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 Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis (NCT00530075), a na phase 2 study. The primary outcome is Remission of Vasculitis.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00530075 |
| Phase | Phase 2 |
| Condition | Wegener's Granulomatosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 45 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Nippon Kayaku Co., Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with active, refractory Wegener's granulomatosis. Entry required a Birmingham Vasculitis Activity Score (BVAS) of at least 4 and previous treatment with cyclophosphamide or methotrexate. The registry describes Wegener's granulomatosis as a systemic vasculitis that predominantly affects the respiratory tract and kidneys.
Intervention
Participants received gusperimus by self-administered subcutaneous injection at 0.5 mg/kg/day. The planned regimen used six cycles of 21 days, with a seven day washout between cycles. Cycles were stopped early if the white blood count fell below 4,000/mm3. After the sixth cycle, azathioprine was commenced and follow-up continued for a further six months.
Comparator
There was no concurrent comparator group. The study was a single-group, open-label study, and allocation is recorded as not applicable. Prednisolone doses were adjusted according to clinical status, while immunosuppressive drugs were withdrawn at entry. These treatment details do not create a randomized control group.
Primary question
The primary question was whether participants met the registry's definition of complete or partial remission of vasculitis. Complete remission required BVAS of 0 sustained for at least 2 months. Partial remission required a reduction in BVAS of 50% or more from the entry score, also sustained for at least 2 months. The registry record does not report the observed remission result, so it cannot establish how often either endpoint occurred.
3. Trial Design
This was a Phase II, prospective, international, multicentre, single-group, open-label study of gusperimus in patients with refractory Wegener's granulomatosis. The registry records an enrollment of 45 and a completed study. Because it had a single treatment group, the design did not include a randomized or concurrent control arm. Allocation is listed as not applicable, and masking as none.
Eligibility required active disease, with BVAS at least 4, and previous treatment with cyclophosphamide or methotrexate. At entry, immunosuppressive drugs were withdrawn. Prednisolone doses could be adjusted according to clinical status. Gusperimus was self-administered by subcutaneous injection at 0.5 mg/kg/day over six treatment cycles. Each cycle lasted 21 days, with a seven day washout between cycles. A cycle was stopped early if the white blood count fell below 4,000/mm3. Azathioprine was commenced after the sixth cycle, followed by a further six months of follow-up.
The primary efficacy outcome was remission of vasculitis, assessed during treatment at entry and on Day 22 of cycles 1–6, up to 24 weeks. The study also listed secondary outcomes including duration of clinical response, haematuria, creatinine, ANCA, CRP, Vasculitis Damage Index, and SF-36. The registry record does not report a primary outcome result, details of the analysis used, or results for these secondary outcomes. The design therefore describes the intended assessment but does not provide enough posted outcome information to estimate remission frequency or compare outcomes with another 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.
Overall Study
| Stage | Gusperimus |
|---|---|
| STARTED | 45 |
| COMPLETED | 38 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Gusperimus |
|---|---|
| Adverse Event | 1 |
| Death | 1 |
| Lack of Efficacy | 3 |
| Protocol Violation | 1 |
| Withdrawal by Subject | 1 |
Analysis population for the primary outcome, as defined in the registry: Efficacy Population
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.
| Characteristic | Unit | Gusperimus |
|---|---|---|
| Participants analysed | 45 | |
| Age, Continuous | Years | 51 (19 to 77) |
| Gender: Female | Participants | 16 |
| Gender: Male | Participants | 29 |
| Region of Enrollment: Europe | participants | 45 |
6. Primary Endpoint
The registry lists the primary outcome as Remission of Vasculitis, measured over At Entry (Day 1 of Cycle 1), Day 22 of cycles 1-6, up to 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was remission of vasculitis, recorded as a percentage of participants in the efficacy population. It combined two possible response states, each defined using the Birmingham Vasculitis Activity Score (BVAS). Complete remission required a BVAS of 0 sustained for at least 2 months. Partial remission required a reduction in BVAS of 50% or more compared with the entry score, sustained for at least 2 months. Entry required active disease, measured by BVAS, with a score of at least 4.
