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 Open-label Safety Extension Study Assessing Safety and Tolerability of LAI in Patients Who Participated in Study INS-212 (NCT02628600), a non-randomized phase 3 study. The primary outcome is Number of Participants With Treatment-Emergent Adverse Events (TEAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02628600 |
| Phase | Phase 3 |
| Condition | NTM Lung Infection Due to MAC |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 163 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Insmed Incorporated |
| Results posted | Yes |
2. Clinical Question
Population
Adults with nontuberculous mycobacterial lung infections due to Mycobacterium avium complex (MAC) who were refractory to therapy and had failed to convert in Study INS-212 (NCT02344004). The primary endpoint uses the safety population. Further eligibility details are not reported in the registry record.
Intervention
Once-daily LAI 590 mg added to a multidrug regimen. The registry describes this as an open-label safety extension. All participants in the extension received LAI with a multidrug regimen.
Comparator
There was no concurrent untreated or randomized control group in the extension. Participants are described according to whether, in Study INS-212, they had received LAI with a multidrug regimen or a multidrug regimen alone. These are prior-treatment groups, not contrasting assigned treatments in the extension.
Primary question
How many participants experienced treatment-emergent adverse events during the period from baseline to 28 days after the end of treatment, up to 13 months? The registry defines these events by their timing relative to study medication in INS-312. The extension is described as assessing safety and tolerability.
The clinical question is primarily descriptive: it concerns adverse events during exposure to LAI plus a multidrug regimen in a selected group of participants continuing from an earlier study. The posted record also lists culture conversion by Month 6 and Month 12, time to culture conversion, and change in 6MWT distance from baseline to Month 6 and Month 12 as secondary outcomes. Those outcomes address clinical status, but they do not change the central limitation of the safety question: every participant in the extension received the same regimen.
Because treatment assignment in the extension was not randomized and there was no concurrent regimen-only group, differences between the prior-treatment groups cannot be interpreted as effects caused by treatment during the extension. The history of treatment in INS-212 distinguishes the groups, but does not supply a like-for-like control for the extension regimen. Any comparison between them should therefore be read as a descriptive comparison of groups with different prior treatment histories.
3. Trial Design
This was a Phase 3, open-label, non-randomized safety extension with a parallel-group structure. The registry lists enrollment of 163 and identifies Insmed Incorporated as the sponsor. The study started on 2016-02-05, reached primary completion on 2018-10-17, and has status COMPLETED.
The extension followed Study INS-212, whose participants had refractory NTM lung infection due to MAC and had failed to convert. The registry describes two groups based on prior INS-212 treatment: participants who had received LAI plus a multidrug regimen, and participants who had received a multidrug regimen alone. In the extension, all participants received LAI plus a multidrug regimen. Thus, the prior-treatment labels describe study history, not the treatments being compared during the extension.
“Open-label” means treatment was not masked according to the registry. “Non-randomized” means the extension did not randomly allocate participants to the prior-treatment groups. In general, random allocation helps balance measured and unmeasured characteristics between treatment groups; without it, groups may differ for reasons that also relate to outcomes. The registry does not report the reasons participants entered one prior-treatment group rather than the other, or how the groups were formed beyond their previous treatment in INS-212.
The record identifies LAI 590 mg and a multidrug regimen as the interventions. Details of the multidrug regimen, treatment adherence, dose changes, and individual duration of exposure are not reported in the registry record. The primary endpoint timeframe is described as baseline to 28 days after the end of treatment, up to 13 months. The record does not specify further operational details about follow-up or how exposure varied among participants.
This design is suited to describing safety events during the extension among participants who continued from the earlier study. It is not a randomized comparison of LAI plus a multidrug regimen against a multidrug regimen alone in the extension. The prior-treatment groups may provide context about the enrolled participants, but they do not isolate the effect of the extension regimen from prior treatment history or other differences between groups.
