Evaluation of SPH3127 in Patients With Mild-to-Moderate Ulcerative Colitis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Evaluation of SPH3127 in Patients With Mild-to-Moderate Ulcerative Colitis (NCT05019742), a randomized phase 2 study. The primary outcome is Number of Patients With Clinical Remission.

2. Clinical Question

The registry describes SPH3127-US-01 as a randomized, double-blind, placebo-controlled study intended to examine safety, pharmacokinetics, and preliminary efficacy in mild-to-moderate ulcerative colitis. The posted primary outcome is a count: the number of participants with clinical remission between screening (baseline) and Day 56. It is not a continuous score or a time-to-event measure.

The recorded counts are 0 of 2 participants in the placebo group, no participants enrolled in the SPH3127 50 mg group, and 1 of 1 participant in the SPH3127 100 mg group. These small and unequal group sizes describe what was recorded, but they do not establish a stable treatment comparison. In particular, the 50 mg group has no enrolled participants, and the 100 mg group has only one.

The intended clinical question and the question answerable from the posted data are therefore different in scope. The trial was designed to investigate several aspects of SPH3127, while the available remission counts provide only a very limited descriptive account. The registry record does not report a comparative estimate for the primary outcome or an inferential analysis that would quantify uncertainty around a treatment contrast.

3. Trial Design

This was a Phase 2, randomized, quadruple-masked, parallel-group trial sponsored by Shanghai Pharma Biotherapeutics USA Inc. Its title describes a double-blind, placebo-controlled investigation of SPH3127 pharmacokinetics, pharmacodynamics, safety, and tolerability. The brief summary also identifies preliminary efficacy as an objective. The registry status is terminated.

The registry lists placebo, SPH3127 50 mg, and SPH3127 100 mg arms. During the initial 8 weeks, the placebo arm received 2 placebo tablets daily, one in the morning and one in the evening. The 50 mg arm received one 50 mg SPH3127 tablet in the morning and one placebo tablet in the evening. The 100 mg arm received one 50 mg SPH3127 tablet in the morning and one 50 mg tablet in the evening. The primary outcome timeframe runs from screening (baseline) to Day 56.

The arm descriptions also refer to a possible additional treatment period after the initial 8 weeks. Participants in the placebo arm could optionally be randomized to one of two SPH3127 daily treatment arms for an additional 10 months. The active-treatment arm descriptions refer to optional continuation of daily treatment for an additional 10 months. These descriptions do not establish how many participants entered or completed any optional period.

The registry reports a start date of 2022-03-21 and a primary completion date of 2022-11-01. Enrollment was 3. The record does not report why the trial was terminated, the allocation ratio, the planned sample size, or the details of sequence generation and concealment. Those features should not be inferred from the randomized label or from the observed group sizes.

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: Patients must meet I/E criteria to enroll.

Overall Study

Analysis population for the primary outcome, as defined in the registry: No (0) subjects enrolled in SPH3127 50 mg group

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: Only 3 patients enrolled, none (0) in the SPH3127 50 mg group.

6. Primary Endpoint

The registry lists the primary outcome as Number of Patients With Clinical Remission, measured over Screening (baseline) to Day 56.

The primary endpoint is the number of participants with clinical remission from baseline to Day 56. The registry identifies the unit as participants and the measure as a count. This is a binary outcome at the participant level: for each person, the endpoint records whether clinical remission was present over the stated timeframe, and the posted result counts participants meeting that condition.

Clinical remission is used as an endpoint because it expresses a clinically interpretable status rather than a laboratory measurement alone. In a randomized study, a comparison of remission frequencies can describe whether the outcome was recorded more often in one assigned group than another. The registry record does not provide the criteria used to define clinical remission, so the specific threshold or assessment rules are not reported in the registry record.

