A Phase II Study in Patients With Moderate to Severe Active Ulcerative Colitis.: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Phase II Study in Patients With Moderate to Severe Active Ulcerative Colitis. (NCT03675477), a randomized phase 2 study. The primary outcome is The Percentage of Subject Achieve Clinical Response at Week 8.

2. Clinical Question

3. Trial Design

This was a randomized, double-blind, parallel-group, placebo-controlled phase II study in moderate to severe active ulcerative colitis. The official title describes a four-arm dose-ranging study. Enrollment was 164, and the registry lists the study as completed. The sponsor was Reistone Biopharma Company Limited.

The first treatment phase lasted 8 weeks. Participants who completed it could enter a blinded 8-week extension. Those assigned to an SHR0302 regimen could continue that regimen; participants assigned to placebo were re-randomized to one of the three active regimens for the extension. The registry describes total participation, including extension and follow-up, as approximately 18 weeks. The primary outcome was assessed at Week 8, before the extension could affect that comparison.

The study began on 2019-04-13, and primary completion was recorded as 2020-11-30. Randomization and double masking are features intended to reduce systematic differences in treatment assignment and outcome assessment. The registry record does not report additional details on the randomization procedure, allocation concealment, or how masking was maintained. It also does not report the original sample-size justification or the trial’s planned statistical analysis.

The posted arm results list 41 participants for each of the four initial treatment groups. Extension results concern participants who entered that phase and have different at-risk counts across groups. Those extension data should not be treated as a continuation of the original placebo-controlled comparison, because the placebo group was re-randomized to active treatment.

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.

Treatment Phase

Extension Phase

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 The Percentage of Subject Achieve Clinical Response at Week 8, measured over Week 8.

The primary endpoint is the percentage of participants who achieved clinical response at Week 8. A response required a decrease from baseline of at least 2 points and at least 30% in the 9-point modified Mayo score, along with an improvement in rectal bleeding: a reduction in its subscore of at least 1 point, or an absolute rectal-bleeding subscore of 0 or 1.

The modified Mayo score excludes the Physician Global Assessment subscore and ranges from 0 to 9. Its components are stool frequency, rectal bleeding, and mucosal appearance at endoscopy. Thus, the endpoint combines a change in a clinical score with a condition on rectal bleeding rather than counting any single symptom change as a response.

As a binary outcome, each participant either meets the response definition or does not; the reported percentage describes the share meeting it in each group. In general, a prespecified response definition makes a trial outcome interpretable by stating what change qualifies before results are compared. Combining symptom and endoscopic components can represent more than one aspect of disease activity, but the endpoint remains a measure at the stated assessment time. It does not by itself describe how long a response lasts or establish what happened after Week 8.

7. Results Posted in the Registry

• SHR0302 8mg QD: 46.3% (19/41) versus 26.8% (11/41) on control; risk difference 19.5 percentage points (95% CI -0.9 to 39.9), risk ratio 1.73 (0.94–3.16); unadjusted two-proportion z-test p = 0.067.
• SHR0302 4mg BD: 46.3% (19/41) versus 26.8% (11/41) on control; risk difference 19.5 percentage points (95% CI -0.9 to 39.9), risk ratio 1.73 (0.94–3.16); unadjusted two-proportion z-test p = 0.067.
• SHR0302 4mg QD: 43.9% (18/41) versus 26.8% (11/41) on control; risk difference 17.1 percentage points (95% CI -3.3 to 37.4), risk ratio 1.64 (0.89–3.02); unadjusted two-proportion z-test p = 0.106.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

The Percentage of Subjects Achieve Clinical Remission

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • SHR0302 8mg QD: 22.0% (9/41) versus 4.9% (2/41) on control; risk difference 17.1 percentage points (95% CI 2.8 to 31.4), risk ratio 4.50 (1.03–19.57); unadjusted two-proportion z-test p = 0.023.
  • SHR0302 4mg BD: 24.4% (10/41) versus 4.9% (2/41) on control; risk difference 19.5 percentage points (95% CI 4.8 to 34.2), risk ratio 5.00 (1.17–21.43); unadjusted two-proportion z-test p = 0.012.
• Interpretation:
  • The 95% confidence interval excludes no difference, so the proportion with the event is statistically higher for SHR0302 8mg QD than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.
  • The 95% confidence interval excludes no difference, so the proportion with the event is statistically higher for SHR0302 4mg BD than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.

