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 A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis (NCT06471361), a na phase 3 study. The primary outcome is Percentage of Effective Self-administrations (SA) of Zilucoplan (ZLP) Using the Zilucoplan-auto-injector (ZLP-AI) From Visit 1 to Visit 8.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT06471361 |
| Phase | Phase 3 |
| Condition | Generalized Myasthenia Gravis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 31 |
| Arms in the primary outcome | n/a |
| Lead sponsor | UCB Biopharma SRL |
| Results posted | Yes |
2. Clinical Question
Population
Participants with generalized myasthenia gravis who received zilucoplan using the auto-injector combination product were the population of interest. The registry record reports enrollment of 31 participants. The Safety Set was defined as participants who received at least 1 dose of zilucoplan with the auto-injector combination product.
Intervention
Participants self-administered zilucoplan subcutaneously with the zilucoplan auto-injector. Dosing was based on body weight. The study evaluated use of this auto-injector for self-administration, including whether the entire dose was delivered as intended.
Comparator
There was no control group or randomized comparison. The design was single-group and open-label, so the results concern observed use of the auto-injector within the study rather than a contrast between treatments or devices.
Primary question
From Visit 1 (Day 1) to Visit 8 (Day 14), what percentage of used and returned auto-injectors had complete dose delivery, as confirmed by the Investigator? The registry record defines effective self-administration by complete delivery of the investigational medicinal product. It does not report the observed percentage or its 95% confidence interval.
The central statistical question is therefore about device-level delivery, not the proportion of participants who experienced a clinical benefit. Devices, rather than participants, are the stated unit for the primary endpoint. The number of devices used and returned, and the resulting endpoint estimate, are not reported in the registry record. The study also registered outcomes concerning self-administration at particular visits and adverse events, but numerical results for those outcomes are not reported in the registry record.
3. Trial Design
This was a Phase 3, multicenter, open-label, outpatient, single-group study of zilucoplan self-administration with an auto-injector. The registry record marks the study completed and reports enrollment of 31 participants. It lists the study start as 2024-08-27 and primary completion as 2025-01-28. The number of participating centers is not reported in the registry record.
Participants self-administered zilucoplan subcutaneously using the auto-injector, with the dose based on body weight. Because the design had a single group and no randomized comparator, the study does not estimate a between-group treatment effect. The absence of masking is consistent with an open-label design; the registry record does not describe additional steps to conceal treatment or device use from participants or investigators.
The primary endpoint covers Visit 1 (Day 1) through Visit 8 (Day 14). The endpoint is defined using auto-injectors that were used and returned in clinic, and the Safety Set includes participants who received at least 1 dose using the combination product. The registry record does not report the number of devices included in the primary endpoint denominator or the number of visits completed by participants.
Secondary outcomes include effective self-administration at Visit 1 and Visit 8, serious adverse events, treatment-emergent adverse events, non-serious adverse device effects, and serious adverse device effects. The record lists 7 outcome measures overall. Allocation is recorded as not applicable, and there is no like-for-like control comparison. The registry record does not report the protocol’s sample-size justification, detailed analysis plan, or whether interim analyses were planned.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: The Participant Flow refers to the Enrolled Set.
Overall Study
| Stage | Zilucoplan |
|---|---|
| STARTED | 31 |
| COMPLETED | 31 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product. Number of units analyzed were defined as total no. of ZLP-AIs used and returned in clinic.
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: Baseline refers safety set (SS) which consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product.
| Characteristic | Unit | Zilucoplan |
|---|---|---|
| Participants analysed | 31 | |
| Age, Continuous | years | 61.4 (SD 11.7) |
| Age, Customized: 18 - <65 years | Participants | 15 |
| Age, Customized: 65 - <85 | Participants | 16 |
| Age, Customized: >=85 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 12 |
| Sex: Female, Male: Male | Participants | 19 |
| Race/Ethnicity, Customized: Asian | Participants | 1 |
| Race/Ethnicity, Customized: Black or African American | Participants | 3 |
| Race/Ethnicity, Customized: White | Participants | 25 |
| Race/Ethnicity, Customized: Other or Mixed | Participants | 2 |
| Race/Ethnicity, Customized: Hispanic or Latino | Participants | 1 |
| Race/Ethnicity, Customized: Not Hispanic or Latino | Participants | 30 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Effective Self-administrations (SA) of Zilucoplan (ZLP) Using the Zilucoplan-auto-injector (ZLP-AI) From Visit 1 to Visit 8, measured over From Visit 1 (Day 1) to Visit 8 (Day 14).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | Percentage of ZLP-AI devices |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the percentage of zilucoplan auto-injectors with effective self-administration from Visit 1 (Day 1) to Visit 8 (Day 14). Effectiveness for this endpoint means that the full dose was delivered, with completeness confirmed by the Investigator. The operational description says the yellow plunger visible through the device window was completely depressed.
