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 Liposomal Amikacin for Inhalation (LAI) for Nontuberculous Mycobacteria (NCT01315236), a randomized phase 2 study. The primary outcome is Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01315236 |
| Phase | Phase 2 |
| Condition | Mycobacterium Infections, Nontuberculous |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 90 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Insmed Incorporated |
| Results posted | Yes |
2. Clinical Question
Population
Patients with treatment-refractory nontuberculous mycobacterial lung disease. The primary endpoint population is identified as mITT; the registry record does not define that abbreviation or report the population count for the primary analysis.
Intervention
Liposomal amikacin for inhalation (LAI), at 590 mg once daily, during the 84-day double-blind phase.
Comparator
Placebo once daily during the 84-day double-blind phase.
Primary question
How did semi-quantitative mycobacterial culture results change from baseline to Day 84 in the LAI and placebo groups? The registry record defines the endpoint and scale, but does not report the arm-level results or a numerical comparison.
The trial was designed to evaluate efficacy, safety and tolerability of daily LAI versus placebo in patients with recalcitrant disease. The primary outcome concerns culture growth, not a direct measure of symptoms or health status. The registry also lists secondary outcomes involving negative culture, time to negative culture, symptom scores, global health ratings and use of rescue drugs. These outcomes address different aspects of the study and should not be treated as substitutes for the primary endpoint.
The primary question is therefore a comparison of change on a culture scale over the double-blind treatment period. The record explains that negative changes correspond to reduced colony growth, while higher scale steps represent more growth. It does not report the observed changes in either arm, how participants were distributed across response categories, or a statistical estimate comparing the groups. The statistical evidence for a difference on this endpoint cannot be determined from the registry results described in the record.
3. Trial Design
This was a phase 2, randomized, double-blind, placebo-controlled, parallel-group study. The registry reports quadruple masking, but does not identify the specific roles that were masked. Enrollment was 90. The study status is completed. The recorded start date is 2012-04-19, and the primary completion date is 2014-08-18.
The study evaluated 590 mg of LAI once daily against placebo once daily. The main double-blind phase lasted 84 days. Its purpose, as stated in the registry, was to evaluate efficacy, safety and tolerability in patients with treatment-refractory nontuberculous mycobacterial lung disease. The primary endpoint was assessed from baseline to the end of that phase, identified as Day 84.
Random allocation is intended to make treatment groups comparable on average with respect to factors that could affect outcomes. Double blinding is intended to limit the influence of knowledge of assigned treatment on participant experience, care and outcome assessment. These are general features of trial design; the registry record does not report how randomization was generated, how assignments were concealed, or which study roles were masked.
The registry also contains safety groups labelled as double-blind and open-label. It reports safety counts for LAI and placebo in each of those settings. The available record does not describe the transition between the phases or provide enough detail to interpret the open-label groups as part of the randomized primary comparison. The double-blind phase and its primary endpoint are the focus of the stated treatment comparison.
The registry does not report the protocol’s sample-size justification, recruitment targets by arm, planned interim analyses, or a full statistical analysis plan. Nor does the recorded enrollment figure establish how many participants contributed data to the primary endpoint. Those details are not reported in the registry record.
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.
Double-blind Phase
| Stage | LAI 590 mg QD | Placebo |
|---|---|---|
| STARTED | 44 | 45 |
| COMPLETED | 40 | 45 |
| NOT COMPLETED | 4 | 0 |
Reasons for not completing this period:
| Reason | LAI 590 mg QD | Placebo |
|---|---|---|
| Death | 1 | 0 |
| Adverse Event | 1 | 0 |
| Withdrawal of Consent | 1 | 0 |
| Lost to Follow-up | 1 | 0 |
Open-label Phase
| Stage | LAI 590 mg QD | Placebo |
|---|---|---|
| STARTED | 35 | 43 |
| COMPLETED | 34 | 41 |
| NOT COMPLETED | 1 | 2 |
Reasons for not completing this period:
| Reason | LAI 590 mg QD | Placebo |
|---|---|---|
| Death | 1 | 0 |
| Adverse Event | 0 | 1 |
| Other | 0 | 1 |
12-Month Follow-up Period
| Stage | LAI 590 mg QD | Placebo |
|---|---|---|
| STARTED | 27 | 32 |
| COMPLETED | 26 | 31 |
| NOT COMPLETED | 1 | 1 |
Reasons for not completing this period:
| Reason | LAI 590 mg QD | Placebo |
|---|---|---|
| Death | 0 | 1 |
| Did not return to the site for Month 12 | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: mITT
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: Double-blind Phase
| Characteristic | Unit | LAI 590 mg QD | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 44 | 45 | 89 | |
| Age, Continuous | years | 58.0 (SD 16.61) | 59.1 (SD 15.20) | 58.5 (SD 15.83) |
| Sex: Female, Male: Female | Participants | 38 | 40 | 78 |
| Sex: Female, Male: Male | Participants | 6 | 5 | 11 |
| Race/Ethnicity, Customized: White (not of Hispanic origin) | Participants | 42 | 40 | 82 |
| Race/Ethnicity, Customized: Hispanic | Participants | 0 | 2 | 2 |
| Race/Ethnicity, Customized: African | Participants | 0 | 1 | 1 |
| Race/Ethnicity, Customized: Asian | Participants | 2 | 2 | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84., measured over Baseline and end of double-blind phase of 84 days.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was change in semi-quantitative mycobacterial culture results from baseline to Day 84. The registry describes the result using a 7-step semi-quantitative scale for cultures assessed in solid and liquid growth media. Step 1 is culture negative in both media, and step 2 is growth in liquid medium only. The remaining steps reflect progressively greater growth in solid medium, with growth in liquid medium also specified for steps 4 through 7. The full scale runs from 1, the best score, to 7, the worst score.
