This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for An Open-label, Phase 2 Study to Assess the Efficacy and Safety of Nemolizumab in Subjects With Systemic Sclerosis (NCT05214794), a na phase 2 study. The primary outcome is Change From Baseline in Modified Rodnan Total Skin Thickness Score (Total mRSS) at Week 24.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05214794 |
| Phase | Phase 2 |
| Condition | Systemic Sclerosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Maruho Co., Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
People with systemic sclerosis enrolled in this Phase 2 study. The registry record describes the full analysis set as including all enrolled participants.
Intervention
Nemolizumab, listed as the study drug. The registry record does not describe a dose or administration schedule.
Comparator
None. This is a single-group study, with no control arm or like-for-like comparison group.
Primary question
What change from baseline in the Modified Rodnan Total Skin Thickness Score was recorded at Week 24?
The registry describes an open-label study in which participants received nemolizumab and the primary outcome was change from baseline in total mRSS at Week 24. The mRSS is a clinician-assessed measure of skin thickening across specified body sites. Because the study has one group and no masking, the question is descriptive: it concerns the observed before-and-after score in the enrolled group, rather than a contrast between randomized treatments.
The record also names secondary measures covering total mRSS at Week 52, disability, lung function, and physician assessment at specified follow-up times. It states that the study evaluated efficacy, safety, and pharmacokinetics. However, numerical results for the primary endpoint and the listed secondary measures are not reported in the registry record. The record therefore identifies what the study set out to assess, but does not supply an effect estimate for the primary question.
The listed enrollment is 6. That small enrolled group makes clear that the study describes a limited set of participants; the registry record does not report enough outcome detail to quantify a change in skin score or its uncertainty.
3. Trial Design
This was a completed, open-label, Phase 2 study of nemolizumab in people with systemic sclerosis. The registry identifies the allocation as not applicable, the masking as none, and the model as single group. There is no randomized control arm in the record, so the design does not support a direct treatment-versus-control comparison.
The listed enrollment is 6. The study started on 2022-04-20, reached primary completion on 2024-06-04, and is recorded as completed. These dates describe the registry timeline; they do not establish how long each participant was followed or whether each participant completed every assessment. Those details are not reported in the registry record.
The intervention is listed as nemolizumab, a drug. The arm description contains no further details. Dose, route, treatment frequency, treatment duration, eligibility criteria beyond systemic sclerosis, and the study setting are not reported in the registry record. No protocol details should be inferred from the study title or the fact that it was open-label.
The primary endpoint is change from baseline in total mRSS at Week 24. The registry specifies a median and full range as the summary format, and gives baseline and Week 24 as the timeframe. It also lists 12 outcome measures overall, including secondary outcomes for total mRSS at Week 52; HAQ-DI at Week 24 and Week 52; percent FVC at Week 24 and Week 52; percent DLco at Week 24 and Week 52; and physician global assessment at Week 24. Numerical results for these measures are not reported in the registry record.
The design is useful for describing what was measured in the treated group, but it cannot separate change associated with treatment from change due to other influences, such as the natural course of disease, concurrent care, or measurement variability. The registry record does not report a protocol rationale, sample-size justification, interim analyses, or further details of the statistical plan.
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
| Stage | Nemolizumab |
|---|---|
| STARTED | 6 |
| COMPLETED | 6 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: FAS population included all participants who were enrolled.
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: FAS population included all participants who were enrolled.
| Characteristic | Unit | Nemolizumab |
|---|---|---|
| Participants analysed | 6 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 1 |
| Age, Continuous | year | 53.5 (50 to 66) |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 6 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 6 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: Japan | participants | 6 |
| Classification of Primary Disease: Diffuse Cutaneous SSc | Participants | 4 |
| Classification of Primary Disease: Limited Cutaneous SSc | Participants | 2 |
| Modified Rodnan Total Skin Thickness Score (Total mRSS) | scores on scale | 16 (12 to 22) |
| percent forced vital capacity (%FVC) | percentage of actual to predicted FCV | 99.7 (82.7 to 109.0) |
| percent diffusing capacity of lung for carbon monoxide (% DLco) | percentage of actual to predicted DLco | 101.75 (83.2 to 109.2) |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Modified Rodnan Total Skin Thickness Score (Total mRSS) at Week 24, measured over Baseline and week 24.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | scores on scale |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is change from baseline in the Modified Rodnan Total Skin Thickness Score, or total mRSS, at Week 24. The mRSS is a clinician-assessed score of skin thickening. The investigator assessed 17 sites using a 4-point scale: 0 for normal skin, 1 for mild thickness, 2 for moderate thickness, and 3 for severe thickness. The total score is the sum of the site scores.