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: Participants in Study INS-212 had either received 590 mg LAI plus an MDR (LAI + MDR arm) or a multidrug regimen alone (MDR alone arm). All participants in this safety extension study, INS-312, were to continue the multidrug antimycobacterial regimen that they were receiving during Study INS-212 and received LAI 590 mg QD for up to 12 months.
Overall Study
| Stage | Prior LAI + Multidrug Regimen | Prior Multidrug Regimen Alone |
|---|---|---|
| STARTED | 73 | 90 |
| Participant Discontinued the Study | 24 | 32 |
| COMPLETED | 49 | 58 |
| NOT COMPLETED | 24 | 32 |
Reasons for not completing this period:
| Reason | Prior LAI + Multidrug Regimen | Prior Multidrug Regimen Alone |
|---|---|---|
| Death | 2 | 1 |
| Adverse Event | 3 | 20 |
| Protocol Violation | 0 | 1 |
| Lack of Efficacy | 2 | 0 |
| Withdrawal by Subject | 13 | 8 |
| Lost to Follow-up | 1 | 0 |
| Physician Decision | 2 | 2 |
| Other not specified | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety 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.
Population: Safety population, defined as participants who received at least 1 dose of LAI.
| Characteristic | Unit | Prior LAI + Multidrug Regimen | Prior Multidrug Regimen Alone | Total |
|---|---|---|---|---|
| Participants analysed | 73 | 90 | 163 | |
| Age, Continuous | years | 64.9 (SD 9.12) | 64.8 (SD 10.33) | 64.8 (SD 9.78) |
| Sex: Female, Male: Female | Participants | 51 | 54 | 105 |
| Sex: Female, Male: Male | Participants | 22 | 36 | 58 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 28 | 22 | 50 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 | 3 | 5 |
| Race (NIH/OMB): White | Participants | 41 | 60 | 101 |
| Race (NIH/OMB): More than one race | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 5 | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment-Emergent Adverse Events (TEAEs), measured over From baseline to 28 days after end of treatment, up to 13 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with treatment-emergent adverse events (TEAEs). It measures a count of participants who experienced at least one event meeting the registry definition; it is not a count of all event episodes. The registry defines TEAEs as adverse events occurring on or after the first dose of study medication in INS-312 and within 28 days after the last dose. If a partial onset date made it impossible to determine whether an event was treatment emergent, the event was classified as treatment emergent.
The timeframe is from baseline to 28 days after the end of treatment, up to 13 months, and the stated population is the safety population. A safety endpoint of this kind is used to describe the occurrence of adverse events during and shortly after treatment exposure. It can help identify the types and frequency of reported events in the participants observed, while the specified timing rule determines which events are counted as treatment emergent.
The endpoint does not, by itself, establish that treatment caused an event. Events occurring during treatment can have other explanations, and an open-label extension without a concurrent control group cannot distinguish treatment-related risk from background illness, prior treatment, or other influences. The registry record identifies the endpoint and its definition, but does not report an overall primary-endpoint result in the registry record for this interpretation. Serious and other adverse-event counts are listed separately and should not be mistaken for a reported total count of all TEAEs.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted adverse-event counts are descriptive. For the prior LAI plus multidrug regimen group, 20 of 73 participants are recorded as affected by serious adverse events, and 45 of 73 are recorded in the other adverse-event category. For the prior multidrug regimen alone group, the corresponding counts are 32 of 90 and 75 of 90. The registry also records 2 deaths in the first group and 4 in the second. These values describe the groups as labeled by their treatment in Study INS-212; in the extension, both groups received LAI plus a multidrug regimen.
These counts are not a randomized treatment contrast. The group labels reflect prior treatment, and the registry states that allocation was non-randomized. It would therefore be inappropriate to treat a difference in the observed counts as an estimate of what would have happened if otherwise comparable participants had received different extension treatments. No adjusted analysis or causal estimate is reported in the registry record.
The adverse-event listing includes Mycobacterium avium complex infection (5.6%), pneumonia (4.4%), chronic obstructive pulmonary disease (4.4%), infective exacerbation of bronchiectasis (3.3%), cataract (2.7%), and hip fracture (2.7%) among the top serious terms. The registry record does not identify the denominator used for these percentages in the registry entries, so they should be read as listed rather than converted into participant counts or compared with the group counts.