The change in step represents the Day 84 culture result compared with baseline. The record states that negative values represent a reduction in colony growth. Thus, the endpoint keeps the direction of change meaningful: a lower scale step is associated with less observed culture growth, while a higher step reflects more growth. This is a microbiological measure and does not directly measure symptoms, function or quality of life.
A semi-quantitative scale can describe gradations in culture growth when a simple positive-versus-negative classification would omit distinctions between levels of growth. As a general statistical point, an ordinal scale ranks categories but does not guarantee that the distance between adjacent categories is equal. The registry record does not report the statistical method used to compare changes or category distributions.
The endpoint is specified for the mITT population, but the record does not define that population, report its size, or give numerical change results by treatment group. It also does not report an effect estimate or uncertainty interval for the primary comparison. The endpoint definition alone cannot show the observed treatment-group difference.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the trial’s primary endpoint, its measurement scale and the population label used for the analysis. It does not report the numerical primary results. In particular, arm-level changes, counts at each scale step, an estimated between-group difference, a confidence interval and a p-value are not reported in the registry record. Consequently, the direction and precision of the primary comparison cannot be assessed from this record.
The meaning of an individual scale change is described: negative values represent reduction in colony growth, and lower scale steps are better on the stated scale. That definition does not reveal how many participants had negative, zero or positive changes, nor whether the distributions differed between treatment groups. A direction rule is not a treatment-effect estimate.
The registry lists several secondary outcomes, including negative culture at Day 84, time to negative culture during the 84-day double-blind phase, ordinal response, changes in symptom and global health scores, and rescue-drug use. Their inclusion indicates that the study tracked multiple outcome dimensions. Numerical results for those comparisons are not reported in the registry record described in the registry record, so they cannot fill the gap in the primary result.
Statistical precision concerns how uncertain an estimated effect is, not simply whether a trial enrolled participants. A confidence interval would describe a range of estimates compatible with the data and statistical model; no such interval for the primary endpoint is reported in the registry record. Without it, the precision of any primary comparison cannot be judged. The record also does not report a test statistic or p-value that could be interpreted as evidence against a no-difference model.
Enrollment was 90, but that total should not be mistaken for the number analyzed on the primary endpoint. The registry labels the population mITT without giving its size or the treatment-group denominators for the endpoint. The safety risk counts are presented separately and do not establish the primary endpoint analysis population.
Overall, the posted information supports a description of the question and outcome measurement, but not a conclusion about the observed comparative result. The absence of a reported estimate is not evidence that the groups were identical, and it is not evidence of a difference. The primary result is not reported in the registry record.
9. Statistical Methods Explained
What does the scale measure? The primary outcome uses an ordered seven-step scale for semi-quantitative culture results. In general, ordinal measurements place observations into categories with a meaningful order, while the distance between one category and the next need not be equal. Here, step 1 is designated the best score and step 7 the worst. The registry describes the culture findings represented by each step, including growth in liquid medium and increasing colony counts in solid medium. This makes the scale a graded description of culture growth rather than a direct count of symptoms or a general health measure.
How should change be read? The endpoint compares the Day 84 step with the baseline step. The record states that negative values represent reduced colony growth. As a general principle, change scores express movement relative to a starting measurement, so their interpretation depends on the scale’s direction and the way change is calculated. The registry does not give the formula used to calculate the change or the summary statistic used to compare groups. The endpoint description therefore clarifies direction but does not specify the analysis.
