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 Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis (NCT03686969), a randomized phase 3 study. The primary outcome is MMT-8.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03686969 |
| Phase | Phase 3 |
| Condition | Dermatomyositis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Octapharma |
| Results posted | Yes |
2. Clinical Question
Population
Patients with dermatomyositis were the intended study population. The registry record states that the trial was terminated prematurely after one patient was enrolled and treated, so the record does not describe a completed group of participants from which to estimate an average treatment effect.
Intervention
The active intervention was subcutaneous human immunoglobulin, identified as Octanorm. The arm description specifies 0.5g/kg/week octanorm 16.5%. The registry describes the study as evaluating efficacy and safety.
Comparator
The comparator was placebo. The study was described as randomized, double-blind, placebo-controlled, and parallel-group. Details about the randomization procedure and who was masked are not reported in the registry record.
Primary question
The primary outcome was MMT-8 at 32 weeks. In broad terms, the study asked whether the Octanorm group would have a different MMT-8 outcome from the placebo group. The registry record reports that efficacy and safety analyses were not performed because only one patient was enrolled and treated. It therefore contains no analyzed treatment comparison that answers this question.
In a completed trial, random allocation and masking can help make a comparison less vulnerable to systematic differences in care or assessment. Those design features do not, by themselves, create evidence of a treatment difference: an analyzable outcome comparison is still needed. That comparison is absent from this record.
3. Trial Design
The registry describes a randomized, double-blind, placebo-controlled Phase III study with a parallel-group design. It evaluated subcutaneous human immunoglobulin (Octanorm) in patients with dermatomyositis, with placebo as the comparator. Octapharma is listed as the sponsor. The active-arm description gives a dose of 0.5g/kg/week octanorm 16.5%.
The record lists the study start as 2018-08-02 and primary completion as 2018-11-22. Its status is terminated. The primary outcome description explains that the study ended prematurely and that only one patient was enrolled and treated. Efficacy and safety analyses were not performed. Thus, although the study was designed to compare two groups, the record does not contain a completed comparative analysis.
In general, randomization assigns participants to study groups according to a chance-based process, while double blinding is intended to limit the influence of treatment knowledge on participants and relevant study personnel. The specific allocation procedure, concealment process, and identities of those masked are not reported in the registry record. Nor does the record explain why the trial was terminated. A sample-size justification and detailed statistical analysis plan are not reported in the registry record.
The record names Extra-Muscular Disease Activity, several muscle enzymes, the Health Assessment Questionnaire, and the SF-36v2 Health Survey among the secondary outcomes. It lists 59 outcome measures and zero analyses. Outcome listings indicate what was to be assessed; they do not establish that a result was obtained or analyzed. The available record therefore describes the intended trial structure, but not an interpretable efficacy comparison.
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 | Octanorm | Placebo |
|---|---|---|
| STARTED | 1 | 0 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 1 | 0 |
Reasons for not completing this period:
| Reason | Octanorm | Placebo |
|---|---|---|
| Study terminated | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.
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: Early study termination.
| Characteristic | Unit | Octanorm | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 1 | 0 | 1 | |
| Age, Customized: Age | years | 52 | — | 52 |
| Sex: Female, Male: Female | Participants | 1 | — | 1 |
| Sex: Female, Male: Male | Participants | 0 | — | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | — | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | — | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | — | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | — | 0 |
| Race (NIH/OMB): White | Participants | 1 | — | 1 |
| Race (NIH/OMB): More than one race | Participants | 0 | — | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | — | 0 |
6. Primary Endpoint
The registry lists the primary outcome as MMT-8, measured over 32 weeks.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome was MMT-8, assessed at 32 weeks. The registry describes MMT-8 as a set of 8 designated muscles tested bilaterally, with a potential score of 0 - 150. It does not give a unit or a more detailed scoring description in the primary outcome entry.
As a general clinical measurement concept, testing muscle strength can be relevant in a condition that involves muscle disease. A standardized assessment can turn observations across specified muscles into a score that may be compared between study groups or over time. For an endpoint to support a treatment comparison, however, the study needs usable outcome measurements and an analysis that relates those measurements to the assigned groups.