The posted primary results are 0 participants in the placebo group, 0 participants in the SPH3127 50 mg group, and 1 participant in the SPH3127 100 mg group. The corresponding group sizes are 2.0, 0.0, and 1.0. Because no participants were enrolled in the 50 mg arm and only one was enrolled in the 100 mg arm, these counts cannot support a precise estimate of comparative remission frequency. The registry does not post a confidence interval or a statistical test for this endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is binary at the participant level and is reported as a count of participants with clinical remission. For this kind of endpoint, a comparative analysis would generally summarize the frequency of the event in each group and estimate a contrast between groups. The registry record reports group counts but does not post a comparative estimate, confidence interval, or p-value. It also indicates that values were missing or not estimable for a two-arm comparison of the binary endpoint.

What a confidence interval would describe

General statistical explanation: a confidence interval is a range of values compatible with the observed data under a specified statistical method. It communicates uncertainty around an estimate; it does not guarantee that the true value lies within the interval, nor does it remove limitations caused by a very small sample. No confidence interval for the primary comparison is reported in the registry record, so there is no interval-based precision statement to interpret for this trial.

Why sparse counts matter

General statistical explanation: when an outcome is binary, a small number of participants can produce highly unstable estimates. If a group has no participants, its outcome frequency cannot be estimated from observed participants. If a group has only one participant, that individual’s outcome determines the group’s observed count. These properties apply to the posted arm sizes: there were no participants in the SPH3127 50 mg group and one participant in the SPH3127 100 mg group. Sparse data also limit the usefulness of standard large-sample approximations and make a two-arm comparison difficult or not estimable.

The registry results do not identify which specific statistical method was intended for analysis. The method for handling sparse binary data, the planned contrast, and any adjustment for baseline factors are not reported in the registry record. No method should be attributed to the trial without that information.

What the planning assumptions mean

General statistical explanation: sample-size planning asks how many participants would be needed under stated assumptions to detect a specified effect with a chosen probability of detecting it, if the effect exists. The planning description associated with these results specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the effect to detect as a stated fraction of the observed effect. These are planning assumptions, not evidence that the trial enrolled enough participants or had a particular probability of detecting an effect.

Any sample-size illustrations based on the observed effect should be read as hypothetical planning exercises. They are not observed power, do not recover the original design rationale, and do not show what the trial was intended to enroll. The registry record does not report the protocol’s sample-size justification. Given the sparse posted outcomes, an illustration derived from an observed effect would also be sensitive to the very limited data underlying that effect.

Analyses and multiplicity

The registry reports no analysis entries. It lists clinical remission as the primary outcome and identifies adverse events as a secondary outcome; it also indicates 3 outcome measures overall. The record does not report the analysis hierarchy, the full statistical analysis plan, or any adjustment for testing multiple outcomes or treatment comparisons. General statistical explanation: when multiple outcomes or comparisons are tested, the chance of at least one false-positive finding can increase unless the analysis plan accounts for multiplicity. That principle does not establish that a particular adjustment was required or used in this trial.

The registry record also does not report analysis populations, such as whether the primary analysis was intended to include all randomized participants or another defined group. Although the study is described as randomized, the posted results do not give enough information to reconstruct the planned analysis set or verify how participants were handled after randomization.

Missing data and interpretation

General statistical explanation: missing outcome data can affect a treatment comparison if outcome availability differs between groups or is related to participants’ outcomes. A sound interpretation depends on knowing how missing assessments were defined and handled. The registry record does not report a missing-data method, rules for withdrawals, or whether the posted counts include all expected assessments. It would therefore be inappropriate to assume that unreported or unavailable outcomes were treated as either remission or non-remission.

These methodological limits mean the posted counts should be treated as descriptive. The registry record does not contain enough information to reproduce a comparative analysis, assess its assumptions, or determine whether any planned inferential methods were followed.

