An Open-Label, Phase 2a Study to Evaluate Safety and Efficacy of GR-MD-02 for Treatment of Psoriasis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for An Open-Label, Phase 2a Study to Evaluate Safety and Efficacy of GR-MD-02 for Treatment of Psoriasis (NCT02407041), a na phase 2 study. The primary outcome is Number of Participants With PASI-75, or a 75% Improvement From Baseline in PASI Score.

2. Clinical Question

This setup frames a descriptive question about the number of participants meeting the specified improvement threshold during the study. It does not set up a direct estimate of how GR-MD-02 compares with another treatment or with a concurrent control group. The registry lists psoriasis as the condition and identifies the study as phase 2a. Its brief summary describes participants with moderate to severe plaque psoriasis.

The primary question is distinct from the safety summaries. The primary endpoint concerns a threshold change in PASI score, while the safety record separately reports serious events, other events and deaths. The registry does not report a primary-endpoint count or analysis, so the clinical question can be described, but its answer cannot be determined from the posted results.

3. Trial Design

This was a phase 2a, single-center, open-label clinical trial of GR-MD-02 for moderate to severe plaque psoriasis. The registry lists the model as single-group, allocation as not applicable and masking as none. The study is marked completed. Enrollment is recorded as 5.

The intervention listing names GR-MD-02 as a drug and describes it as the active arm. The safety summary labels its group “GR-MD-02 8 mg/kg.” These details describe the intervention group, not a comparison between randomized groups. The registry does not list a placebo group or another active comparator.

The study dates recorded are a start in 2015-09 and primary completion in 2018-03. These dates describe the registry record’s timeline; they do not establish when each participant began or completed follow-up. The protocol details, recruitment process and participant-level follow-up are not reported in the registry record.

The primary outcome is a count of participants meeting PASI-75, defined as a 75% improvement from baseline in PASI score. The endpoint description identifies baseline as day 1, prior to first infusion, and says assessment was at the 30 day follow up visit. The stated timeframe is 6 months. Since the record does not reconcile these time references, the intended timing cannot be inferred beyond what is explicitly recorded.

Because this is a single-group, open-label study, it can describe what was recorded among participants receiving the intervention, but the design does not provide a concurrent control for separating treatment-associated change from other explanations. The registry does not report its statistical plan, sample-size justification or analysis populations.

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

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 PASI-75, or a 75% Improvement From Baseline in PASI Score, measured over 6 months.

The primary endpoint is the number of participants with PASI-75, defined in the registry as a 75% improvement from baseline in PASI score. Baseline is described as day 1, before the first infusion. The endpoint is recorded as a participant count, so it asks how many participants crossed a prespecified improvement threshold rather than how much the group’s average score changed.

A threshold endpoint can make a clinical response criterion easier to interpret: each participant either meets the stated threshold or does not. That is a general explanation of this type of outcome, not a claim about the result in this study. The threshold also leaves out the degree of improvement among participants who do not reach it, so it does not capture every possible change in PASI score.

The registry gives the endpoint timeframe as 6 months, while the description says the outcome is assessed at the 30 day follow up visit. The record does not explain the relationship between those timing statements. It reports no endpoint count, so the number meeting the threshold and the precision of any estimated response rate are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is binary at the participant level: each participant either meets the PASI-75 criterion or does not. The registry expresses the primary measure as a count of participants, not as a group mean. For a binary endpoint, an analysis might report the count meeting the criterion alongside the number assessed, and could also estimate a proportion and its uncertainty. Those are general statistical options; the registry record does not report such an analysis or result for this study.

What does a confidence interval express?

In general, a confidence interval describes the statistical uncertainty around an estimate under specified assumptions. For a binary outcome, an interval around a response proportion can indicate how precisely the observed data estimate the underlying response frequency in a population. An interval is not a guarantee that the true value lies within its limits, and its interpretation depends on the study design, the analysis method and the data included. No confidence interval for the primary endpoint is reported in the registry record.

What can a single-group study estimate?

A single-group study can describe outcomes observed among participants in that group. It does not make a randomized comparison with a control group. In general, a comparison group can help distinguish an intervention-associated outcome from changes that might occur over time, differences in participant characteristics or other influences. Without such a group, the results cannot directly estimate a between-group treatment contrast. This is a design limitation, not evidence that the observed outcomes are caused by any particular factor.