The Percentage of Subjects Achieve Clinical Remission at Week 8

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • SHR0302 8mg QD: 14.6% (6/41) versus 2.4% (1/41) on control; risk difference 12.2 percentage points (95% CI 0.4 to 24.0), risk ratio 6.00 (0.76–47.66); unadjusted two-proportion z-test p = 0.048.
  • SHR0302 4mg BD: 17.1% (7/41) versus 2.4% (1/41) on control; risk difference 14.6 percentage points (95% CI 2.2 to 27.1), risk ratio 7.00 (0.90–54.38); unadjusted two-proportion z-test p = 0.026.
• Interpretation:
  • The 95% confidence interval excludes no difference, so the proportion with the event is statistically higher for SHR0302 8mg QD than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.
  • The 95% confidence interval excludes no difference, so the proportion with the event is statistically higher for SHR0302 4mg BD than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.

The Percentage of Subjects Achieve Endoscopic Remission (Mucosal Healing) at Week 8

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • SHR0302 8mg QD: 26.8% (11/41) versus 14.6% (6/41) on control; risk difference 12.2 percentage points (95% CI -5.2 to 29.5), risk ratio 1.83 (0.75–4.49); unadjusted two-proportion z-test p = 0.173.
  • SHR0302 4mg BD: 29.3% (12/41) versus 14.6% (6/41) on control; risk difference 14.6 percentage points (95% CI -3.0 to 32.3), risk ratio 2.00 (0.83–4.82); unadjusted two-proportion z-test p = 0.109.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between SHR0302 8mg QD and the control. The interval gives the range of effects that remain compatible with the data.
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between SHR0302 4mg BD and the control. The interval gives the range of effects that remain compatible with the data.

Across the posted secondary outcomes, the active-treatment groups generally have higher reported outcome percentages than placebo. The pattern is not uniform in precision: the clinical remission comparisons that have posted estimates have intervals that exclude zero, whereas the endoscopic remission intervals include zero. This provides a broadly similar direction for some outcomes, but not the same degree of statistical separation across measures.

These outcomes complement the primary clinical-response measure rather than replacing it. Clinical remission is a more restrictive clinical outcome than response, while endoscopic remission concerns mucosal appearance. In general, agreement across distinct outcomes can add context, but outcomes do not become independent confirmation merely because each is reported. The secondary endpoints also arise from the same trial groups and are subject to shared design and analysis limitations.

The registry record lists three secondary outcomes, but does not report sponsor analyses for them. The posted comparisons are unadjusted recalculations, and no multiplicity plan is reported. When several outcomes and dose comparisons are examined, the chance of apparently notable findings arising by chance can increase. Consequently, unadjusted secondary results carry less weight than results from a prespecified analysis that accounts for the family of comparisons. They are best treated as exploratory context for the primary result, not as definitive evidence or as a basis for choosing among doses.

The posted secondary summaries also do not provide the analysis population or missing-data handling. Differences in outcome definitions and the available estimates mean that the secondary results should not be combined into a single overall claim. Their main statistical contribution is to show whether the broad pattern is consistent across types of outcome, while leaving considerable uncertainty about interpretation.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• The Percentage of Subjects Achieve Clinical Remission
• The Percentage of Subjects Achieve Clinical Remission at Week 8
• The Percentage of Subjects Achieve Endoscopic Remission (Mucosal Healing) at Week 8

9. Statistical Interpretation

10. Statistical Methods Explained

What does a binary outcome represent?