BVAS records clinical manifestations attributable to active vasculitis using predefined organ-specific items. A remission endpoint based on this score is intended to capture a reduction in measurable disease activity rather than a change in a single symptom or laboratory measurement. The sustained-duration requirement distinguishes a maintained response from a score change that may be brief. Complete and partial remission are distinct levels of response, although the registry describes them together as the primary efficacy outcome.
The assessment schedule was at entry and on Day 22 of cycles 1–6, up to 24 weeks. The registry record does not report the observed percentage meeting the endpoint, the number assessed at each time, or the result for complete versus partial remission separately. It therefore does not permit a reading of the endpoint's observed frequency or precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies remission of vasculitis as the primary efficacy outcome, but does not post a remission percentage or a result for complete or partial remission. The record also does not provide a confidence interval, a p-value, or another statistical estimate for this endpoint. As a result, the posted information does not quantify how often participants met the stated response definition or how precise such an estimate would be.
The study's single-group design is central to interpretation. Without a concurrent comparison group, an outcome observed during treatment would not by itself separate the effect of gusperimus from the course of the underlying disease, changes in prednisolone, the withdrawal of other immunosuppressive drugs, or other influences. The open-label design also means treatment and outcome assessment were not masked, according to the registry record. These design features limit causal comparisons; they do not establish that a treatment effect was present or absent.
The primary endpoint uses BVAS change and requires a sustained response. This gives the outcome a defined clinical activity measure and duration criterion. However, the registry does not report how endpoint components were combined in the result, whether results were reported separately for complete and partial remission, or how participants without a recorded assessment were handled. The efficacy population is named, but its precise membership and analysis rules are not reported in the registry record.
Safety information is more explicit than efficacy information in the posted record. The adverse-event listing gives 17 of 45 participants with a serious adverse event. Listed serious terms include leucopaenia/anaemia and infections, each reported as 15.6%, and bronchopneumonia as 11.1%. These percentages describe safety entries; they do not supply the missing remission result. The registry does not report deaths in the field summarized for deaths, and that absence should be read as information not reported rather than as evidence that none occurred.
Overall, the registry describes the treatment regimen, endpoint definitions, follow-up schedule, and selected serious adverse-event information. It does not provide the primary outcome estimate or an inferential analysis. Interpretation should therefore remain at the level of study design and what outcomes were specified, rather than a claim about the frequency or comparative effect of remission.
9. Statistical Methods Explained
The registry record describes a single-group study and lists no analysis entries. It gives the primary endpoint definition and assessment schedule, but does not report the statistical procedures used to analyze the efficacy or safety outcomes. The explanations below are general statistical context; they do not identify methods that the investigators used unless the registry record explicitly states them.
What does a percentage endpoint represent?
In general, a percentage for a binary outcome expresses the fraction of an analysis population that meets a specified event definition. For this study, the event definition is remission, comprising complete remission or partial remission as described in the endpoint criteria. A percentage is interpretable only alongside its denominator: readers need to know which participants were eligible for the analysis and how missing assessments affected inclusion. Although the registry names an efficacy population, it does not report the number analyzed or the rules used to define membership. No remission percentage is posted.
A percentage alone also does not communicate precision. In general, a confidence interval describes a range of values compatible with the observed data under the interval's statistical assumptions. Wider intervals indicate less precision, while narrower intervals indicate greater precision. The registry does not report a remission estimate or confidence interval, so neither the observed value nor its precision can be assessed from the posted record.
Why does the single-group design matter?
In general, a concurrent control group helps distinguish changes associated with an intervention from changes that might occur without it. This study has one treatment group and no randomized comparison. Accordingly, even if a response rate were posted, that rate would describe the observed group and would not provide a direct treatment-versus-control contrast. Disease course, concomitant care, and other influences could also contribute to outcomes. The registry specifically reports that prednisolone doses were adjusted according to clinical status and immunosuppressive drugs were withdrawn at entry; these details are part of the treatment context.
