Phase II Investigation of Antimycobacterial Therapy on Progressive, Pulmonary Sarcoidosis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Phase II Investigation of Antimycobacterial Therapy on Progressive, Pulmonary Sarcoidosis (NCT02024555), a randomized phase 2 study. The primary outcome is Change in Percent Predicted Absolute Forced Vital Capacity (FVC) in Participants With Pulmonary Sarcoidosis, Comparing Baseline With Performance After Completion of 16 Weeks of Therapy..

2. Clinical Question

The study was designed to investigate efficacy and safety in people with progressive pulmonary sarcoidosis. Its stated expectation was that the CLEAR regimen would improve absolute FVC percent predicted. The registry record identifies change in percent predicted absolute FVC after 16 weeks of therapy as the primary outcome. The posted comparison describes the change in each arm and the difference between arm means. It does not, on its own, establish why any observed difference occurred or whether it reflects a reliable treatment effect.

The trial also listed outcomes addressing radiographic change, walking distance, oxygen saturation, dyspnea, respiratory health status, fatigue, health status more broadly and adverse events. Those outcomes add perspectives on disease status and participants’ experience, but they are separate from the primary lung-function question. The clinical question is therefore focused: compare the change in a specified lung-function measure over the stated treatment period between the multidrug regimen and placebo.

3. Trial Design

This was a phase II, randomized, quadruple-masked, parallel-group, placebo-controlled trial sponsored by Vanderbilt University. The registry record lists an enrollment of 97 and a completed status. The study start is recorded as 2014-03, and primary completion is recorded as 2019-04-01. The trial title describes an investigation of antimycobacterial therapy in progressive pulmonary sarcoidosis.

Participants were assigned to the concomitant levofloxacin, ethambutol, azithromycin and rifampin regimen or to placebo. The intervention description gives levofloxacin 500mg po QD, ethambutol 1200mg po QD, azithromycin 250 mg po QD and rifampin 600mg po QD or rifabutin 300mg po QD. The placebo description says riboflavin was used for rifampin and encapsulated microcrystalline cellulose replaced the other three drugs; the pill count was the same as the comparator regimen. The record does not report further details about the allocation sequence or how masking was maintained.

The primary endpoint was assessed from baseline to 16 weeks. The outcome record states that 43 participants in each arm completed this measure. That endpoint population should be distinguished from the enrollment figure: the registry record does not explain the reasons for the difference, the handling of missing measurements, or the analysis population used for every outcome. Although randomization and masking are listed, details of the trial’s original statistical analysis plan, sample-size justification and any interim analyses are not reported in the registry record.

The posted results include continuous outcomes and a count outcome, as well as adverse-event summaries. They do not include sponsor-reported analyses in the registry record. The numerical arm comparisons discussed in the statistical interpretation are therefore simple unadjusted calculations from posted summary statistics, not descriptions of a reported adjusted model.

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: 446 participants were screened but only 97 participants met inclusion/exclusion and were randomized.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Only 43 participants in each arm completed this measure

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.

6. Primary Endpoint

The registry lists the primary outcome as Change in Percent Predicted Absolute Forced Vital Capacity (FVC) in Participants With Pulmonary Sarcoidosis, Comparing Baseline With Performance After Completion of 16 Weeks of Therapy., measured over Baseline to 16 weeks.

The primary endpoint is change in percent predicted absolute forced vital capacity (FVC), comparing baseline with performance after 16 weeks of therapy. FVC is a measure of the volume of air that can be forcefully exhaled after a full inhalation; expressing it as percent predicted places the observed value in relation to an expected reference value. The registry record describes the measure as a mean continuous outcome and gives standard deviation as its measure of spread.

FVC was selected as the primary outcome because the trial’s stated purpose was to investigate the efficacy and safety of oral antimycobacterial therapy in confirmed progressive pulmonary sarcoidosis. The summary specifically says the investigators suspected the CLEAR regimen would improve absolute FVC percent predicted. The endpoint thus focuses on a lung-function measure and its change over the treatment period, rather than relying only on symptoms or participants’ reported health status.