Precision cannot be judged from the counts alone. The registry record does not report confidence intervals or other uncertainty measures for these adverse-event summaries. In general, an estimate based on fewer observed participants is less precise than an estimate based on more observations, but no interval can be inferred without the analysis details. The recorded enrollment of 163 is not evidence that every enrolled participant contributed to every endpoint; the endpoint-specific analysis denominators and missing outcome handling are not reported in the registry record.
The primary endpoint is described as the number of participants with TEAEs, but the registry entries available list serious and other adverse-event counts rather than a distinct overall TEAE count. The registry record does not report a primary-endpoint result or a statistical analysis for it. It is therefore not possible to characterize the overall TEAE count, estimate a treatment contrast, or assess statistical significance from these registry entries.
In general, statistical significance and clinical importance are different questions. A significance test concerns compatibility of data with a specified statistical model, while clinical importance concerns the meaning of an effect in context. Neither can be assessed for the primary endpoint without a reported estimate, an uncertainty measure, and an appropriate analysis. This extension is most appropriately read as a registry description of safety events among treated participants, not as evidence from a controlled comparative efficacy analysis.
9. Statistical Methods Explained
What does a participant count represent?
The primary outcome is a count of participants with at least one treatment-emergent adverse event, rather than the total number of event episodes. This distinction matters because one participant can experience more than one event, while a participant-level count records that person once for the endpoint. The registry defines the event window by the timing of the event relative to study medication in INS-312. When the onset date was partial and treatment-emergent status could not be determined, the event was classified as treatment emergent.
The adverse-event information also separates serious events from other adverse events. These categories describe different aspects of safety reporting and should not be conflated with the primary endpoint unless the registry explicitly reports them as its result. The registry entries available do not give a separate overall TEAE result, so the serious and other category counts do not supply that missing result.
Why does the non-randomized design matter?
In general, randomization makes treatment assignment independent of participant characteristics at the point of allocation, apart from chance. This helps support a causal comparison when groups receive different treatments. Here, the groups are defined by treatment received in Study INS-212, while everyone in the extension received LAI plus a multidrug regimen. Differences between the groups may reflect prior treatment, disease history, selection into the extension, or other characteristics. The registry record does not report an adjustment strategy that would address such differences.
Consequently, group-specific counts are useful for describing events in the observed participants, but they are not estimates of the causal effect of adding LAI during the extension. The design does not include a concurrent group continuing on multidrug regimen alone, and the listed prior-treatment groups do not serve as an equivalent substitute for such a control.
What can uncertainty measures add?
General statistical explanation: a confidence interval expresses the range of values reasonably compatible with an estimate under a particular model and sampling procedure. It can show whether a summary is precise or remains uncertain. Counts alone do not communicate that uncertainty fully. No confidence intervals for the adverse-event summaries or primary endpoint are reported in the registry record, so their precision cannot be quantified from the registry entries.
For event counts, inference also depends on the definition of the analysis population, the time at risk, and how participants with incomplete follow-up are handled. If exposure or observation time differs among participants, raw counts may not be directly comparable as rates. The registry lists participant counts and timeframes, but exposure-adjusted rates and detailed follow-up information are not reported in the registry record.
What does the sample-size illustration mean?
The record describes sample-size planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect; time-to-event illustrations use a total event count, while other illustrations refer to participants per arm. These are planning illustrations, not observed power and not evidence that the study was designed with those assumptions. The registry does not report the trial's own sample-size justification in the registry record.
In general, a planning calculation asks what sample size might be needed to detect a chosen effect under specified assumptions. Its answer depends on the assumed effect, variability or event frequency, allocation, significance level, and statistical model. It does not measure the probability that a completed study has established a treatment effect. The numeric outputs of the planning illustrations are not included in the registry entries for this interpretation, so no planning sample size can be stated.
Why are analysis details important?