Open-label means masking was absent in the registry record. In general, knowledge of treatment assignment can affect care decisions, reporting, and assessment, particularly for outcomes involving clinical judgment. The registry does not describe any blinded outcome assessment or procedures intended to address this concern. It would therefore be inappropriate to assume such procedures were used.
How should repeated assessments be understood?
The primary outcome timeframe includes assessments at entry and on Day 22 of cycles 1–6, up to 24 weeks. Repeated assessments can describe the course of a participant's outcome over time. In general, observations from the same participant are related, so an analysis of repeated measurements may need to account for that dependence. The registry does not report whether the investigators analyzed each visit separately, selected a particular time point, or modeled the repeated measurements together. Nor does it report how the duration requirement for remission was operationalized in the analysis.
The primary response definition requires remission to be sustained for at least 2 months. That condition is more than a single score threshold: it requires information about duration. In general, sustained-response outcomes can depend on the timing and completeness of follow-up assessments. The registry describes the visits and definition but does not explain how incomplete or irregular assessments were handled.
What do the planning assumptions mean?
The record describes sample-size calculations as planning illustrations based on an observed effect, with two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. It further describes illustrations for detecting stated fractions of that effect. These are assumptions for hypothetical planning, not evidence of power achieved in the completed study. In particular, a planning calculation is not an observed result, and the record does not report an analysis demonstrating that the study had a particular probability of detecting an effect. The listed study itself was single-group, whereas the planning assumptions refer to two-arm allocation. Those assumptions should not be mistaken for the design actually recorded.
In general, a significance level is a threshold used in a prespecified testing procedure; it does not measure the probability that a treatment works. The registry does not provide a p-value or a statistical analysis plan, and it does not report whether any such threshold was used for the primary endpoint. No test result can be inferred from the planning assumptions.
What does the safety information measure?
The adverse-event information includes participants with serious events and percentages for selected serious terms. In general, participant-level event percentages and counts of event occurrences answer different questions: one participant may experience more than one event. The record does not clarify whether the listed term percentages represent participants, event episodes, or another summary convention. They should be read as registry-reported safety entries, not reconstructed into an event total. General statistical practice also distinguishes descriptive safety summaries from causal comparisons; the single-group design does not offer a concurrent basis for comparing event risk.
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.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Gusperimus | 44 | 42 | 95.5% | 84.5% to 99.4% | 84.9% to 98.7% | 84.9% to 98.6% |
R code and output
arm <- c("Gusperimus")
n <- c(44)
events <- c(42)
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 Gusperimus 44 42 95.5 84.5 99.4 84.9 98.7 84.9 98.611. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary remission outcome and eight outcome measures overall. Secondary outcomes include duration of clinical response, haematuria, creatinine, ANCA, CRP, Vasculitis Damage Index, and SF-36. Assessing several outcomes or time points can raise multiplicity concerns: in general, the chance of at least one chance-positive finding can increase when many statistical tests are conducted. A prespecified hierarchy, adjustment procedure, or clear distinction between primary and exploratory analyses can help readers interpret multiple findings. The statistical plan and any multiplicity strategy are not reported in the registry record.
The record names an efficacy population but does not define its inclusion rules or give the number analyzed. It also does not report whether the primary analysis used all enrolled participants, only participants with an available endpoint, or another analysis population. Missing outcome counts, reasons for missingness, and methods for handling missing data are not reported in the registry record. Consequently, the impact of incomplete outcome information on any remission estimate cannot be evaluated.
The assessment schedule includes repeated visits through Day 22 of cycles 1–6, up to 24 weeks, and the remission definition requires a response sustained for at least 2 months. In general, both repeated observations and a duration requirement make the treatment of missed visits relevant: a missing measurement might affect whether a participant can be classified as having a sustained response. The registry does not describe the classification rules for such cases.