A change score asks how the measure at the later assessment differs from the baseline assessment within the endpoint definition. The arm comparison then contrasts the mean changes. It does not show each participant’s trajectory, and a group mean can conceal variation among individuals. The posted results give the mean and standard deviation for each arm, along with an unadjusted mean difference and its confidence interval. The record does not report the detailed measurement protocol, handling of missing values, or whether the primary comparison was adjusted for baseline FVC.

7. Results Posted in the Registry

• Concomitant Levaquin, Ethambutol, Azithromycin and Rifampin: mean 0.35 versus 0.17 on control; difference 0.18 (95% CI -2.77 to 3.13), standardized effect d = 0.03; z-test p = 0.905.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Change in the Saint George's Respiratory Questionnaire (SGRQ)

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard errors.
• Result:
  • Concomitant Levaquin, Ethambutol, Azithromycin and Rifampin: mean -2.23 versus -6.30 on control; difference 4.07 (95% CI -25.17 to 33.31), standardized effect d = 0.06; z-test p = 0.785.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Concomitant Levaquin, Ethambutol, Azithromycin and Rifampin and the control. The interval gives the range of effects that remain compatible with the data.

Failure of Standard Therapy

• Endpoint type: Binary: the number of participants with the event, compared as proportions.
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Concomitant Levaquin, Ethambutol, Azithromycin and Rifampin: 7.0% (3/43) versus 4.2% (2/48) on control; risk difference 2.8 percentage points (95% CI -6.7 to 12.3), risk ratio 1.67 (0.29–9.55); unadjusted two-proportion z-test p = 0.557.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Concomitant Levaquin, Ethambutol, Azithromycin and Rifampin and the control. The interval gives the range of effects that remain compatible with the data.

The secondary outcomes cover several kinds of information: radiographic improvement, walking distance, oxygen saturation, dyspnea, respiratory health status, fatigue, health status assessed with the King's Sarcoidosis Questionnaire, adverse events and failure of standard therapy. This breadth can help characterize what was measured beyond the primary lung-function endpoint, but the outcomes do not all represent the same construct or use the same statistical scale. They should not be combined into a single claim about the overall result.

Across the posted secondary comparisons, the estimates remain uncertain. Their confidence intervals include no difference, and the registry record does not report sponsor analyses or an analysis hierarchy. Taken together, the posted results do not resolve the uncertainty in the primary comparison. Secondary findings can complement the primary endpoint by identifying patterns worth further investigation, but they cannot replace a clear primary analysis or establish a consistent effect across different outcomes.

Unadjusted secondary results carry less weight when many outcomes are examined because the chance of at least one apparently unusual result can rise as separate comparisons accumulate. Statistical adjustment or a prespecified hierarchy can address this issue, but neither is described in the registry record. The record lists 12 outcome measures, including 11 posted secondary outcomes, and does not say whether all were planned for formal hypothesis testing. The safest reading is descriptive: consider the direction and uncertainty of each result, avoid selecting isolated findings as definitive, and give primary emphasis to the prespecified primary endpoint. The registry record does not report enough detail to determine whether secondary outcomes were exploratory, confirmatory or handled in another way.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Radiographic Improvement in Sarcoidosis Lung Disease by Frontal Chest X-ray .
• Six Minute Walk, Distance in Meters
• Change in Oxygen Saturation
• Change in Level of Dyspnea
• Change in the Saint George's Respiratory Questionnaire (SGRQ)
• Fatigue Assessment Scale (FAS).
• Change in the King's Sarcoidosis Questionnaire (KSQ) for the Assessment of Health Status;
• Adverse Events

9. Statistical Interpretation

10. Statistical Methods Explained

The primary measure is continuous, so the posted arm summaries use means and standard deviations. A mean describes the central value of the observed changes within an arm. A standard deviation describes how spread out individual observations are around that mean; it is not the uncertainty in the mean itself. The comparison of interest is the difference between the arm means. The posted unadjusted estimate is 0.18 in the stated endpoint unit.

What does the confidence interval add?