The stated unit is the auto-injector device, not the participant. The endpoint is based on the number of devices with complete dose delivery divided by the total number of devices used and returned. It therefore describes observed device-level delivery among the devices included in the assessment. It is not a measure of symptom change, clinical response, or the percentage of participants who completed all visits.
As a general statistical point, a device-level percentage can describe how consistently the intended delivery was achieved in the observed uses. The confidence interval communicates uncertainty around that estimated percentage, rather than establishing that every use will have the same result. The registry record specifies a 95% confidence interval but does not report the primary estimate, its interval limits, or the number of devices in the denominator. The result and its precision therefore cannot be assessed from the registry record.
The outcome’s interpretation depends on the recorded definition and denominator. Because only used and returned devices are included, the registry record’s stated endpoint does not by itself describe devices that were not returned or uses outside the study assessment.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a study intended to evaluate self-administration of zilucoplan with an auto-injector, including effectiveness, safety, and tolerability. Its primary endpoint is a device-level measure of complete dose delivery across the stated visit period. However, the registry record does not report the observed primary percentage, confidence interval limits, or number of devices assessed. Without those results, the extent of complete delivery and the precision of the estimate cannot be characterized.
The record reports enrollment of 31 participants. This enrollment count is not the denominator for the primary device-level endpoint: that endpoint uses the number of auto-injectors used and returned. The number of devices is not reported in the registry record, and it is not appropriate to substitute participant enrollment for the endpoint denominator. The record also does not report the number of participants contributing to each visit-specific secondary outcome.
The adverse-event summary records 0 serious adverse events among 31 participants at risk, and 3 events in the other-adverse-event count among 31 at risk. These are counts in the registry summary, not measures of comparative risk. No deaths are recorded in that summary. The record does not provide event-level descriptions for the serious-event terms, and its serious-event term list is empty. The listed secondary outcomes include treatment-emergent adverse events and adverse device effects, but their outcome-specific numerical results are not reported in the registry record. The other-adverse-event count should not be treated as a result for any particular listed secondary outcome without a clearer mapping in the record.
As a general statistical point, confidence intervals matter because an estimate from a study is subject to sampling variability. An interval’s width helps convey how much uncertainty remains, while its limits show a range of values compatible with the analysis under its assumptions. No such interval limits are reported for the primary endpoint, so the registry record cannot support a statement about its precision.
The single-group, open-label design can describe observed self-administration and recorded events in the study, but it does not provide a randomized comparison. Consequently, the record cannot establish how device use or event counts would compare with another device, usual care, or a control group. The registry record also does not report a clinical efficacy endpoint result. Its listed primary endpoint concerns completeness of dose delivery, which is distinct from clinical benefit.
9. Statistical Methods Explained
The registry record defines the primary endpoint as a percentage of auto-injectors with complete dose delivery. The numerator is the number of used and returned devices for which the Investigator confirmed complete delivery. The denominator is the total number of auto-injectors used and returned in clinic. This makes the analysis unit a device. A participant may contribute device observations, but the record does not report the number of devices per participant or how within-participant clustering was handled.
The primary endpoint is specified for the period from Visit 1 (Day 1) to Visit 8 (Day 14). Complete delivery is described operationally: the entire investigational medicinal product dose was delivered, as confirmed by the Investigator, with the yellow plunger completely depressed and visible through the device window. This definition connects the statistical outcome to a concrete observation. It does not, by itself, indicate how disagreements, incomplete observations, or missing device returns were classified.
The registry record specifies a 95% confidence interval for the primary percentage. In general, a confidence interval expresses uncertainty in an estimate under the assumptions of the statistical method. Its width depends in part on the amount of information available and the pattern of observed outcomes. The interval’s calculation method is not reported in the registry record. The record also does not give the primary percentage, interval limits, or device denominator, so neither the result nor its precision can be evaluated.
The parameter is identified as a number, while the endpoint is expressed as a percentage of devices. Those descriptions point to a summarized count-based outcome reported on a percentage scale. The record does not give further detail about the estimator, interval construction, or any adjustment for repeated devices contributed by the same participant. General statistical methods for proportions can differ in how they account for small denominators or correlated observations; which approach was used is not reported in the registry record.
The study is single-group and open-label, with no randomized control comparison. This means the primary analysis is descriptive of observed device use in the study group rather than an estimate of a contrast between groups. No treatment effect, comparative confidence interval, or between-group hypothesis test is reported in the registry record. The design also does not permit separating device-related observations from other influences through a randomized comparison.