This measure is used to represent the extent and degree of skin thickening across several body areas rather than relying on a single-site assessment. In general, combining assessments across sites can give a structured way to describe a clinical feature that varies across the body. The score remains an assessment, however, and its interpretation depends on how consistently assessors apply the scale. The registry record does not report information about assessor training or reliability.
The endpoint is a change from baseline, so interpretation would ordinarily require the baseline and follow-up score or a calculated change summary. The record specifies median and full range as the summary format. It does not report the numerical median change, range, individual scores, or a confidence interval. Thus, the endpoint definition is available, but the magnitude and precision of the observed change cannot be evaluated from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record provides a clear description of the primary outcome but not its numerical result. It identifies change from baseline in total mRSS at Week 24 as the primary endpoint, specifies the scoring approach, and lists median and full range as the summary format. It does not report the observed median change or the range. As a result, the direction, size, and spread of the primary endpoint change cannot be described from the registry record.
The study is single group and open label. Consequently, any reported before-and-after change would describe what happened among participants in the study, not what would have happened under a comparator treatment or no study drug. In general, a control group helps distinguish a treatment-associated difference from changes that may occur over time for other reasons. This design does not provide that contrast. Open-label assessment also means that masking is not part of the recorded design; the registry record does not provide information that would allow the influence of awareness on assessments to be evaluated.
The enrollment is 6, and the full analysis set is described as including all participants enrolled. Even if a numerical result were available, a result from a group of this size would need to be read with attention to its uncertainty and to the observed values across participants. The record does not report a confidence interval, participant-level outcome values, or another uncertainty measure for the primary endpoint. A median and full range can describe the center and spread of observations, but they do not by themselves establish how precisely a broader population effect has been estimated.
The registry names secondary outcomes related to skin score, disability, lung function, and physician assessment. Their results are not reported in the registry record. Therefore, no cross-outcome pattern can be evaluated, and the primary endpoint cannot be contextualized against those measures using numerical results.
The safety record reports 0 participants with serious adverse events among 6 at risk, 6 with other adverse events among 6 at risk, and 0 deaths among 6 at risk. These counts describe the recorded categories in this study group. They do not, by themselves, establish how frequently such events might occur in a wider population or whether an event was caused by the study drug. The record does not list serious adverse-event terms.
Any planning illustrations based on an observed effect should not be confused with evidence about the effect or with observed power. General sample-size calculations use assumptions about the effect to detect and the analysis design. The registry describes an illustration using a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and fractions of the observed effect. This trial itself is single group, and the numerical observed primary effect is not reported in the registry record. The illustration therefore cannot substitute for a reported result or an account of the study's design rationale.
9. Statistical Methods Explained
The registry record identifies the primary endpoint, the summary format, and the analysis population, but gives little information about the statistical analysis. It states that the primary endpoint is change from baseline in total mRSS at Week 24, summarized by median and full range. It defines the full analysis set as all participants enrolled. It does not report a numerical endpoint result, a statistical test, a confidence interval, or an analysis model.
What does a median and full range describe?
In general, the median is the middle observation when values are ordered. It is a measure of location that is less influenced by an unusually high or low value than the arithmetic mean. The full range describes the lowest and highest observed values. Together, these summaries can show a typical observed value and the span of the observations, but they do not show the full distribution or the precision of an estimate. With a small group, individual observations can have substantial influence on the reported summary and range.
For this trial, the record names the median and full range as the dispersion format but does not provide their values. It is therefore not possible to characterize the observed distribution of change, determine whether observations were similar or widely spread, or compare the result with any other group. No calculation of the median or range can be reconstructed from the registry record.
What does a change-from-baseline outcome mean?
In general, a change score compares a follow-up measurement with a baseline measurement for the same participant. It can describe how measurements shifted over time within the observed group. It is not inherently a treatment effect: a treatment effect is usually a contrast between outcomes under different treatment conditions, commonly estimated by comparing randomized groups. In a single-group study, the change score has no concurrent control contrast.
The registry specifies baseline and Week 24 for the primary endpoint. It does not report the baseline score distribution, the follow-up score distribution, individual paired measurements, or the resulting change values. The endpoint name alone cannot determine whether scores increased, decreased, or remained stable.
What is the analysis population?
The record says the full analysis set included all participants who were enrolled. This identifies the intended group for the primary outcome according to the registry entry. It does not say how many participants had an evaluable Week 24 assessment, whether missing assessments occurred, or what method was used to handle any missing data. Those details are not reported in the registry record.
In general, the analysis population and missing-data approach matter because results can depend on which participants contribute outcome measurements and how unavailable measurements are handled. Without the number of observed assessments and the analysis method, a reader cannot assess whether the reported endpoint—if it were available—represents all enrolled participants or only those with a recorded follow-up value.
What can significance tests and confidence intervals add?