The registry lists zero analysis entries. This describes the analysis information posted in the record; it does not establish that no analyses were conducted. No multiplicity adjustment, missing-data procedure, or outcome analysis is reported in the registry record.
12. 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 |
|---|---|---|---|
| Gusperimus | 17 | 45 | 37.8% |
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 |
|---|---|
| Gusperimus | 45 of 45 |
Most frequent serious adverse event terms
| Event term | Organ system | Gusperimus |
|---|---|---|
| Leucopaenia/Anaemia | Blood and lymphatic system disorders | 7 of 45 |
| Infections | Infections and infestations | 7 of 45 |
| Bronchopneumonia | Infections and infestations | 5 of 45 |
| Relapses of Wegener's granulomatosis | Respiratory, thoracic and mediastinal disorders | 4 of 45 |
| Acute upper airway obstruction | Respiratory, thoracic and mediastinal disorders | 2 of 45 |
| Dental abscess | Infections and infestations | 1 of 45 |
| Campylobacter jejuni diarrhoea | Infections and infestations | 1 of 45 |
| Active Wegener's granulomatosis | Respiratory, thoracic and mediastinal disorders | 1 of 45 |
| Food poisoning | Gastrointestinal disorders | 1 of 45 |
| Diarrhoea | Gastrointestinal disorders | 1 of 45 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Gusperimus |
|---|---|---|
| Injection site pain/pruritus | General disorders | 26 of 45 |
| Pain oral cavity/throat | Gastrointestinal disorders | 19 of 45 |
| Diarrhoea | Gastrointestinal disorders | 18 of 45 |
| Leucopaenia | Blood and lymphatic system disorders | 17 of 45 |
| Nausea | Gastrointestinal disorders | 16 of 45 |
| Lower respiratory tract infection | Infections and infestations | 12 of 45 |
| Acne | Skin and subcutaneous tissue disorders | 11 of 45 |
| Alopecia | Skin and subcutaneous tissue disorders | 11 of 45 |
| Vomoting | Gastrointestinal disorders | 10 of 45 |
| Upper respiratory tract infection | Infections and infestations | 9 of 45 |
Serious events with the highest arm-level rate: leucopaenia/anaemia (15.6%), infections (15.6%), bronchopneumonia (11.1%), relapses of wegener's granulomatosis (8.9%), acute upper airway obstruction (4.4%).
The adverse-event record lists 17 of 45 participants as affected by serious adverse events. It also lists serious-event terms with percentages: leucopaenia/anaemia, 15.6%; infections, 15.6%; bronchopneumonia, 11.1%; relapses of Wegener's granulomatosis, 8.9%; acute upper airway obstruction, 4.4%; and dental abscess, 2.2%. These are descriptions of the posted safety information, not comparisons with another treatment group.
In general, a participant can experience more than one type of event, so percentages for event terms should not be added together or interpreted as mutually exclusive categories. The registry record does not explain whether each listed percentage counts participants with that term or event episodes, nor does it provide further detail on timing or duration. The serious-event listing is separate from the primary remission endpoint and cannot substitute for its unreported result.
The field for deaths does not contain a reported value. This means deaths are not reported in the registry record; it should not be interpreted as confirmation that no deaths occurred. The single-group design also means the safety figures do not provide a comparative estimate of risk.
13. What the Result Does, and Does Not, Mean
The registry record documents a completed Phase II study of gusperimus in patients with refractory Wegener's granulomatosis. It specifies a single-group, open-label design, the treatment regimen, the remission definition, and a schedule of assessments. It also reports selected serious adverse-event information. These details allow readers to understand what the study set out to evaluate and some of the safety events recorded.
The central efficacy result is not reported in the registry record. There is no posted percentage for complete remission, partial remission, or the combined primary endpoint, and there is no confidence interval or inferential result. Therefore, the posted information does not show how many participants met the remission definition or how precisely that result was estimated. It cannot establish a treatment effect or a lack of one.