The record identifies the study as open-label and lists masking as none. In general, knowledge of treatment assignment can influence expectations, reporting or assessments, depending on how outcomes are collected. The record does not report assessor masking, standardized scoring procedures or safeguards against such influence. Those procedures therefore cannot be assumed.

What analysis was recorded?

The registry lists no analyses for the outcome. The statistical model, any covariate adjustment, the method for handling missing observations and any inferential test are not reported in the registry record. It is not possible to determine whether the intended analysis was limited to a descriptive count or included additional methods. A count endpoint does not, by itself, reveal which statistical procedure was used.

The record includes planning assumptions for sample-size illustrations: a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation and an effect to detect expressed as a fraction of the observed effect. It also distinguishes total events for time-to-event illustrations from participants per arm for other illustrations. These are planning assumptions, not evidence that this single-group trial used a two-arm design. The sample-size illustrations are not the trial’s own design justification, and they are not observed power. The record does not report a trial-specific sample-size justification.

In general, planning calculations depend on assumptions about the outcome, effect size, variability, allocation and analysis. If an illustration is based on an observed effect, it describes a hypothetical future design under stated assumptions; it does not establish the probability that the completed study detected an effect. Here, the primary endpoint result itself is not reported, so the registry entry does not support interpretation of an observed PASI-75 effect from these planning assumptions.

The endpoint timing also affects analysis. The registry gives a 6 months timeframe and describes evaluation at the 30 day follow up visit, but does not reconcile these statements. The primary assessment time therefore cannot be clarified from the record. In general, prespecifying the assessment time is important because outcomes may vary over follow-up, and selecting among assessment times after seeing data can complicate interpretation. The registry does not report whether more than one assessment was analyzed.

Finally, no analysis population is specified. In general, results may differ depending on whether analyses include everyone enrolled, only participants who received the intervention, or only those with an observed endpoint. These populations answer different questions and can be affected differently by missing data. The registry does not report how many participants had an endpoint assessment or how missing observations were treated. Consequently, no analysis denominator, response proportion or uncertainty estimate can be reconstructed from the posted primary outcome information.

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("GR-MD-02 8 mg/kg")
n      <- c(5)
events <- c(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  GR-MD-02 8 mg/kg  5  1  20.0  0.5  71.6  3.6  62.4  4.3  64.1

11. Multiplicity, Missing Data and Analysis Populations

The registry lists one primary outcome measure and no analyses. It does not report a statistical analysis plan, any secondary outcomes, planned comparisons or whether additional analyses were conducted outside the registry. The approach to multiplicity—how to interpret false-positive risk when several outcomes or tests are examined—is therefore not reported in the registry record. With no posted primary-endpoint analysis, there is no reported test result to interpret in that context.

Missing-data handling is also not described. The primary endpoint is a count of participants meeting a threshold, but the record does not say how participants without an assessment were classified or whether any imputation method was used. It does not report how many participants had a PASI assessment at the stated endpoint time. The mismatch between the 6 months timeframe and the 30 day follow up visit description further limits interpretation of when outcome data were expected.

The analysis population is not specified. The record does not say whether the primary analysis was intended to include all enrolled participants, participants who received the intervention, or only participants with endpoint data. These are distinct analysis choices, and the registry record does not identify which applies. As a result, the posted entry does not support an assessment of attrition, missingness or how those issues may have affected the primary endpoint.

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 safety summary records the GR-MD-02 8 mg/kg group, with 5 at risk for serious events and 0 serious events. It also records 5 at risk for other events and 0 other events, with 0 deaths. These are counts in the registry record, not estimates of comparative risk: the study has no listed control group.

A count of 0 means no events of that category are recorded for the stated group in this summary. It does not establish that the risk is absent. As a general statistical point, a study with limited observation can fail to capture uncommon events even when some underlying risk exists. The record does not report event definitions, follow-up duration for safety, severity details beyond the serious-event category, or how completely adverse events were collected.

The safety counts should therefore be read descriptively. They do not establish a safety comparison with another treatment, and they do not determine the primary PASI-75 result. Further safety interpretation is limited because the registry record does not provide additional event details.