In general, a significance test evaluates how compatible data are with a specified null hypothesis under the assumptions of the test. A p-value does not measure the size or clinical importance of an effect, and it does not give the probability that the null hypothesis is true. A confidence interval gives a range of values compatible with the data and the interval procedure; it communicates uncertainty in an estimate, subject to the assumptions used.
No p-value, confidence interval, or test result for the primary endpoint is reported in the registry record. The record also does not state a statistical test, model, or adjustment method. Statistical significance and estimate precision therefore cannot be evaluated from this entry.
How should the planning illustration be read?
The registry describes a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the target effect as a stated fraction of the observed effect. In general, such calculations estimate the sample size needed under chosen assumptions; they are planning aids, not observed outcomes and not evidence that a study had a particular probability of detecting an effect.
The registry record does not report the numerical observed primary effect or the sample-size values for the illustrations. The stated equal-allocation, two-arm assumption also differs from this single-group study design. The illustration should therefore be interpreted as a hypothetical planning calculation rather than as a description of the trial's actual design or results. The trial's own sample-size justification and statistical plan are not reported in the registry record.
Why does dispersion matter?
In general, dispersion describes variation among participant measurements. When data vary substantially, a group summary alone may give an incomplete picture of individual responses. The full range can make the extremes visible, while a confidence interval can describe uncertainty about an estimated summary. These are different ideas: the range concerns observed values, whereas a confidence interval concerns estimation uncertainty.
Because neither the endpoint summary values nor a confidence interval are reported, the amount of observed variation and the precision of the primary estimate cannot be assessed. No additional inference should be made from the endpoint definition or the listed enrollment alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 12 outcome measures. The primary endpoint is change from baseline in total mRSS at Week 24. Named secondary outcomes include total mRSS at Week 52; HAQ-DI at Week 24 and Week 52; percent FVC at Week 24 and Week 52; percent DLco at Week 24 and Week 52; and physician global assessment at Week 24. Numerical results for these outcomes are not reported in the registry record.
When a study assesses multiple outcomes or multiple time points, general statistical practice considers whether the analysis plan addresses the increased opportunity for chance findings. Common approaches include designating a primary outcome, prespecifying a testing sequence, or adjusting inference across a defined family of comparisons. The registry record identifies a primary endpoint but does not report a multiplicity strategy, a hierarchy for secondary endpoints, or which comparisons were formally tested. It is therefore not possible to determine how multiplicity was handled.
The record says the full analysis set included all participants who were enrolled. It does not report the number with measurements at each follow-up, the extent or reasons for missing data, or the method used to handle missing observations. It also does not describe whether participants who discontinued treatment or missed visits remained in each analysis. These omissions limit assessment of potential missing-data effects.
The registry reports zero analyses in its analysis listing. It does not provide inferential results for the primary or secondary outcomes. Thus, the outcome definitions are available, but the analysis population at each time point, missing-data handling, statistical testing, and multiplicity control are not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Nemolizumab | 0 | 6 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Nemolizumab | 0 of 6 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Nemolizumab | 6 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Nemolizumab |
|---|---|---|
| Nasopharyngitis | Infections and infestations | 3 of 6 |
| Adenoviral conjunctivitis | Infections and infestations | 1 of 6 |
| Gastroenteritis | Infections and infestations | 1 of 6 |
| Paronychia | Infections and infestations | 1 of 6 |
| Tinea pedis | Infections and infestations | 1 of 6 |
| Oral herpes | Infections and infestations | 1 of 6 |
| COVID-19 | Infections and infestations | 1 of 6 |
| Animal bite | Injury, poisoning and procedural complications | 1 of 6 |
| Ligament sprain | Injury, poisoning and procedural complications | 1 of 6 |
| Vaccination complication | Injury, poisoning and procedural complications | 1 of 6 |
The safety counts are recorded for the nemolizumab group. Serious adverse events were recorded for 0 of 6 participants at risk. Other adverse events were recorded for 6 of 6 participants at risk. Deaths were recorded for 0 of 6 participants at risk. These are counts of participants, not counts of separate event episodes.
The record does not list serious adverse-event terms. The count of 0 serious adverse events means no participant is recorded in that serious-event category in this entry; it does not establish that the risk of serious events is absent. The other-adverse-event count indicates that each participant in the recorded group was included in that category, but the registry summary does not specify the types, severity, timing, or number of events behind the count.
In general, adverse-event counts are descriptive safety observations. They do not establish whether an event was caused by treatment, and a small study group gives limited information about uncommon events. The registry record does not provide further attribution or event-level details, so broader safety conclusions cannot be drawn from these counts alone.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed, open-label, single-group Phase 2 study evaluated nemolizumab in systemic sclerosis. It identifies change from baseline in total mRSS at Week 24 as the primary endpoint and describes how the score was assessed. It also reports the enrolled group size and summary safety counts. These details describe the study's scope and the outcomes it was designed to assess.