Even with a posted remission result, a single-group study would describe outcomes among participants receiving the study regimen rather than provide a randomized comparison. A before-and-after change or an observed response percentage would not by itself identify what would have happened under another treatment or without gusperimus. Prednisolone adjustment, withdrawal of immunosuppressive drugs, disease course, and other influences form part of the clinical context, but the registry does not estimate their separate contributions.
The serious adverse-event listing offers a descriptive account of events in the study group. Without a concurrent comparator, those figures do not quantify comparative safety. The absence of a reported death value is not evidence that no deaths occurred, and selected event terms are not a complete account of all possible safety outcomes.
In short, the record supports a description of the trial's structure, eligibility, endpoint, treatment schedule, and listed safety information. It does not support a numerical conclusion about remission frequency, efficacy precision, or comparative benefit and harm.
14. Why This Trial Matters Statistically
This study illustrates why a clearly defined endpoint is not the same as a reported result. The registry specifies remission through BVAS thresholds and a sustained-duration requirement, and it states when assessments were planned. Those details define the intended clinical outcome. Without an observed remission estimate, however, readers cannot assess its frequency or precision.
It also highlights the distinction between treatment-group outcomes and treatment comparisons. A single-group study can document what occurred among people who received a regimen, but a concurrent control is needed for a direct comparison of outcomes under different strategies. The open-label design and adjustment of prednisolone according to clinical status add context to how outcomes might be observed; they do not create a controlled contrast.
The trial's reporting also shows why an analysis population and missing-data rules matter. The efficacy population is named, but its membership, analyzed denominator, and handling of incomplete assessments are not reported. For a response requiring sustained remission over time, the way missed or unavailable assessments are treated can influence classification. Without those details and the endpoint result, statistical uncertainty cannot be examined.
Finally, the posted safety information and efficacy information are not equally complete. Serious-event counts and selected percentages are listed, while the primary efficacy result and statistical analyses are not. This difference reinforces the need to separate a description of a registered study from evidence that permits an estimate or comparison. The study remains informative about the planned regimen and outcome framework, while the registry record leaves key inferential questions unanswered.
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 limitation in interpreting the posted record is that it does not report the primary remission result. No remission percentage, analysis denominator, confidence interval, or p-value is given. The statistical analysis plan and the methods used for the primary endpoint are also not reported in the registry record.
The single-group, open-label design has no concurrent control for estimating a comparative treatment effect. Allocation is listed as not applicable and masking as none. Because prednisolone could be adjusted according to clinical status and immunosuppressive drugs were withdrawn at entry, the treatment context included changes beyond the gusperimus regimen itself. The registry does not provide analyses separating these influences.
The outcome definition requires remission to be sustained for at least 2 months, with assessments at entry and on Day 22 of cycles 1–6, up to 24 weeks. The record does not explain how sustained status was established across visits, or how missing or incomplete observations were classified. Although an efficacy population is named, its exact definition and size are not reported in the registry record.
The registry lists secondary measures but does not report their results or any approach to multiple outcomes and repeated assessments. It also lists zero analysis entries, which means analysis details are not posted in the record; it does not establish that no analyses were conducted. Safety entries are limited in the record to the listed serious-event information and selected terms. The deaths field has no reported value, and event-term counting conventions are not explained.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis — NCT00530075.
- Registry record last updated 2017-03-06; status last verified 2007-09.
- Results first posted 2017-03-06.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
The registry record identifies NCT00530075 as a completed Phase II study of gusperimus in refractory Wegener's granulomatosis. It describes an international, multicentre, prospective, single-group, open-label design and an enrollment of 45. Participants received gusperimus by subcutaneous injection in six treatment cycles, with post-cycle azathioprine and further follow-up.
The primary outcome was remission of vasculitis, defined using BVAS and a sustained response requirement. The registry record does not report the observed primary outcome or statistical analyses. It lists 17 of 45 participants with serious adverse events and gives percentages for selected serious-event terms. The record does not report a value for deaths. Thus, it provides study and endpoint descriptions, along with selected safety information, but not a numerical efficacy result or comparative treatment estimate.