Why is the comparison between groups important? A change within one group does not by itself describe the comparative treatment effect. In general, a randomized comparison evaluates how outcomes differ between assigned groups, with the comparison interpreted in light of variability and uncertainty. Changes can occur in both groups over time, including changes unrelated to the assigned treatment. The registry record does not provide the group-level primary results or the statistical estimate of their contrast, so the comparative effect cannot be reconstructed from the endpoint definition.
What does a confidence interval convey? In general, a confidence interval communicates statistical uncertainty around an estimate under the assumptions of the analysis. A narrower interval indicates greater precision than a wider interval, while the location of the interval helps show which values are compatible with the data and method. It is not a guarantee that the true value lies within a particular calculated interval. No confidence interval for the primary outcome is reported in the registry record, so the precision of the primary result is unavailable.
What does a p-value convey? In general, a p-value describes how unusual data at least as incompatible with a specified null model would be, if that model and the assumptions used for the test were correct. It is not the probability that a treatment works, nor does it measure the size or clinical importance of an effect. The registry record does not report a primary-endpoint p-value or identify the test used. A significance threshold or test procedure cannot be inferred from the trial’s phase or design.
Why can over-dispersion matter? In general, over-dispersion occurs when observed variability is greater than a statistical model assumes. If a model underestimates variability, its standard errors and uncertainty intervals may be too small, and tests may appear more conclusive than warranted. This is a general issue for statistical modelling, not a finding reported for this trial. The registry does not identify a model for the primary outcome or report a dispersion assessment.
What does the mITT label tell the reader? The primary population is labelled mITT. In general, analysis-population labels indicate which participants are included under a stated analysis definition, but the meaning of a modified intention-to-treat population depends on the protocol’s specific rules. The registry record does not define mITT, specify exclusions, report the analyzed number, or explain how participants with missing endpoint measurements were handled. The label alone is not enough to establish which randomized participants contributed to the reported endpoint.
How should sample-size illustrations be understood? In general, a sample-size calculation uses assumptions about the effect to detect, statistical significance level, allocation and analysis method to illustrate a planning requirement. Such an illustration is not an observed outcome and is not observed power. The registry’s stated illustration assumptions refer to a two-sided significance level of 0.05, equal allocation, a normal approximation and an effect expressed as a fraction of the observed effect. Those assumptions are described as a planning illustration, not as the trial’s own design justification. The registry record does not report the protocol’s sample-size justification, and no numerical illustration is available in the record details.
The primary result is not reduced to one comparison because the outcome is represented by several categories or time points. In general, collapsing an ordinal outcome into a single summary can lose information or impose assumptions about category spacing. The registry record does not identify the chosen method for handling the endpoint’s categories or provide the results needed to examine alternative summaries. Statistical methods beyond the registry entries stated in the record are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple secondary outcomes alongside the primary culture endpoint. These include culture status at Day 84, time to negative culture during the double-blind period, an ordinal culture response, symptom and global health score changes, and rescue-drug use. Testing or estimating several outcomes raises a general multiplicity issue: when many questions are evaluated, the chance of at least one apparently notable finding can increase if each is considered independently. This is a general statistical concern, not a claim about the trial’s analysis.
The registry record does not report whether the investigators adjusted for multiplicity, specified a hierarchy among outcomes, or treated secondary analyses as exploratory. The statistical plan for multiple comparisons is not reported in the registry record. No primary comparison result is reported, and the endpoint is not reduced to one comparison because it has several categories or time points. Therefore, neither the primary nor secondary inferential results can be interpreted from numerical estimates in this record.
The primary endpoint population is labelled mITT. The registry does not define this analysis population, state its size, describe which participants were excluded, or report how missing baseline or Day 84 cultures were handled. Missing-data methods, including any imputation or sensitivity analyses, are not reported in the registry record. These details matter because the amount and pattern of missing outcome data can affect both the analysis population and the interpretation of estimates.
The record also does not report analysis populations for the secondary outcomes or identify whether all randomized participants were included in any particular analysis. Enrollment of 90 does not, by itself, establish the number contributing to each endpoint. These omissions limit assessment of the analysis denominators, missing-data assumptions and multiplicity strategy.