In general, a binary outcome classifies each participant into one of two categories, such as meeting or not meeting a response definition. The reported percentage is the proportion in the group who meet that definition. For the primary endpoint, the event is clinical response by Week 8. The registry reports the group percentages and the participant counts used in the posted comparisons. This presentation is straightforward to interpret, but it compresses a range of clinical experiences into a yes-or-no classification: people just above and well above the response threshold are counted alike.

What is a risk difference?

In general, a risk difference subtracts the event proportion in the comparator group from the event proportion in the treatment group. A value of zero represents equal proportions; a positive value means the event proportion is higher in the treatment group under that comparison’s direction. The posted primary risk differences are 0.195 for 8mg QD, 0.195 for 4mg BD, and 0.171 for 4mg QD. They are simple, unadjusted comparisons of the posted summaries. They are not adjusted for baseline factors or other covariates.

A risk difference describes absolute separation between proportions. Other measures, such as a risk ratio or odds ratio, describe relative contrasts and can give a different impression of the same data. A measure should be read alongside the underlying group percentages and its uncertainty, rather than as a standalone verdict. The posted primary comparison is framed as a risk difference.

How should an interval and p-value be read?

In general, an interval estimate gives a range of values reasonably compatible with the observed data under the method used. A wider interval signals less precision than a narrower one. For a risk difference, an interval spanning zero includes equal event proportions among the values compatible with the data. The posted primary intervals span zero for each dose comparison.

A p-value describes how unusual the data, or data more extreme, would be under a specified null model and its assumptions. It is not the probability that the null hypothesis is true, and it does not measure the size or clinical importance of an effect. The posted p-values are unadjusted recalculations. The registry record does not report the sponsor’s analysis method, a confidence level for the posted intervals, or a multiplicity adjustment. Therefore, the numerical summaries should be interpreted as descriptive unadjusted comparisons, not as a substitute for a reported trial analysis.

Why does the analysis population matter?

In general, an analysis needs to specify which randomized participants are included and how outcomes unavailable at the planned assessment are handled. Different choices can affect group estimates, particularly when missingness is related to outcome or treatment. The registry record does not report the analysis population or missing-data method for the primary result. The displayed group counts do not, by themselves, establish whether the results follow an intention-to-treat strategy, a per-protocol strategy, or another approach.

Randomization can support a fair comparison when the assigned groups are analyzed in a way that preserves the randomized assignment and when outcome availability is handled appropriately. It does not remove uncertainty from limited observations, guarantee complete follow-up, or answer whether missing outcomes differ systematically between groups. No such details are reported in the registry record.

What does double masking contribute?

In general, masking is intended to limit the influence of treatment knowledge on care, participant reports, and outcome assessment. A placebo comparator can help keep expectations and study procedures similar during the controlled phase. The registry identifies the design as double-masked, but does not report which roles were masked or whether masking was assessed. The extension changes the comparison structure: participants originally assigned to placebo who entered the extension were re-randomized to active regimens. That phase can describe outcomes after active treatment assignment, but it does not retain the original placebo comparison.

The registry record reports a phase II dose-ranging design and the endpoint definition, but detailed methods for the statistical analysis are not reported in the registry record. General explanations of risk differences, intervals, and p-values clarify what the posted summaries mean; they do not fill in the unreported trial analysis plan.

11. Confidence Intervals and Precision

The posted primary interval estimates are broad. For SHR0302 8mg QD versus placebo, the risk difference is 0.195, with interval limits -0.009 and 0.399. The same estimate and limits are posted for 4mg BD versus placebo. For 4mg QD versus placebo, the estimate is 0.171, with limits -0.033 and 0.374. All three intervals include zero.