A confidence interval expresses the uncertainty around an estimated difference using the observed data and a statistical method. A narrower interval indicates greater precision; a wider interval indicates less precision. For the primary comparison, the interval runs from -2.772 to 3.132. Because it crosses zero, the data are compatible with no mean difference as well as with differences on either side of zero. The interval should not be read as a range containing every plausible value with certainty, nor does it resolve whether any value within it would be clinically important. The registry record does not state a threshold for clinical importance.

How should the p value be understood?

The unadjusted p value for the primary comparison is 0.905. In general, a p value measures how unusual data at least as incompatible with a null hypothesis would be under the assumptions of the statistical test. It is not the probability that the null hypothesis is true, and it does not measure the size or importance of an effect. The record does not report a complete analysis plan or identify a sponsor-reported test for this endpoint. The recalculated comparison is therefore best treated as a descriptive, unadjusted analysis of the posted summary statistics.

Why does variability matter?

Variation among participants affects how precisely a group mean and an arm difference can be estimated. When individual outcomes vary widely relative to the between-arm estimate, a given sample may yield a broad interval. Here, the posted standard deviations are 6.76 and 7.2, while the estimated mean difference is 0.18. These values are not interchangeable: the first describe observed spread within arms, while the latter describes the estimated contrast. A standard error, by contrast, describes uncertainty in an estimate; the registry record identifies standard deviation for the primary outcome.

The analysis record also includes a secondary continuous outcome, change in the Saint George's Respiratory Questionnaire (SGRQ), with standard error listed as the measure of spread. A standard error and a standard deviation answer different questions. Standard error concerns the precision of an estimated quantity, while standard deviation concerns dispersion among observations. The record does not explain the method used to generate the SGRQ summary or the unusually broad recalculated comparison interval. Such uncertainties reinforce the need to interpret each posted comparison in light of the method actually documented, rather than assuming an analysis that is not described.

What does an unadjusted comparison leave out?

An unadjusted comparison uses the arm summaries without accounting for other variables in a statistical model. It does not demonstrate that the groups were balanced on every prognostic factor, nor does it correct for missing outcome data. Randomization is intended to support fair comparisons on average, but the registry record does not report baseline characteristics, the exact analysis population, or a model adjusting for baseline outcome values. The recalculated comparison should not be described as a covariate-adjusted estimate or as a sponsor’s prespecified primary analysis.

The secondary binary outcome, failure of standard therapy, is expressed as a risk difference. In general, a risk difference contrasts the proportions with an event in two groups. The posted estimate is 0.028, with a confidence interval from -0.067 to 0.123 and an unadjusted p value of 0.557. The interval crosses zero, and the numbers of events are small. These features make the comparison imprecise. The registry record does not report an adjusted binary-outcome model or a multiplicity correction.

What can over-dispersion change?

For count outcomes, over-dispersion means that observed variation exceeds what a simple count model assumes. A model that ignores over-dispersion can understate uncertainty and produce confidence intervals that are too narrow. The posted primary endpoint is continuous, not a count, and the registry record does not report a count-model analysis for the failure-of-standard-therapy outcome. This is a general methodological consideration, not a claim that over-dispersion occurred in this trial.

The registry lists 12 outcome measures and 11 posted secondary outcomes. Multiple outcome measures create opportunities for chance findings when each is analyzed separately. A prespecified hierarchy or adjustment can help control that issue, but neither is reported in the registry record. The record also reports no sponsor analyses for the posted outcomes. Consequently, the simple recalculations can describe estimates and uncertainty, but they do not reveal the full inferential strategy used by investigators, if any.

The planning calculations use a normal approximation and equal allocation between arms, with a two-sided significance level of 0.05. They use stated fractions of the observed effect as targets. These assumptions are explicitly for a planning illustration, not a reconstruction of the trial’s original design; the original design assumptions and sample-size justification are not reported in the registry record. The calculations concern prospective planning under specified assumptions. They do not measure the probability that the completed study detected an effect.

11. Confidence Intervals and Precision

The primary mean difference is 0.18, and its confidence interval extends from -2.772 to 3.132. This interval is wide relative to the point estimate and crosses zero. It therefore leaves room for a difference favoring either arm, as well as no difference. The posted result is not a precise estimate of the contrast, and its point estimate should not be interpreted without the interval.