The listed safety outcomes include serious adverse events, treatment-emergent adverse events, non-serious adverse device effects, and serious adverse device effects. These are distinct categories in the registered outcome descriptions. The adverse-event summary records serious events and an other-event count, but does not report numerical results against each of the listed outcome definitions. It is therefore important not to assign the other-event count to a specific category without an explicit definition in the registry record.
General statistical interpretation of event counts requires attention to the denominator, the length and completeness of observation, and how events are defined and collected. The record gives 31 participants at risk for the summary counts, but does not report event-level detail, person-time, or participant-level outcome data. The recorded count cannot be translated into an incidence rate or compared across treatments from the information reported.
The registry record lists 7 outcome measures. It does not report whether a hierarchy or adjustment for multiple outcome analyses was specified. When several outcomes are examined, general statistical interpretation should distinguish pre-specified primary questions from secondary or exploratory questions and should consider the risk of emphasizing chance findings. The registry record does not provide enough information to determine how multiplicity was handled.
The study’s sample-size justification and detailed statistical analysis plan are not reported in the registry record. No interim analyses are reported. Planning calculations are design illustrations, not evidence that a study had a particular probability of detecting an effect after the results were observed. Any sample-size planning illustration should not be read as observed power. There are no reported primary endpoint estimates in the registry record from which to assess a recalculation or an observed effect.
For missing data, the record does not describe how missing visits, unreturned devices, incomplete assessments, or withdrawals were handled in the primary analysis. As a general matter, missing outcome information can affect both the denominator and the interpretation of an estimate. The stated denominator includes devices used and returned; the record does not explain whether excluded or unavailable devices were summarized separately.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 7 outcome measures, including the primary device-level endpoint and secondary outcomes for self-administration at Visit 1 and Visit 8, serious adverse events, treatment-emergent adverse events, and serious and non-serious adverse device effects. It does not report an outcome hierarchy beyond identifying the primary endpoint, nor does it describe adjustment for multiple comparisons or a strategy for interpreting secondary outcomes. Whether a multiplicity procedure was planned is not reported in the registry record.
As a general statistical point, examining several outcomes can increase the chance that at least one result appears notable by chance. A prespecified hierarchy or adjustment can help define how evidence across outcomes should be interpreted. Without a reported plan, secondary results should not be treated as if they were independent confirmatory tests. The registry record does not provide numerical results for the listed secondary outcomes.
Missing-data procedures are not reported in the registry record. The primary endpoint denominator is the total number of auto-injectors used and returned in clinic, but the record does not describe how unreturned devices, missing visit assessments, incomplete observations, or participant withdrawal were handled. It also does not report whether sensitivity analyses were conducted.
The Safety Set is described as participants who received at least 1 dose of zilucoplan with the auto-injector combination product. The record does not describe additional analysis populations or explain whether the primary endpoint used any population beyond devices contributed by members of this Safety Set. The number of devices analyzed and participant-level analysis counts are not reported. These omissions limit assessment of outcome completeness, denominator selection, and the robustness of any endpoint estimate.
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 |
|---|---|---|---|
| Zilucoplan | 0 | 31 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Zilucoplan | 0 of 31 |
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 |
|---|---|
| Zilucoplan | 3 of 31 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Zilucoplan |
|---|---|---|
| Injection site pain | General disorders | 3 of 31 |
The adverse-event summary records 0 serious adverse events among 31 participants at risk. It also records 3 events in the other-adverse-event count among 31 participants at risk, and 0 deaths. These are counts as recorded, not percentages; no percentages should be inferred from them without calculating a new measure.
The summary does not identify the other-event count as a result for a particular registered secondary outcome. The registry record lists treatment-emergent adverse events and serious and non-serious adverse device effects separately, but outcome-specific numerical results for those categories are not reported in the registry record. The serious-event term list is empty, and the record does not provide event-level descriptions.
As a general statistical point, a zero count in a study group does not establish that an event is impossible or that the risk is zero in other settings. Counts are interpreted in light of the number of people observed, observation duration, event definitions, and completeness of reporting. The registry record does not report enough detail about duration or event-level assessment to characterize those factors fully. Because there is no control group, these counts cannot be used to estimate a comparative safety difference.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a completed study of zilucoplan self-administration with an auto-injector among participants with generalized myasthenia gravis. It defines complete dose delivery as the primary endpoint and identifies the relevant period as Visit 1 (Day 1) to Visit 8 (Day 14). It also reports enrollment of 31 participants and a Safety Set definition based on receiving at least 1 dose with the combination product.