The record does not report the numerical primary endpoint result. It therefore does not show the direction or magnitude of change in total mRSS, the range of participant changes, or the precision of an estimated change. No conclusion about the primary endpoint can be drawn from the endpoint definition alone. Numerical results for the listed secondary outcomes are also not reported in the registry record.
Even if a within-group change were available, the single-group design would not establish what would have occurred under a comparator. In general, disease course, measurement variability, concurrent care, and other influences can contribute to changes over time. Without a concurrent comparison group, those influences cannot be separated from a treatment-associated change using this design alone.
The safety counts document the categories recorded in the study group: 0 participants with serious adverse events, 6 with other adverse events, and 0 deaths, with 6 at risk for each category. They do not determine causation or characterize event types in detail. Nor can a study with this enrollment establish the frequency of uncommon events in a broader population.
The results should therefore be understood as incomplete in the registry entry: design and outcome definitions are available, but numerical efficacy findings, uncertainty estimates, detailed safety descriptions, and key analysis methods are not reported in the registry record. The record supports a description of what was studied, not a quantified estimate of efficacy or a broad treatment comparison.
13. Why This Trial Matters Statistically
This study is statistically notable for the contrast between a clearly defined clinical measurement and the limited numerical results available in the registry record. The primary endpoint is specified as change in total mRSS at Week 24, with an explicit description of the site-level assessment and score scale. That definition allows readers to understand what the study intended to measure. The absence of the numerical endpoint result, however, prevents assessment of the observed change or its variability.
The single-group, open-label design also makes the distinction between describing change and estimating a comparative treatment effect especially important. In general, repeated measurements in one group can describe how outcomes vary over time, but they do not supply the counterfactual comparison needed to determine how outcomes would have differed under another treatment or no treatment. This is a design limitation, not a calculation issue that can be resolved by reanalyzing the same group summary.
The enrollment of 6 places particular importance on transparent reporting of participant-level outcome availability and uncertainty. A median and full range may be useful descriptive summaries, but without their values, follow-up completeness, and an uncertainty measure, readers cannot evaluate the observed distribution or precision. The registry record does not report these details.
Finally, the record lists several secondary domains and safety categories but does not report numerical secondary outcomes or detailed event terms. The statistical lesson is that an outcome plan and a trial status do not substitute for outcome data. Interpretation depends on the estimates, their uncertainty, the analysis population, and the methods used to address missing observations and multiple outcomes. Those elements are not reported in the registry record.
14. 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 primary limitation for interpreting efficacy is that the registry record does not report the numerical primary endpoint result. It names the median and full range as the summary format but gives neither value. No confidence interval or test result is reported. Consequently, the observed change, its dispersion, and its precision cannot be evaluated.
The study is open-label and single group, with no comparator. A change from baseline, if reported, would not by itself distinguish treatment-associated change from other changes over time. The registry record also does not report details about assessor training, participant follow-up completeness, or missing-data handling.
The listed enrollment is 6. The full analysis set is defined as all enrolled participants, but the record does not say how many had evaluable primary endpoint measurements at Week 24. It does not provide individual outcome values or explain how unavailable measurements were addressed. These gaps make it impossible to assess how the analysis reflects the enrolled group.
Other limitations include the absence of numerical results for the named secondary outcomes, no reported multiplicity strategy, and no reported statistical test or model. The registry record does not provide dose, administration schedule, eligibility criteria beyond systemic sclerosis, or further treatment details. It also does not report a sample-size justification or full statistical plan. These are limitations of what can be learned from the registry entry; they should not be filled in by assumptions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: An Open-label, Phase 2 Study to Assess the Efficacy and Safety of Nemolizumab in Subjects With Systemic Sclerosis — NCT05214794.
- Registry record last updated 2026-02-13; status last verified 2024-11.
- Results first posted 2026-02-13.
- 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.
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18. Record Summary
The registry record describes a completed, open-label, single-group Phase 2 study of nemolizumab in systemic sclerosis. Enrollment was 6. The study started on 2022-04-20, and primary completion was recorded on 2024-06-04.
The primary endpoint was change from baseline in total mRSS at Week 24, with median and full range specified as the summary format. The record defines the full analysis set as all enrolled participants. It lists 12 outcome measures and names secondary assessments of skin score, disability, lung function, and physician global assessment.
Safety counts include 0 participants with serious adverse events, 6 with other adverse events, and 0 deaths, with 6 at risk in each category. Numerical efficacy results, detailed serious adverse-event terms, missing-data methods, and the statistical analysis plan are not reported in the registry record.