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

The registry establishes that a completed, single-center, open-label phase 2a study of GR-MD-02 was recorded for moderate to severe plaque psoriasis, with enrollment of 5. It specifies a primary endpoint based on whether participants achieved a 75% improvement from baseline in PASI score. It also reports safety counts of 0 serious events, 0 other events and 0 deaths in the listed group.

It does not report how many participants met PASI-75. The absence of that result is not evidence that the endpoint was or was not achieved. There is no reported response estimate, confidence interval or primary-endpoint analysis to indicate either the size or precision of an outcome. The relationship between the 6 months timeframe and the 30 day follow up visit description is also unresolved in the record.

Because the study is single-group and open-label, it does not provide a randomized comparison with another treatment or a control. As a general matter, outcomes in a single group cannot isolate an intervention’s effect from other explanations for change. The recorded safety counts are likewise descriptive and do not establish comparative safety or the absence of risk.

The study can be characterized by its design, endpoint definition, enrollment and posted safety counts. The information recorded does not support a conclusion about the primary efficacy outcome, a comparative treatment effect or the precision of any such effect. Those conclusions would require outcome and analysis information that is not reported in the registry record.

14. Why This Trial Matters Statistically

This trial is statistically notable because its primary question is framed as a participant count for a threshold-based response, while the registry does not report the count or an analysis. A clearly defined endpoint does not by itself provide evidence about the endpoint’s observed value. For readers, separating the endpoint definition from its result is essential: the record tells what was intended to be measured, but not how many participants met the criterion.

The single-group, open-label design also sets boundaries on what the study can establish. Descriptive outcomes can characterize participants in the intervention group, but without a concurrent comparator there is no randomized estimate of a treatment contrast. That distinction matters even when a study’s stated goal includes evaluating efficacy. The label or aim does not replace the evidence needed to estimate an effect.

The safety summary illustrates another key statistical distinction: observed counts and underlying risk are not the same. The registry reports zero events in the recorded categories, but does not provide event detail or comparative data. The counts are useful as a description of what the record captures, not as proof that an event cannot occur.

Finally, the unresolved timing statements and absent analysis details make it difficult to understand how the primary outcome was to be evaluated. The registry gives a 6 months timeframe and a 30 day follow up visit in the endpoint description, but does not explain their relation. This case underscores why endpoint timing, analysis populations, missing-data methods and complete outcome reporting are important for interpreting a clinical study.

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 limitations are specific to the information recorded for this study. The registry describes a single-center, open-label, single-group design, with no randomized comparator. The results therefore cannot be read as a controlled comparison, and the record does not describe procedures that might reduce bias in outcome assessment.

Most importantly, the primary endpoint is defined but its result is absent. The number of participants with PASI-75, the number assessed and any measure of uncertainty are not reported in the registry record. The analysis population and handling of participants without endpoint data are also not reported. This prevents assessment of the primary outcome and of the completeness of its analysis.

The endpoint timing is unclear: the registry states a timeframe of 6 months but describes assessment at the 30 day follow up visit. No explanation reconciles these statements. Protocol details, eligibility criteria beyond the brief population description, dosing and administration information, and the trial-specific sample-size justification are not reported in the registry record.

The safety summary provides counts but no further event descriptions or details about the safety observation period. The registry also lists no analyses and does not report a statistical analysis plan, multiplicity approach or missing-data methods. These gaps constrain interpretation of both efficacy and safety. They do not establish that unreported analyses or procedures were absent from the full study; they mean those details are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: An Open-Label, Phase 2a Study to Evaluate Safety and Efficacy of GR-MD-02 for Treatment of Psoriasis — NCT02407041.
• Registry record last updated 2020-09-07; status last verified 2020-09.
• Results first posted 2020-09-07.
• 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

The registry describes a completed phase 2a, single-center, open-label, single-group study of GR-MD-02 for moderate to severe plaque psoriasis. Enrollment is recorded as 5. The primary endpoint is the number of participants with PASI-75, defined as a 75% improvement from baseline in PASI score. The timeframe is listed as 6 months, while the endpoint description refers to assessment at the 30 day follow up visit.

No primary-endpoint result or analysis is reported. The safety summary lists 0 serious events, 0 other events and 0 deaths in the GR-MD-02 8 mg/kg group. The study has no listed control group, and the registry does not report the analysis population, missing-data methods or statistical analysis plan. The posted information supports a description of the design and recorded safety counts, but not an estimate of the primary outcome or a comparative treatment effect.