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("Placebo", "SPH3127 100 mg")
n      <- c(2, 1)
events <- c(0, 1)
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  Placebo  2  0  0.0  0.0  84.2  0.0  65.8  0.8  70.8
ARM  SPH3127 100 mg  1  1  100.0  2.5  100.0  20.7  100.0  15.8  98.7

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists clinical remission as the primary outcome and identifies the number of patients reporting adverse events as a secondary outcome. It also indicates 3 outcome measures overall, but the other outcome measures are not reported in the registry record. No analysis entries are posted. The planned hierarchy among outcomes, the number of treatment comparisons intended, and any multiplicity adjustment are not reported in the registry record.

General statistical explanation: multiplicity arises when several hypotheses are tested, such as across multiple outcomes or treatment comparisons. Without an analysis plan, readers cannot tell whether testing was intended to be confirmatory or exploratory, or how any increased false-positive risk was to be managed. No conclusion about the trial’s actual multiplicity strategy can be drawn from the posted information.

The registry record does not describe the analysis population, the handling of participants who withdrew, or procedures for missing outcome measurements. It does not report an imputation method or a sensitivity analysis for missing data. The observed arm sizes and outcome counts do not establish whether all expected endpoint assessments were available.

In particular, the primary outcome population description states that no subjects were enrolled in the SPH3127 50 mg group. With no participants in that group, there is no observed remission outcome for that arm. The record does not report how the original analysis plan would have treated this circumstance. These omissions prevent assessment of whether missingness or deviations from a planned analysis could influence a comparison.

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.

Most frequent non-serious adverse event terms

The registry reports serious adverse events for each group with participants at risk. In the placebo group, 0 serious adverse events were reported among 2 participants at risk. In the SPH3127 100 mg group, 0 serious adverse events were reported among 1 participant at risk. The SPH3127 50 mg group had 0 participants at risk, so the record provides no safety observation for that arm. No deaths were reported in any of the listed groups.

These figures describe events recorded among the participants represented in the registry. They do not demonstrate that serious adverse events cannot occur, and they cannot provide a precise estimate of risk in a larger population. With so few participants, the absence of a recorded serious event is especially limited evidence about uncommon events. It also does not permit a meaningful comparison of serious adverse-event frequency between SPH3127 and placebo.

The record separately reports other adverse events for 2 of 2 participants at risk in the placebo group and 0 of 1 participant at risk in the SPH3127 100 mg group. No participants were at risk in the SPH3127 50 mg group. Those counts should not be confused with serious adverse events. The registry does not provide serious adverse-event terms; the list of top serious terms is empty. The record does not report enough detail to evaluate event timing, severity beyond the serious classification, or causal attribution.

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

The results establish a narrow descriptive fact: among the participants represented in the posted primary outcome counts, clinical remission was recorded for 0 participants in the placebo group and 1 participant in the SPH3127 100 mg group. The SPH3127 50 mg group had no enrolled participants. These counts reflect a total enrollment of 3 and should be interpreted in that context.

They do not establish that SPH3127 changes the chance of remission, that the treatment groups are equivalent, or that any apparent difference would persist in a larger study. The record contains no comparative analysis, confidence interval, or p-value. It also does not report the clinical remission criteria or the planned method for evaluating the primary outcome. For these reasons, the outcome counts alone cannot support a conclusion about the size or precision of a treatment effect.

The same restraint applies to safety. No serious adverse events were recorded among the 2 placebo participants and the 1 participant in the SPH3127 100 mg group represented in the safety results. This does not rule out adverse events, particularly uncommon events, and it cannot establish comparative safety. No participant was at risk in the SPH3127 50 mg group.

The trial’s stated scope included pharmacokinetics, pharmacodynamics, safety, tolerability, and preliminary efficacy. The available registry information does not provide detailed results for all of those aims. It lists adverse events as a secondary outcome and indicates 3 outcome measures overall, but other measure details are not reported in the registry record. The posted remission and safety counts should not be taken as a complete account of every study objective.

The registry status is terminated, but the reason is not reported in the registry record. It is not possible to determine why the study ended or whether the posted results represent all assessments planned by the investigators. The most defensible conclusion is that the record contains very limited descriptive outcome information and no reported inferential comparison.