In general, an interval spanning zero for a risk difference means that equal proportions remain compatible with the data under the interval method, while positive values are also compatible. Here, the ranges additionally include negative values. The point estimates are positive, but the lower limits do not exclude no difference or a difference in the opposite direction. The intervals therefore do not pin down the direction or magnitude precisely. Their upper limits also allow larger positive differences than the point estimates.

The registry record does not report the confidence level or interval-construction method for these posted intervals. The interval limits should be read as uncertainty summaries, not as a guarantee that the true value lies within them. The lack of a reported sponsor analysis also limits interpretation of how these intervals relate to a prespecified statistical plan. The unadjusted p-values—0.067 for 8mg QD, 0.067 for 4mg BD, and 0.106 for 4mg QD—are consistent with the uncertainty apparent in the intervals, but they do not remove it.

Precision is distinct from the observed percentage. A point estimate can appear separated from placebo while remaining imprecise, particularly when the interval covers a broad range. The group percentages and interval limits should therefore be considered together.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are participants per arm, not totals across arms and not total events. They are illustrations based on the observed effect, not the original trial’s sample-size plan. The listed assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The effect to detect is the stated fraction of the observed effect. The original design assumptions and sample-size justification are not reported in the registry record.

For the 8mg QD and 4mg BD primary comparisons, planning for 100% of the observed effect gives 95 participants per arm for 80% power and 126 for 90% power. Planning for 75% of the observed effect gives 164 per arm for 80% power and 219 for 90% power. Planning for 50% of the observed effect gives 356 per arm for 80% power and 477 for 90% power. For the 4mg QD comparison, the corresponding figures are 122 and 163 for 100% of the observed effect, 212 and 283 for 75%, and 461 and 616 for 50%.

In plain terms, the 80% column illustrates the per-arm size associated with an 80% chance of detecting the specified effect under the stated assumptions; the 90% column uses a higher target chance and therefore requires more participants in these illustrations. Smaller effects require more participants to distinguish them from random variation under the same assumptions. These figures are planning illustrations, not observed power.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, p₀ and p₁ are the control and treatment proportions, and p̄ is their average.

Worked calculation for this trial

1. Control proportion p₀ = 0.268; treatment proportion p₁ = 0.463; average p̄ = 0.366.
2. First term: z_1−α/2 × √[2p̄(1−p̄)] = 1.96 × 0.6812 = 1.3351.
3. Second term: z_1−β × √[p₀(1−p₀) + p₁(1−p₁)] = 0.842 × 0.6671 = 0.5614.
4. n = (1.3351 + 0.5614)² / (0.1951)² = 94.5, rounded up to 95 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
p0 <- 0.268292682926829                          # control proportion
p1 <- 0.463414634146341                          # treatment proportion
n_per_arm <- function(power, frac = 1) {
  q1 <- p0 + (p1 - p0) * frac               # smaller effect = fraction of the observed difference
  pbar <- (p0 + q1) / 2
  z_b <- qnorm(power)
  ceiling(round((z_a * sqrt(2 * pbar * (1 - pbar)) + z_b * sqrt(p0 * (1 - p0) + q1 * (1 - q1)))^2 / (q1 - p0)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (unrounded n per arm)
cat("power.prop.test:", round(power.prop.test(p1 = p0, p2 = p1, power = 0.80)$n, 1), "\n")

# Simulation: how often does the z-test reject at the 80%-power sample size?
set.seed(3675477)
n    <- n_per_arm(0.80)
reps <- 20000
x0 <- rbinom(reps, n, p0); x1 <- rbinom(reps, n, p1)
ph <- (x0 + x1) / (2 * n)
z  <- ((x1 - x0) / n) / sqrt(ph * (1 - ph) * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(z) > z_a, na.rm = TRUE), reps))

Output from running the code

100% of observed effect: n per arm = 95 (80% power), 126 (90% power)
 75% of observed effect: n per arm = 164 (80% power), 219 (90% power)
 50% of observed effect: n per arm = 356 (80% power), 477 (90% power)
power.prop.test: 94.5 
Simulated power at n = 95 per arm: 0.809 (20000 replicates)