The record lists four outcome measures and zero analyses. It therefore does not provide model specifications, estimates, standard errors, confidence intervals, p-values, covariate adjustments, or outcome-specific denominators in the registry record. For binary outcomes such as culture conversion, an analysis would need to state how the event and analysis population were defined and how missing status was treated. For time to culture conversion, methods would need to account for censoring and specify how time was measured. For change in 6MWT distance, the analysis would need to define the baseline and follow-up measurements and the approach to incomplete observations. These are general considerations; the registry record does not report the methods used for these outcomes.
Because the study is open-label, knowledge of treatment may also influence event reporting or assessment. This does not show that reporting was biased, but it is a design consideration when interpreting subjective or investigator-assessed outcomes. The registry record does not report any masking or independent adjudication procedures beyond masking NONE.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists the primary TEAE endpoint and secondary outcomes covering culture conversion by Month 6 and Month 12, time to culture conversion, and change in 6MWT distance from baseline to Month 6 and Month 12. When a study evaluates multiple outcomes or time points, the chance of at least one apparently notable result can increase if each is tested separately without an appropriate plan. General approaches to address this include prespecifying a hierarchy or adjusting inference for multiple tests. The registry record does not report a multiplicity strategy, and it lists zero analyses, so no adjusted or unadjusted inferential results are available in the registry record for these outcomes.
Missing-data handling is not reported in the registry record. The record does not state how participants with incomplete follow-up, missing culture results, unavailable 6MWT measurements, or uncertain endpoint status were handled in outcome analyses. For the primary safety endpoint, the definition addresses partial adverse-event onset dates by classifying the event as treatment emergent when status could not be determined; it does not describe how missing follow-up or other incomplete safety information was treated.
The primary endpoint specifies the safety population, but the registry record does not provide a detailed population definition or endpoint-specific denominators. It does not report the analysis populations for the secondary outcomes. Enrollment of 163 should not be assumed to equal the number contributing data to every endpoint. Without analysis populations and missingness details, readers cannot determine whether endpoint summaries use all enrolled participants, only those with available measurements, or another subset.
These omissions limit assessment of bias and precision. In general, missingness can matter when the probability of having an observed outcome is related to health status or treatment experience. A statistical method may address some forms of incomplete data under assumptions, but those assumptions and methods must be reported to evaluate them. Such methods 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 |
|---|---|---|---|
| Prior LAI + Multidrug Regimen | 20 | 73 | 27.4% |
| Prior Multidrug Regimen Alone | 32 | 90 | 35.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Prior LAI + Multidrug Regimen | 2 of 73 |
| Prior Multidrug Regimen Alone | 4 of 90 |
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 |
|---|---|
| Prior LAI + Multidrug Regimen | 45 of 73 |
| Prior Multidrug Regimen Alone | 75 of 90 |
Most frequent serious adverse event terms
| Event term | Organ system | Prior LAI + Multidrug Regimen | Prior Multidrug Regimen Alone |
|---|---|---|---|
| Mycobacterium avium complex infection | Infections and infestations | 2 of 73 | 5 of 90 |
| Pneumonia | Infections and infestations | 3 of 73 | 4 of 90 |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | 2 of 73 | 4 of 90 |
| Infective exacerbation of bronchiectasis | Infections and infestations | 1 of 73 | 3 of 90 |
| Atrial fibrillation | Cardiac disorders | 1 of 73 | 2 of 90 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 1 of 73 | 2 of 90 |
| Cataract | Eye disorders | 2 of 73 | 0 of 90 |
| Hip fracture | Injury, poisoning and procedural complications | 2 of 73 | 0 of 90 |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | 0 of 73 | 2 of 90 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 0 of 73 | 2 of 90 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Prior LAI + Multidrug Regimen | Prior Multidrug Regimen Alone |
|---|---|---|---|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | 5 of 73 | 39 of 90 |
| Cough | Respiratory, thoracic and mediastinal disorders | 9 of 73 | 32 of 90 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 9 of 73 | 16 of 90 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 10 of 73 | 9 of 90 |
| Nasopharyngitis | Infections and infestations | 10 of 73 | 7 of 90 |
| Fatigue | General disorders | 3 of 73 | 13 of 90 |
| Infective exacerbation of bronchiectasis | Infections and infestations | 6 of 73 | 10 of 90 |
| Weight decreased | Investigations | 7 of 73 | 8 of 90 |
| Nausea | Gastrointestinal disorders | 5 of 73 | 9 of 90 |
| Diarrhoea | Gastrointestinal disorders | 4 of 73 | 9 of 90 |
Serious events with the highest arm-level rate: mycobacterium avium complex infection (5.6%), pneumonia (4.4%), chronic obstructive pulmonary disease (4.4%), infective exacerbation of bronchiectasis (3.3%), cataract (2.7%).