The record does not report the primary endpoint percentage, the 95% confidence interval limits, or the number of devices used and returned. It therefore does not show how often complete delivery was observed or how precisely that rate was estimated. Nor does the record report numerical results for the listed visit-specific self-administration outcomes. These absences prevent a result-level conclusion about device-level delivery from the registry record alone.
The adverse-event summary records 0 serious adverse events among 31 participants at risk, 3 in the other-event count, and 0 deaths. These figures describe the posted summary, not a comparison with another intervention. The other-event count is not clearly mapped to each registered safety outcome in the record. No comparative safety effect can be estimated from this single-group study.
As a general statistical distinction, successful completion of a device-use endpoint is not the same as demonstrating clinical efficacy. The primary endpoint concerns whether the intended dose was completely delivered, while the registered study description also refers broadly to effectiveness, safety, and tolerability. The registry record does not report a clinical efficacy result.
The design can describe observations made during the study, but it cannot show how results would differ under a randomized alternative. The registry record also does not give the detailed analysis plan, missing-data handling, or sample-size justification. Those gaps mean that the statistical assumptions and the completeness of the analysis cannot be fully assessed from the reported information.
13. Why This Trial Matters Statistically
This study raises a statistical question that differs from the usual comparison of clinical outcomes between treatment groups. Its primary endpoint is the proportion of used and returned devices with complete delivery, making the device the stated unit of analysis. That distinction matters because participant enrollment is not the primary endpoint denominator, and the number of devices contributing to the outcome is not reported in the registry record.
The endpoint uses an operational definition tied to investigator confirmation and a visible device indicator. A clearly described measure can help readers understand what was counted, but interpretation still depends on how observations were collected, what happened when a device was unavailable for assessment, and how repeated devices from the same participant were treated. Those analysis details are not reported in the registry record.
The single-group design is also important. It can describe the observed experience of self-administration within the study, but without a control group it cannot estimate a comparative difference in device use, safety, or clinical outcomes. The open-label design means participants and investigators were not masked, and the registry record does not describe additional steps to reduce possible assessment influences.
Finally, the distinction between device delivery and clinical efficacy is central. Complete dose delivery is a use-related outcome; it does not itself establish a change in the underlying condition. The registry record defines the delivery endpoint but does not report its result or a clinical efficacy result. The statistical importance of the study is therefore in the type of question it asks and the denominator it uses, while the posted information leaves the outcome estimate and its precision unknown.
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 main limitation for interpreting the primary endpoint is that the registry record does not report the observed percentage, confidence interval limits, or number of devices used and returned. Since the endpoint is device-based, the reported participant enrollment of 31 cannot substitute for the missing device denominator. Without the result and denominator, the magnitude and precision of the primary outcome are unknown.
The study is single-group and open-label, with no randomized comparator. Its observations cannot establish a comparative effect against another device or care approach. The registry record does not report the number of centers, details of device-use training, or the process used to assess and document completeness beyond the stated Investigator confirmation and device-window indicator.
The record does not describe handling of missing visits, unreturned devices, incomplete assessments, or withdrawals. It also does not report how repeated device observations within participants were handled statistically. These details matter for understanding the analyzed population and whether the reported device-level estimate, if available, represents all intended study observations.
Although the registered outcomes include several safety categories and visit-specific self-administration outcomes, numerical results for those endpoints are not reported in the registry record. The adverse-event summary’s other-event count is not explicitly linked to a particular listed outcome. Event descriptions and observation duration are also not reported in the available record.
The registry record does not report the sample-size justification, detailed analysis plan, interim analyses, or multiplicity and missing-data procedures. These limitations are specific to the information available for this study and restrict assessment of the precision, completeness, and analysis choices behind its outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis — NCT06471361.
- Registry record last updated 2026-03-27; status last verified 2026-03.
- Results first posted 2026-03-27.
- 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.
18. Record Summary
The registry record describes a completed Phase 3, multicenter, open-label, outpatient, single-group study of zilucoplan auto-injector self-administration in generalized myasthenia gravis. Enrollment was 31 participants. The study start is listed as 2024-08-27 and primary completion as 2025-01-28.
The primary endpoint is the percentage of used and returned devices with complete dose delivery, confirmed by the Investigator, from Visit 1 (Day 1) to Visit 8 (Day 14). The record specifies a 95% confidence interval but does not report the endpoint estimate, interval limits, or device denominator.
The adverse-event summary records 0 serious adverse events and 3 events in the other-event count among 31 participants at risk, with 0 deaths. Numerical results for the registered secondary outcomes are not reported in the registry record. The study has no randomized comparator, and its detailed statistical plan, missing-data procedures, and sample-size justification are not reported.