The interval’s width reflects both the observed variability and the amount of outcome information available for the comparison. The record states that 43 participants in each arm completed the primary measure; the standard deviations were 6.76 and 7.2. Those summaries indicate that individual changes varied appreciably within each arm relative to the estimated difference. The interval makes the resulting uncertainty visible in a way that the point estimate alone cannot.

Precision and statistical significance are related but not identical to clinical importance. The registry record does not define a minimum clinically important difference for FVC change. It is therefore not possible to label the interval’s endpoints as clinically meaningful or unimportant based on this record alone. Nor does an interval that includes zero establish equivalence: an equivalence conclusion requires a prespecified margin and an appropriate analysis, neither of which is reported in the registry record.

The SGRQ and failure-of-standard-therapy comparisons also have intervals spanning zero. Their uncertainty is compounded by the absence of a reported multiplicity approach and detailed analysis methods. Across outcomes, confidence intervals should be read as measures of statistical precision under the analysis used, not as guarantees about future patients or as proof of a treatment effect.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are stated as participants per arm for 80% power and participants per arm for 90% power. They are not total sample sizes and do not describe the number observed in the trial. The planning assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, a normal approximation, and a target effect set to the stated fraction of the observed effect. The original trial’s own design assumptions and sample-size justification are not reported in the registry record.

For planning at 100% of the observed effect, the illustration gives 23,629 participants per arm for 80% power and 31,632 participants per arm for 90% power. For planning at 75% of the observed effect, it gives 42,007 participants per arm for 80% power and 56,235 participants per arm for 90% power. For planning at 50% of the observed effect, it gives 94,514 participants per arm for 80% power and 126,528 participants per arm for 90% power.

The 80% power column represents a planning target under which a study would have an 80% chance of detecting the specified effect if the assumptions and target were true. The 90% column uses a higher chance of detection, which requires more participants under the same assumptions. The curve is another way to view the planning relationship: as the target effect becomes smaller, the required number of participants increases. These numbers are planning illustrations based on the observed estimate, not observed power.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, Δ is the difference in means to detect and σ is the common standard deviation.

Worked calculation for this trial

1. Observed difference Δ = 0.180; pooled standard deviation σ = 6.983.
2. n = 2 × (1.96 + 0.842)² × 6.983² / 0.180² = 23,628.4, rounded up to 23,629 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
delta <- 0.18                      # observed difference in means
sigma <- 6.98346618807595                     # pooled standard deviation
n_per_arm <- function(power, frac = 1) {
  ceiling(round(2 * (z_a + qnorm(power))^2 * sigma^2 / (delta * frac)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (t-based, so very slightly larger)
cat("power.t.test:", round(power.t.test(delta = delta, sd = sigma, power = 0.80)$n, 1), "\n")

# Simulation: exact two-sample t-test using simulated means and variances
set.seed(2024555)
n    <- n_per_arm(0.80)
reps <- 20000
m_diff <- rnorm(reps, delta, sigma * sqrt(2 / n))             # difference in sample means
s2     <- sigma^2 * rchisq(reps, 2 * (n - 1)) / (2 * (n - 1))   # pooled sample variance
tstat  <- m_diff / sqrt(s2 * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(tstat) > qt(0.975, 2 * (n - 1))), reps))

Output from running the code

100% of observed effect: n per arm = 23,629 (80% power), 31,632 (90% power)
 75% of observed effect: n per arm = 42,007 (80% power), 56,235 (90% power)
 50% of observed effect: n per arm = 94,514 (80% power), 126,528 (90% power)
power.t.test: 23629.4 
Simulated power at n = 23,629 per arm: 0.798 (20000 replicates)

The simulation draws many sets of group means and variances at the planned sample size and counts how often the two-sample t-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Multiplicity, Missing Data and Analysis Populations

The registry record lists multiple outcome measures, including a primary FVC outcome and secondary measures spanning respiratory symptoms, function, health status, radiographic findings and adverse events. When many outcomes are assessed, separate statistical tests can produce apparently notable results by chance. A prespecified hierarchy, a limited set of confirmatory comparisons or an adjustment for multiple testing can help define how results should be interpreted. The registry record does not report whether any such procedure was planned or used. The posted comparisons are unadjusted, so they should not be read as multiplicity-controlled evidence.