The safety listing records serious adverse events for the prior LAI plus multidrug regimen group as 20 affected participants among 73 at risk, and for the prior multidrug regimen alone group as 32 among 90 at risk. Other adverse events are listed as 45 among 73 and 75 among 90, respectively. Deaths are recorded as 2 and 4 in those groups. These are counts in the registry record, not causal comparisons.
The group names refer to treatment in Study INS-212. During the extension, all participants received LAI plus a multidrug regimen. Since the study was non-randomized and had no concurrent extension group receiving only a multidrug regimen, the counts cannot establish that one prior treatment history led to more or fewer events during the extension.
The serious-event terms listed include Mycobacterium avium complex infection (5.6%), pneumonia (4.4%), chronic obstructive pulmonary disease (4.4%), infective exacerbation of bronchiectasis (3.3%), cataract (2.7%), and hip fracture (2.7%). The registry entries do not specify the denominator for these listed percentages, so they should not be translated into counts. They are event-term summaries, not estimates of treatment-attributable risk.
In general, safety counts are easier to interpret when accompanied by clear definitions, observation time, denominators, and uncertainty measures. The registry record gives some denominator counts but does not report confidence intervals, exposure-adjusted rates, or a detailed analysis of these events. The primary endpoint's overall TEAE result is also not reported in the registry entries.
12. What the Result Does, and Does Not, Mean
The registry record establishes the setting and treatment exposure: an open-label safety extension for participants with refractory MAC lung infection who had not converted in Study INS-212, with LAI 590 mg added to a multidrug regimen for all participants. It also records serious and other adverse-event counts, deaths, and a list of serious event terms. These data describe what was recorded in the study groups; they do not by themselves determine why an event occurred.
The record does not establish comparative safety between LAI plus multidrug regimen and multidrug regimen alone during the extension, because the latter regimen was not a concurrent extension treatment group. Prior treatment labels are not a substitute for random assignment. The counts likewise cannot establish whether treatment caused an event, whether the regimen changed the underlying course of MAC infection, or whether it affected functional status.
Although culture conversion, time to conversion, and change in 6MWT distance appear as secondary outcomes, the registry entries do not include their results or analysis details. No efficacy conclusion can be drawn from their presence in the outcome list. The primary TEAE endpoint is defined, but its overall result is not reported in the registry record for this interpretation. Nor are confidence intervals, statistical tests, or adjusted estimates reported.
In general, a trial result is most informative when its estimate, uncertainty, analysis population, and design limitations are considered together. Here, the non-randomized extension design supports a descriptive reading of events among participants receiving LAI with a multidrug regimen. It does not provide the controlled contrast needed to attribute differences to a treatment choice. The fact that the registry status is COMPLETED indicates study status, not that a treatment benefit or safety conclusion has been established.
The findings should therefore be read narrowly: they are registry-recorded safety observations in a selected extension population, alongside an outcome plan that includes microbiologic and functional measures. Missing statistical results and analysis details limit what can be concluded about magnitude, precision, and comparisons. These data do not support medical advice for an individual or a claim that treatment does or does not work.