14. Why This Trial Matters Statistically

This trial illustrates a basic distinction between a recorded result and a result that can answer a comparative clinical question. Counts can be accurate descriptions of individual study groups while still being too sparse to support a stable estimate of how groups differ. Here, enrollment was 3, the SPH3127 50 mg group had no participants, and the SPH3127 100 mg group had one. Those details sharply limit what can be learned from the remission outcome.

The case also shows why an endpoint’s definition and analysis plan matter. Clinical remission is presented as the primary outcome, but the registry record does not state the criteria used to classify remission or the method planned for comparing groups. Without those details, readers cannot assess how the endpoint was operationalized or reproduce an inferential analysis.

Randomization and masking are important design features, but their labels do not overcome a lack of outcome information. A randomized, quadruple-masked, parallel-group design can reduce certain sources of bias when implemented as intended. It cannot make a sparse sample precise, fill an arm with no participants, or supply missing analysis details. The registry does not report enough information to assess implementation of those design features.

Finally, the termination status and small enrollment underscore the value of distinguishing planned design from observed evidence. The record does not report the reason for termination or the original sample-size justification. Planning illustrations based on the observed effect are not a substitute for that design information, and they are not evidence of observed power. Statistically, this record is useful as an example of how careful interpretation depends not only on the direction of counts, but also on the amount of information and the transparency of the analysis.

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 important trial-specific limitation is the very small enrollment: 3 participants. The posted group sizes are 2.0 in the placebo arm, 0.0 in the SPH3127 50 mg arm, and 1.0 in the SPH3127 100 mg arm. The missing 50 mg arm observations prevent a comparison involving that group, while the single participant in the 100 mg arm provides little information about outcomes in that treatment group.

The registry reports a terminated status but does not report the reason for termination. It also does not report whether enrollment or termination differed from the original plan, the intended sample size, or the design rationale. These omissions limit assessment of how the observed trial relates to its planned objectives.

The primary endpoint is named as clinical remission from baseline to Day 56, but its diagnostic or scoring criteria are not reported in the registry record. The record also does not report outcome-assessment procedures, assessor roles, analysis populations, handling of withdrawals, or missing-data methods. Thus, the reported counts cannot be independently placed within a fully specified analysis framework.

No inferential analysis is posted, and values are missing or not estimable for a two-arm comparison of the binary endpoint. The record does not provide a comparative estimate or interval describing its precision. The safety results are likewise restricted to very few participants, with no participants at risk in the 50 mg group. They cannot characterize less common events or support a stable comparison of safety outcomes.

The arm descriptions mention optional treatment after the initial 8 weeks, but the registry does not report how many participants entered or completed that period. The record also indicates 3 outcome measures overall while naming only one secondary outcome. These gaps constrain interpretation of longer-term follow-up and of the full set of measured outcomes.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Evaluation of SPH3127 in Patients With Mild-to-Moderate Ulcerative Colitis — NCT05019742.
• Registry record last updated 2025-06-26; status last verified 2025-06.
• Results first posted 2025-06-26.
• 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

NCT05019742 is a Phase 2 randomized, quadruple-masked, parallel-group, placebo-controlled trial of SPH3127 in patients with mild-to-moderate ulcerative colitis. The sponsor is Shanghai Pharma Biotherapeutics USA Inc. The registry reports a start date of 2022-03-21, a primary completion date of 2022-11-01, a terminated status, and enrollment of 3.

The primary outcome is the number of participants with clinical remission from baseline to Day 56. The posted counts are 0 of 2.0 in the placebo group, no participants in the SPH3127 50 mg group, and 1 of 1.0 in the SPH3127 100 mg group. No analysis entries or comparative estimate are reported. The record also reports no serious adverse events among participants at risk in the placebo and SPH3127 100 mg groups; the 50 mg group had no participants at risk.

The registry does not report the reason for termination, the primary endpoint criteria, the analysis plan, or missing-data methods. The available results are descriptive and do not provide a precise treatment comparison.