Here, the registry record says that the study was terminated prematurely and that efficacy and safety analyses were not performed because only one patient was enrolled and treated. It does not report an MMT-8 result, a change from baseline, a group-level estimate, or an uncertainty interval. The planned assessment at 32 weeks therefore cannot be interpreted as evidence of a difference between Octanorm and placebo.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the registry record reports one patient enrolled and treated, premature termination, and no efficacy or safety analyses. It gives no analyzed MMT-8 comparison between Octanorm and placebo. Consequently, there is no treatment-effect estimate to interpret and no basis in this record for concluding that the groups differed or were similar.
The primary outcome was MMT-8 at 32 weeks, but no primary outcome value or comparison is reported. A difference between groups, a confidence interval, and a p-value are not reported in the registry record. These absences are not evidence that the treatments had equal outcomes. They mean that the posted results do not quantify the treatment contrast.
In general, a confidence interval expresses the range of effect sizes compatible with the data and the statistical model, while a p-value addresses how compatible the data are with a specified null model. Neither can be interpreted without an analyzed result. With no efficacy analysis reported, the precision of a possible treatment effect cannot be assessed from this record.
The record lists 59 outcome measures and zero analyses. The presence of many planned or registered measures does not mean that each was observed, analyzed, or reported as a result. The registry names secondary outcomes including extra-muscular disease activity, muscle enzymes, health assessment, and health-related quality of life. It does not provide comparative estimates for them. The outcome list is not a substitute for results.
The adverse-event entries are counts rather than a comparative safety analysis. One Octanorm participant is listed as at risk for other adverse events and one as affected; no participant is listed as at risk in the placebo group. The serious-event counts are zero in both listed groups, and deaths are listed as zero. These counts do not establish a comparative risk or demonstrate that either treatment is safe or unsafe. The denominators are too limited for a reliable comparison, and the record does not report an efficacy or safety analysis.
The design labels describe how the trial was intended to operate, not what its results establish. Randomization and blinding can strengthen a well-conducted comparison, but they cannot overcome the absence of an analyzed comparison. The appropriate interpretation is therefore narrow: this registry record documents an early-terminated study with insufficient reported outcome analysis to estimate efficacy or compare safety between Octanorm and placebo.
9. Statistical Methods Explained
The registry identifies a randomized, double-blind, parallel-group Phase III design, but it does not report a detailed statistical analysis plan. The design description should therefore be distinguished from the analyses actually reported. The record states that efficacy and safety analyses were not performed and lists zero analyses.
What would a randomized comparison estimate?
In general, a randomized trial aims to compare outcomes among participants assigned to different study groups. The treatment effect is a contrast between group outcomes, defined according to the endpoint and analysis plan. For MMT-8, that might involve a comparison of scores or score changes, but the registry record does not report which analysis was planned. It also reports no MMT-8 outcome estimate. It is not appropriate to infer a treatment contrast from the design label alone.
Randomization is intended to balance, across groups on average, factors that could affect outcomes. Blinding can reduce the influence of expectations on care, reporting, or assessment. These are general methodological protections; they do not guarantee balance in a particular realized study or ensure that results are precise. The registry does not report the randomization sequence method, allocation concealment, or which roles were masked.
How should an endpoint estimate be read?
For a quantitative endpoint, an estimated group contrast would describe the direction and size of the observed difference under a specified analysis. A confidence interval would show the statistical uncertainty around that estimate. In general, a narrow interval indicates greater precision than a wide interval, while an interval spanning effects in different directions may leave the result uncertain. No MMT-8 contrast or confidence interval is reported in this record, so these features cannot be assessed.
A p-value is also conditional on a statistical model and a null hypothesis. It does not measure the probability that a treatment works, and it cannot substitute for an estimate of the effect or its precision. Since no efficacy analysis was performed, the registry record reports no primary comparison or corresponding p-value. The statistical significance of an unreported comparison cannot be inferred.
What does the planned sample-size illustration represent?
The stated planning assumptions describe an illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The effect to detect is framed as a stated fraction of the observed effect. In general, such calculations explore how a hypothetical future study might be sized under selected assumptions. They are planning illustrations, not observed power, and do not demonstrate that a treatment effect exists.
The registry record does not report an efficacy analysis or an observed MMT-8 treatment contrast. A sample-size illustration based on an observed effect should therefore not be read as confirmatory evidence from this trial. The record also does not report the trial's own sample-size justification, so it is not possible to establish whether the illustration corresponds to the original design rationale.