The simulation draws many pairs of event counts at the planned sample size and counts how often the two-proportion z-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Multiplicity, Missing Data and Analysis Populations

The trial compares three SHR0302 regimens with placebo for the primary outcome and reports three secondary outcomes. Multiple comparisons matter because, in general, evaluating a larger set of hypotheses increases the chance that at least one result appears unusual under a null model. A multiplicity strategy can define how such a family of tests is controlled or how results are prioritized. The registry record does not report a multiplicity adjustment, a testing hierarchy, or another plan for interpreting the several comparisons. The posted p-values are unadjusted and should be understood in that context.

The registry record lists no reported analyses for the primary or secondary outcomes. It does not report the sponsor’s model, analysis population, covariate adjustments, missing-data procedure, or whether all randomized participants were included in each endpoint analysis. The arm counts associated with the posted results do not resolve those questions. In general, missing outcomes can affect estimates if their occurrence is related to assignment or response, and the direction of any such effect cannot be inferred without information about the missing data and analysis approach.

The primary endpoint is defined at Week 8, but detailed rules for intercurrent events, withdrawals, or other unavailable measurements are not reported in the registry record. The record also does not report whether interim analyses were planned or performed. The original sample-size justification and trial-specific design assumptions are not reported either. The separate planning illustration uses stated assumptions, but it does not establish what assumptions governed the trial’s original design.

These omissions do not show that a particular analysis choice was inappropriate; they mean its details cannot be assessed from the registry record. They also limit how confidently the posted unadjusted comparisons can be interpreted as the study’s prespecified inferential results.

15. 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: colitis ulcerative (10.0%), anal abscess (2.4%), cytomegalovirus infection (2.4%), liver abscess (2.4%), synovitis (2.4%).

The registry reports serious adverse events and other adverse events separately for the treatment and extension phases. During the treatment phase, serious adverse events were reported for 2 participants in the 8mg QD group, 0 in the 4mg BD group, 2 in the 4mg QD group, and 2 in the placebo group. The registry reports 0 deaths in each treatment-phase group.

For extension groups, the reported serious-event counts are 1 for continued 8mg QD, 0 for continued 4mg BD, 0 for continued 4mg QD, 1 for placebo followed by 8mg QD, 1 for placebo followed by 4mg BD, and 0 for placebo followed by 4mg QD. The registry reports 0 deaths in each extension group. These groups have different at-risk counts, and the extension is not the original placebo-controlled comparison.

In general, adverse-event counts describe events reported under the registry’s definitions; they do not by themselves establish that treatment caused an event or provide a comparative safety analysis. The record does not report inferential comparisons of serious adverse events. It lists “Colitis ulcerative” at 10.0% among the top serious-event terms, with Anal abscess, Cytomegalovirus infection, Liver abscess, and Synovitis each listed at 2.4%. The definitions and coding details should be considered when interpreting these summaries.

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

The posted results describe clinical response at Week 8 in a randomized, double-blind phase II study. The observed response percentages are higher in each SHR0302 group than in placebo. The unadjusted risk-difference estimates are positive, but their intervals include zero and extend across a range of possible differences. The data therefore describe a numerical pattern while leaving substantial uncertainty about the size and direction of the underlying difference.

This is not, by itself, a definitive answer about treatment efficacy. The registry record does not report the sponsor’s outcome analyses, analysis populations, missing-data methods, or multiplicity plan. Those details matter when interpreting estimates and p-values, particularly in a dose-ranging study with multiple outcomes. The posted comparisons are unadjusted recalculations from the summary statistics, not a replacement for a complete prespecified analysis.

The endpoint captures a defined response threshold at one assessment time. It does not describe the full distribution of score changes, establish how long a response lasts, or determine outcomes beyond the recorded endpoint. Although the study included an extension, placebo participants who entered it were re-randomized to active treatment, so the extension does not preserve the initial placebo comparison.