13. Why This Trial Matters Statistically
This study matters statistically because its treatment history can easily be mistaken for a treatment comparison. The arm descriptions name prior LAI plus multidrug regimen and prior multidrug regimen alone, but both groups received LAI plus a multidrug regimen in the extension. Recognizing the distinction prevents the group labels from being read as if the extension randomized participants between different treatments.
It also illustrates why a safety extension answers a different question from a controlled efficacy trial. A safety extension can describe events observed during additional exposure in participants who continue from an earlier study. Without a concurrent control and random allocation, it cannot separate events related to the regimen from events associated with underlying illness, prior treatment, or other differences among participants. That limitation applies even when event counts are presented by group.
The registry's event definitions and timeframe are important for interpretation. The primary endpoint counts participants with treatment-emergent adverse events and specifies a window around study medication exposure. The adverse-event listings separate serious from other events, while also recording deaths and selected serious terms. Keeping those measures distinct avoids treating different summaries as interchangeable.
Finally, the record shows the importance of complete statistical reporting. A stated endpoint is not the same as a reported endpoint result. The registry entries do not include a primary TEAE result, analysis estimates, uncertainty measures, multiplicity handling, or missing-data methods. Without those details, statistical precision and inferential conclusions cannot be assessed. The study can still provide descriptive safety context, but its design and reporting determine how far that context can be taken.
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 trial-specific limitation is the absence of a concurrent control group in the extension. All participants received LAI plus a multidrug regimen, so the registry does not offer a randomized comparison against continued multidrug regimen alone. The groups are categorized by their prior treatment in Study INS-212, and treatment history may be associated with other differences. The registry record does not report adjustment methods that would account for such differences.
The selected population also limits interpretation. Participants had refractory MAC lung infection, had participated in INS-212, and had failed to convert in that study. These registry entries define the extension population; they do not show how the results would apply to people outside that setting. Further eligibility criteria and reasons for entering or remaining in the extension are not reported in the registry record.
The study was open-label. In general, lack of masking may influence event recognition, reporting, or assessment, although the registry entries do not establish that this occurred. Independent event adjudication and detailed assessment procedures are not reported in the registry record.
Reporting gaps further constrain interpretation. The registry entries include serious and other adverse-event counts, deaths, and selected serious-event terms, but do not include a distinct overall primary TEAE result, confidence intervals, or statistical comparisons. The denominator for the listed serious-term percentages is not identified in the registry record. Endpoint-specific analysis populations, exposure-adjusted event rates, missing-data handling, multiplicity methods, and detailed secondary-outcome analyses are not reported in the registry record.
Finally, enrollment is recorded as 163, but that figure does not establish the number observed for each outcome or the completeness of follow-up. No conclusion about precision can be made from enrollment alone. These limitations favor a descriptive interpretation of the recorded safety observations rather than a causal or comparative claim.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Open-label Safety Extension Study Assessing Safety and Tolerability of LAI in Patients Who Participated in Study INS-212 — NCT02628600.
- Registry record last updated 2020-02-10; status last verified 2020-01.
- Results first posted 2019-11-19.
- 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
NCT02628600 is a completed Phase 3, open-label, non-randomized safety extension sponsored by Insmed Incorporated. The study enrolled 163 participants and evaluated LAI 590 mg added to a multidrug regimen in participants with refractory NTM lung infection due to MAC who had failed to convert in Study INS-212. The registry records a start date of 2016-02-05 and primary completion on 2018-10-17.
Participants were grouped according to their prior treatment in INS-212, but all received LAI plus a multidrug regimen in the extension. The primary endpoint was the number of participants with treatment-emergent adverse events in the safety population, over the stated timeframe from baseline to 28 days after the end of treatment, up to 13 months. The registry lists serious and other adverse-event counts, deaths, and selected serious-event terms. It also lists culture conversion, time to conversion, and 6MWT change as secondary outcomes.
The registry entries available do not report the overall primary endpoint result, statistical analyses, confidence intervals, missing-data methods, or multiplicity handling. Since the study was non-randomized and all extension participants received the same regimen, the adverse-event counts are descriptive and do not provide a controlled treatment comparison.