Why do outcome counts and analyses differ?
An outcome measure is an item intended to be collected or assessed; an analysis is a statistical evaluation of data. The registry lists 59 outcome measures but zero analyses. Those entries describe the breadth of registered assessments, not a set of completed comparisons. The secondary outcomes include extra-muscular disease activity, aldolase, creatine kinase, alanine aminotransferase, aspartate aminotransferase, lactate dehydrogenase, the Health Assessment Questionnaire, and the SF-36v2 Health Survey. Comparative results for these measures are not reported in the registry record.
In general, outcome analysis also depends on choices such as the population to include, the handling of missing measurements, the treatment of repeated assessments, and the model used to estimate the group contrast. These choices can affect interpretation and should be specified in an analysis plan. The detailed analysis plan and these choices are not reported in the registry record. No particular analytic method should be attributed to the trial beyond the stated planning assumptions.
Why can one observation not establish a group effect?
A treatment comparison requires information from the groups being compared. Here, the registry reports one patient enrolled and treated, while the adverse-event group entries list one at risk in the Octanorm group and none at risk in the placebo group. With no reported efficacy analysis, there is no comparative endpoint estimate. A single participant's observation, even if an endpoint value were reported, would not provide a reliable estimate of the average contrast between assigned groups. The absence of a comparative estimate is the key limitation, rather than a failure to meet a particular statistical threshold.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 59 outcome measures, including the primary MMT-8 outcome and secondary assessments of extra-muscular disease activity, muscle enzymes, physical function, and health-related quality of life. When a study evaluates multiple outcomes or multiple comparisons, the chance of at least one apparently notable result can increase. As a general statistical principle, a prespecified approach to multiplicity can help control how results across multiple tests are interpreted.
A multiplicity strategy, including whether any adjustment or hierarchy was planned, is not reported in the registry record. More importantly, the record states that efficacy and safety analyses were not performed and lists zero analyses. There are no reported outcome tests to assess for multiplicity, and no basis for selecting a favorable result from the outcome list.
The record does not describe how missing outcome data would have been handled, which analysis population was intended, or whether analyses were to follow assignment regardless of treatment received. These details are not reported in the registry record. In general, missing-data methods and analysis-population definitions can influence estimates and their interpretation, so their absence limits understanding of the planned analysis even apart from the lack of results.
The stated premature termination and enrollment of one patient treated make the lack of an analyzable comparison especially consequential. The registry does not explain the reason for termination or report whether any planned endpoint data were available for analysis. These details are not reported in the registry record. No assumption about the termination decision, missingness mechanism, or intended analysis population is warranted.
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 |
|---|---|---|---|
| Octanorm | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Octanorm | 0 of 1 |
| Placebo | 0 of 0 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Octanorm | 1 of 1 |
| Placebo | 0 of 0 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Octanorm | Placebo |
|---|---|---|---|
| Dermatomyositis | Skin and subcutaneous tissue disorders | 1 of 1 | n/a of 0 |
| Infusion site haematoma | General disorders | 1 of 1 | n/a of 0 |
| Blood lactate dehydrogenase increased | Investigations | 1 of 1 | n/a of 0 |
| Blood creatine phosphokinase increased | Investigations | 1 of 1 | n/a of 0 |
| Aldolase increased | Investigations | 1 of 1 | n/a of 0 |
The registry separates serious adverse events from other adverse events. In the Octanorm group, one participant is listed as at risk for serious adverse events and zero as affected; one is listed as at risk for other adverse events and one as affected. In the placebo group, zero participants are listed as at risk in either category, and zero as affected. Deaths are listed as zero in both groups. No serious adverse-event terms are listed.
These are registry counts, not a comparative safety analysis. Because the placebo group has no participants listed as at risk, its zero event count cannot be treated as evidence of no risk or compared meaningfully with the Octanorm count. The single other adverse event recorded for Octanorm is not characterized by a term in the record, so its nature and clinical context are not reported in the registry record.
Counts alone do not establish that an event was caused by treatment. They also do not establish that treatment is safe or unsafe. The record states that safety analyses were not performed. The event entries should therefore be read as limited descriptive reporting, not as a reliable estimate of comparative risk.