The secondary outcomes offer additional descriptive context, but unadjusted analyses across several endpoints cannot independently settle the primary question. Safety counts are also descriptive and do not establish causation or comparative risk. Overall, the registry record supports a careful reading of the observed short-term group results and their uncertainty; it does not justify a medical recommendation or a broad conclusion beyond these data.

17. Why This Trial Matters Statistically

This trial is statistically informative as a dose-ranging, placebo-controlled study with a clearly defined binary primary endpoint and three active regimens. The reported Week 8 percentages allow direct description of the outcome in each initial group, while the risk differences express the absolute separation from placebo. The design’s randomized and double-blind features are relevant strengths for interpreting a controlled comparison.

The result also illustrates why point estimates alone are insufficient. The primary estimates are positive, but the posted intervals include zero and cover appreciable uncertainty. In general, that combination means a numerical difference has been observed without tightly determining its size. Reporting the interval alongside the estimate makes that uncertainty visible rather than reducing interpretation to a single p-value.

The record is also a useful example of the distinction between posted results and a reported analysis. It contains arm summaries and recalculated unadjusted comparisons, but no sponsor analysis is listed. Without the analysis population, missing-data approach, and multiplicity strategy, readers cannot fully evaluate how the findings relate to the trial’s planned inferential framework.

Finally, the extension design matters for comparisons over time. Re-randomization of the initial placebo group to active treatment means the later phase answers a different descriptive question from the original placebo-controlled phase. Keeping the Week 8 primary comparison distinct from extension observations is important for a valid statistical interpretation.

18. 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.
• Participant counts were reconstructed from posted percentages and arm sizes, so small rounding differences are possible.

Several trial-specific limitations constrain interpretation. The primary outcome is assessed at Week 8, and the registry’s response definition classifies participants using a threshold. This offers a clear event definition but does not show the full range of score changes or persistence of response beyond that assessment. The extension lasts 8 weeks, but its structure changes the comparison: placebo participants who enter it are re-randomized to an active regimen. Longer-term extension outcomes therefore cannot be read as a continued randomized placebo comparison.

The initial arms each have 41 participants in the posted results. The primary intervals are broad and include zero, reflecting uncertainty in the estimated differences. There are three active-dose comparisons and multiple secondary outcomes, but a multiplicity strategy is not reported. The registry record lists no sponsor analyses, and the analysis population and handling of missing outcomes are not reported. These omissions make it difficult to assess whether the posted comparisons match the planned analysis or how unavailable outcomes may have affected them.

The record describes the study as multicenter, randomized, double-blind, and phase II, but additional operational details are not reported in the registry record. The original sample-size rationale and the trial’s own statistical design assumptions are also not reported. The safety summaries provide counts by phase and group but no inferential comparison, so they should not be interpreted as establishing relative safety differences.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: A Phase II Study in Patients With Moderate to Severe Active Ulcerative Colitis. — NCT03675477.
• Registry record last updated 2023-06-02; status last verified 2022-12.
• Results first posted 2022-11-22.
• 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.

22. Record Summary

NCT03675477 is a completed randomized, double-blind, parallel-group phase II study of SHR0302 in moderate to severe active ulcerative colitis. The initial four groups were SHR0302 8mg QD, SHR0302 4mg BD, SHR0302 4mg QD, and placebo. The primary endpoint was clinical response at Week 8. The registry reports group percentages and posted unadjusted comparisons; no sponsor analyses are listed.

The primary point estimates favor the active groups numerically, but each posted interval includes zero. The registry also reports secondary outcome percentages and adverse-event counts. Multiplicity handling, analysis populations, missing-data methods, and the original sample-size justification are not reported in the registry record. Extension results follow a change in treatment assignment for participants originally allocated to placebo and should be distinguished from the initial controlled comparison.