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 |
|---|---|---|---|
| LAI 590 mg QD - Double Blind | 8 | 44 | 18.2% |
| Placebo - Double Blind | 4 | 45 | 8.9% |
| LAI 590 mg QD - Open Label | 5 | 35 | 14.3% |
| Placebo - Open Label | 5 | 43 | 11.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| LAI 590 mg QD - Double Blind | 1 of 44 |
| Placebo - Double Blind | 0 of 45 |
| LAI 590 mg QD - Open Label | 1 of 35 |
| Placebo - Open Label | 0 of 43 |
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 |
|---|---|
| LAI 590 mg QD - Double Blind | 41 of 44 |
| Placebo - Double Blind | 39 of 45 |
| LAI 590 mg QD - Open Label | 31 of 35 |
| Placebo - Open Label | 42 of 43 |
Most frequent serious adverse event terms
| Event term | Organ system | LAI 590 mg QD - Double Blind | Placebo - Double Blind | LAI 590 mg QD - Open Label | Placebo - Open Label |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 1 of 44 | 0 of 45 | 2 of 35 | 3 of 43 |
| Infective exacerbation of bronchiectasis | Infections and infestations | 2 of 44 | 1 of 45 | 0 of 35 | 0 of 43 |
| Pneumonia | Infections and infestations | 1 of 44 | 2 of 45 | 0 of 35 | 0 of 43 |
| Supraventricular tachycardia | Cardiac disorders | 1 of 44 | 0 of 45 | 0 of 35 | 1 of 43 |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | 0 of 44 | 0 of 45 | 1 of 35 | 1 of 43 |
| Gastroenteritis viral | Infections and infestations | 1 of 44 | 0 of 45 | 0 of 35 | 0 of 43 |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | 1 of 44 | 0 of 45 | 0 of 35 | 0 of 43 |
| Eosinophilic pneumonia | Respiratory, thoracic and mediastinal disorders | 0 of 44 | 1 of 45 | 0 of 35 | 0 of 43 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 1 of 44 | 0 of 45 | 0 of 35 | 0 of 43 |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | 1 of 44 | 0 of 45 | 0 of 35 | 0 of 43 |
Most frequent non-serious adverse event terms
| Event term | Organ system | LAI 590 mg QD - Double Blind | Placebo - Double Blind | LAI 590 mg QD - Open Label | Placebo - Open Label |
|---|---|---|---|---|---|
| Infective exacerbation of bronchiectasis | Infections and infestations | 17 of 44 | 9 of 45 | 9 of 35 | 15 of 43 |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | 19 of 44 | 4 of 45 | 3 of 35 | 12 of 43 |
| Cough | Respiratory, thoracic and mediastinal disorders | 14 of 44 | 6 of 45 | 3 of 35 | 8 of 43 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 4 of 44 | 5 of 45 | 5 of 35 | 5 of 43 |
| Nausea | Gastrointestinal disorders | 5 of 44 | 4 of 45 | 3 of 35 | 5 of 43 |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | 9 of 44 | 1 of 45 | 3 of 35 | 3 of 43 |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 3 of 44 | 1 of 45 | 4 of 35 | 6 of 43 |
| Headache | Nervous system disorders | 3 of 44 | 3 of 45 | 2 of 35 | 5 of 43 |
| Fatigue | General disorders | 7 of 44 | 4 of 45 | 0 of 35 | 0 of 43 |
| Pyrexia | General disorders | 4 of 44 | 3 of 45 | 3 of 35 | 1 of 43 |
Serious events with the highest arm-level rate: infective pulmonary exacerbation of cystic fibrosis (7.0%), infective exacerbation of bronchiectasis (4.5%), pneumonia (4.4%), urinary tract infection (2.9%), urosepsis (2.9%).
The registry reports serious adverse event counts in four groups: LAI and placebo during the double-blind period, and LAI and placebo in groups labelled open-label. During the double-blind period, the LAI group had 8 serious adverse events among 44 participants at risk; the placebo group had 4 among 45 at risk. In the open-label groups, LAI had 5 among 35 at risk, and placebo had 5 among 43 at risk. The record lists 1 death in the double-blind LAI group, none in the double-blind placebo group, 1 in the open-label LAI group, and none in the open-label placebo group.
These are counts, with the registry also giving the number at risk for each group. The record does not report whether the serious events or deaths were judged related to study treatment, nor does it provide details that establish the cause of any event. The open-label groups should not be combined with the double-blind groups as though they were one randomized comparison; the registry record does not describe the phase transition sufficiently for that interpretation.
The listed serious-event terms include infective pulmonary exacerbation of cystic fibrosis (7.0%), infective exacerbation of bronchiectasis (4.5%), pneumonia (4.4%), urinary tract infection (2.9%), urosepsis (2.9%) and acute coronary syndrome (2.9%). The record does not specify the denominator or treatment-group attribution for these listed percentages. Counts and listed terms describe reported safety events; they do not alone establish causation or comparative risk.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a randomized, double-blind, placebo-controlled phase 2 trial of daily LAI versus placebo, with a stated 84-day double-blind phase. It also specifies a primary microbiological endpoint: change in semi-quantitative mycobacterial culture results from baseline to Day 84. The scale ranges from 1, described as best, to 7, described as worst, and negative change values represent reduction in colony growth.