12. What the Result Does, and Does Not, Mean
The record establishes that a randomized, double-blind, placebo-controlled Phase III study of Octanorm in dermatomyositis was terminated prematurely. It reports one patient enrolled and treated and states that efficacy and safety analyses were not performed. The primary outcome was MMT-8 at 32 weeks, but no analyzed MMT-8 comparison is reported.
These registry entries do not show that Octanorm improved, worsened, or had the same effect on the primary outcome as placebo. No treatment-effect estimate, confidence interval, or primary-outcome p-value is reported. The record therefore does not support a conclusion about efficacy, including a conclusion that there was no effect. Lack of an analysis is not a null finding.
The adverse-event counts also do not establish a comparative safety profile. The registry lists one Octanorm participant at risk for other adverse events and one affected, while no placebo participants are listed at risk. Zero serious events and deaths in the listed groups do not establish absence of risk, particularly without a meaningful group comparison or the safety analysis that the record says was not performed.
General statistical interpretation depends on both the estimated effect and its uncertainty. Neither is available for the primary comparison. The registered outcome list and trial design cannot fill that gap: they describe intended assessments and methods, not analyzed results. The most defensible conclusion is limited to the study's early termination and lack of reported efficacy and safety analyses. Any further claim about treatment benefit, lack of benefit, or comparative harm would go beyond the registry record.
13. Why This Trial Matters Statistically
This trial is statistically important as an example of the difference between a planned study and an informative treatment comparison. A randomized, double-blind design can be appropriate for reducing bias, but that design only supports inference when enough usable outcome information is collected and analyzed. The registry record states that this study ended prematurely after one patient was enrolled and treated, with no efficacy or safety analyses.
The case also illustrates why outcome listings must not be mistaken for results. The record lists 59 outcome measures, including a primary muscle-strength assessment and secondary measures spanning disease activity, laboratory values, function, and health-related quality of life. Those listings indicate assessment plans; they do not yield effect estimates or establish that comparative analyses were completed.
It also highlights the distinction between a planning calculation and evidence. The stated sample-size assumptions describe an illustration under specified statistical conditions. Such calculations can support planning in a future study, but they are not observed power and cannot turn an unreported treatment effect into evidence. Here, no efficacy analysis is available from which to interpret the primary comparison.
For readers, the practical statistical lesson is to look for the analyzed contrast, its uncertainty, and the population and methods used—not only the phase, allocation, masking, or endpoint labels. When those results are absent, the correct interpretation is uncertainty due to lack of an analyzable comparison, rather than evidence favoring either treatment.
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 principal limitation is the trial's premature termination after one patient was enrolled and treated. The registry explicitly states that efficacy and safety analyses were not performed. As a result, the primary outcome has no reported comparative estimate, and the record cannot support an efficacy conclusion.
The primary outcome entry gives the MMT-8 assessment and its 32-week timeframe but no outcome value, change, or treatment contrast. The detailed statistical analysis plan, sample-size justification, and rationale for termination are not reported in the registry record. The randomization procedure, allocation concealment, and specific masking roles are also not reported in the registry record.
Although 59 outcome measures are listed, the record lists zero analyses. It does not report the intended analysis population, missing-data methods, or a multiplicity strategy. These omissions limit assessment of how the planned outcomes would have been analyzed. They do not justify assuming a particular method or treating registered outcomes as completed results.
The safety entries are also limited: the placebo group has no participants listed at risk, and the single other adverse event listed for Octanorm is not described by a term. Serious-event and death counts are not a substitute for a comparative safety analysis. Together, the lack of outcome analysis and limited group information prevent reliable estimates of efficacy or comparative safety from this record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis — NCT03686969.
- Registry record last updated 2021-04-23; status last verified 2021-04.
- Results first posted 2021-04-23.
- 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 describes NCT03686969 as a randomized, double-blind, placebo-controlled Phase III parallel-group study of Octanorm in patients with dermatomyositis. The active intervention was subcutaneous human immunoglobulin, described as 0.5g/kg/week octanorm 16.5%; placebo was the comparator. MMT-8 at 32 weeks was the primary outcome.
The record lists the study as terminated and states that it ended prematurely after one patient was enrolled and treated. Efficacy and safety analyses were not performed. It lists 59 outcome measures and zero analyses. The adverse-event entries include no serious events or deaths, but they do not provide a comparative safety analysis.
Accordingly, the registry record documents the trial design and limited event counts, but no analyzed efficacy comparison or reliable comparative safety estimate.