant count with one or more adverse events or any serious adverse events, while the safety summary separately distinguishes serious and other non-serious events. Knowing which event categories are combined helps prevent a count of participants with any event from being mistaken for a count of serious events.

Finally, the record lists pharmacokinetic and antibody outcomes alongside safety outcomes, reflecting several distinct questions addressed by the study. Their results and the statistical methods used for them are not included in the information. The available record is therefore useful for understanding the design and the reported safety counts, while also showing why interpretation must be bounded by the analyses and details actually reported.

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.

Several limits apply specifically to this record. The study enrolled healthy participants and evaluated single subcutaneous doses. Its results do not directly describe a patient population or repeated administration. The primary endpoint covers baseline through Day 31, while the brief summary gives an expected participation period of approximately 8 weeks. These timeframes describe different parts of the study and should not be conflated.

The group sizes are 5 or 6 participants, and the record does not provide uncertainty intervals for the primary counts. The two-arm comparison is marked as missing or not estimable. Thus, the results support descriptive reporting only; the record does not provide a basis for a formal comparative conclusion. A sample-size justification for the trial is not reported in the registry record.

The dose-escalation description says decisions were based on safety results over at least a 7-day evaluation period, but its text is incomplete. Detailed escalation rules, allocation procedures, and masking implementation are not reported in the registry record. The statistical analysis plan, interim analyses, and any stopping rules are also not reported.

Although three secondary outcomes are listed, their numerical results are not described. Details on missing endpoint data, analysis populations beyond the stated participants who received at least one dose, and multiplicity procedures are not reported. The primary endpoint counts do not provide event-specific details, causality assessments, or severity and duration information. Death counts are not reported in the registry record. These limitations constrain interpretation to the registry entries shown and do not support broader conclusions.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study of LY3053102 in Healthy Participants — NCT01736241.
• Registry record last updated 2018-07-19; status last verified 2017-09.
• Results first posted 2018-07-19.
• 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 1 study evaluated single-dose LY3053102 in healthy participants. The registry describes a randomized, double-masked, parallel-group design with six escalating subcutaneous dose levels and a placebo group. It reports 41 participants enrolled overall and lists the study start as 2012-12 and primary completion as 2013-07.

The primary outcome was the number of participants with one or more adverse events or any serious adverse events from baseline through Day 31. The posted group counts range from 4 to 6 participants, with group sizes of 5 or 6. The separate serious adverse event summary reports zero affected participants in each group. The record does not provide an estimable two-arm comparison, confidence intervals, or p-values for the primary endpoint.

Pharmacokinetic and antibody outcomes are listed, but their results are not reported in the registry record. The record also does not report the detailed statistical plan, sample-size justification, or missing-data procedures. Interpretation is therefore limited to the stated design and descriptive safety counts.