Those details explain what the trial asked and how the primary outcome was defined. They do not establish what the trial found. No arm-specific primary outcome results, comparative estimate, confidence interval or p-value are reported in the registry record. Therefore, the posted information does not support a conclusion about the size, direction or statistical precision of the treatment-group difference on the primary endpoint.
The listed secondary outcomes broaden the questions considered, covering culture status and timing, symptom and global health scores, and rescue-drug use. Their numerical results and analysis details are not reported in the registry record. The existence of these outcomes does not provide evidence about their observed results.
Safety counts are available for double-blind and open-label groups, including serious adverse events and deaths. These figures describe events reported in the record, but the record does not report treatment-relatedness for those events or enough detail to make the open-label groups a direct randomized comparison. The safety counts should not be used as a substitute for the missing primary efficacy comparison.
More broadly, an endpoint definition, a completed-study status and an enrollment total are not equivalent to a reported treatment-effect estimate. The available information supports a careful account of the trial design and measurement, while leaving the principal comparative result unresolved. It does not establish that treatment groups were the same, nor that they differed.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of why an outcome definition and a result are different kinds of evidence. The registry describes a graded culture scale, the direction of its scores and the timing of measurement. Those details help readers understand what a future numerical result would represent, but cannot replace the result itself. Without arm-level data or a comparative estimate, the central question remains unanswered in the registry record.
The primary endpoint is ordinal, with categories that encode different levels of culture growth. As a general statistical matter, analyses of ordered categories must account for the scale’s ranking while being cautious about treating the gaps between categories as equal. The registry does not report the analysis method, so readers cannot assess how the ordered response was summarized or what assumptions supported the comparison.
The record also illustrates why analysis populations and missing-data procedures matter. Although the primary population is labelled mITT, the definition and size are not reported. Without those details, enrollment cannot be equated with the number analyzed, and readers cannot assess which participants contributed endpoint data.
Finally, multiple secondary outcomes and separate safety groups require careful interpretation. The registry lists several distinct outcome questions and includes both double-blind and open-label safety counts. Without a reported multiplicity strategy, analysis details, or numerical outcome comparisons, these registry entries should be read descriptively rather than as a complete account of statistical evidence. The main statistical lesson is that design and measurement descriptions provide context, but estimates and uncertainty are needed to evaluate a comparative result.
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 of the registry information is that it does not report numerical results for the primary endpoint. No treatment-group changes, response-category counts, effect estimate, confidence interval or p-value are available in the registry record. This prevents assessment of the magnitude, direction and precision of the primary comparison.
The primary analysis population is identified as mITT, but its definition, size and group denominators are not reported. The handling of missing endpoint data and any sensitivity analyses are also not reported in the registry record. These omissions make it difficult to understand which participants contributed to the primary analysis and how missing measurements may have affected it.
The endpoint uses a seven-step ordered scale, but the record does not state the statistical analysis method or explain how categories and changes were summarized. It also does not give details of the randomization procedure, allocation concealment, or the specific roles covered by quadruple masking. These design and analysis details are not reported in the registry record.
The registry lists multiple secondary outcomes, but does not report numerical results or a multiplicity strategy. It also gives open-label safety counts without describing the phase transition sufficiently to interpret them as a direct randomized comparison. The listed serious adverse event terms include percentages whose denominator and group attribution are not reported in the registry record. These limitations constrain interpretation of both efficacy and safety information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Liposomal Amikacin for Inhalation (LAI) for Nontuberculous Mycobacteria — NCT01315236.
- Registry record last updated 2019-08-21; status last verified 2019-08.
- Results first posted 2019-08-21.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT01315236 is a completed phase 2 randomized, double-blind, placebo-controlled, parallel-group study of liposomal amikacin for inhalation in patients with recalcitrant nontuberculous mycobacterial lung disease. Enrollment was 90. The stated double-blind treatment period was 84 days, comparing LAI 590 mg once daily with placebo once daily. The registry reports quadruple masking.
The primary endpoint was change in semi-quantitative mycobacterial culture results from baseline to Day 84, measured on a 7-step scale from 1, best, to 7, worst. Negative values represent reduction in colony growth. The primary population is labelled mITT.
The registry lists secondary culture, symptom, health-rating and rescue-drug outcomes, and reports serious adverse event counts for double-blind and open-label groups. Numerical results for the primary comparison, its statistical uncertainty, the mITT definition, and details of missing-data and multiplicity methods are not reported in the registry record.