Missing-data methods are also not reported. The primary outcome description states that 43 participants in each arm completed the measure, while the record lists enrollment of 97. These entries do not explain why some participants lack a primary measure, whether their data were missing for different reasons, or how missing observations were handled. The record does not identify an intention-to-treat population, a per-protocol population, an imputation method, or a sensitivity analysis. Without those details, it is not possible to assess how the analysis population or missingness assumptions may have affected the estimate.

The registry record does not report the original sample-size justification, a detailed statistical analysis plan, interim analyses, baseline-adjusted models or sponsor-reported statistical analyses. It also does not explain whether the posted results represent all planned analyses. These omissions limit assessment of prespecification and selective reporting. They do not demonstrate that a particular method was or was not used; they mean the relevant information is not reported in the registry record.

15. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: pneumonia (2.1%), sepsis from abcess (2.1%), post-operative infection (2.1%), leukopenia (2.0%), neurosarcoidosis (2.0%).

The registry record reports serious adverse events in 4 participants among 49 at risk in the multidrug regimen arm and 3 participants among 48 at risk in the placebo arm. Other adverse events were reported in 24 participants among 49 at risk in the regimen arm and 16 participants among 48 at risk in the placebo arm. No deaths were reported in either group. These are descriptive counts, and the at-risk group sizes differ from the primary endpoint completion counts.

The record lists serious-event terms including Pneumonia, Sepsis from Abcess, post-operative infection, Leukopenia, Neurosarcoidosis and Hypotension. Each is accompanied by a percentage in the registry record, but the list does not identify an arm for each term. Those terms should not be interpreted as a comparative estimate between groups. The summaries do not establish that an event was caused by the assigned regimen, and the registry record does not give detailed event timing, severity grading or exposure-adjusted rates. The counts alone cannot support a complete safety comparison.

16. What the Result Does, and Does Not, Mean

The posted primary summary shows a small positive unadjusted mean difference in change in percent predicted absolute FVC, but the confidence interval is broad and includes zero. The result is therefore uncertain: it does not establish a clear difference between the multidrug regimen and placebo, and it does not establish that the groups are equivalent. The registry record does not report a clinically important threshold with which to judge the range of possible differences.

This trial addresses a specific question in participants with confirmed progressive pulmonary sarcoidosis and reports the primary outcome from baseline to 16 weeks. Its result should not be generalized beyond the population, intervention and assessment period described in the record. The regimen combined several antimycobacterial drugs, so the comparison cannot isolate the contribution of any individual drug. The placebo arm also used replacement pills to match the comparator pill count, as described in the record.

The posted secondary outcomes cover additional measures, but their unadjusted comparisons do not remove the uncertainty in the primary result. The registry record does not report the analysis plan, missing-data methods, multiplicity strategy or sponsor-reported inferential analyses. Those omissions make it harder to determine how the investigators intended the results to be interpreted or how sensitive the conclusions might be to analysis choices.

The adverse-event summaries describe reported counts, not causal effects. Differences in the counts cannot by themselves establish that the regimen is safer or less safe, and the term list is not presented as an arm-by-arm comparison. The trial result is statistical evidence from a particular study record, not medical advice. It should not be read as a recommendation to start, stop or change treatment.

17. Why This Trial Matters Statistically

This trial illustrates why an effect estimate needs to be read together with its uncertainty. The primary mean difference is 0.18, but its interval extends from -2.772 to 3.132. A point estimate can look simple and decisive when viewed alone; the interval shows that the underlying comparison remains compatible with a broad range of values, including no difference. That distinction is central to interpreting clinical trial results.

It also shows how variability and outcome completion shape precision. The primary endpoint is reported for 43 participants in each arm, and the posted standard deviations are 6.76 and 7.2. The estimated contrast is small relative to the observed spread. The resulting uncertainty matters more for interpretation than treating the p value as a verdict. A nonsmall p value does not demonstrate equivalence, while a positive point estimate does not establish meaningful benefit.

The record further highlights the role of transparent methods. Randomization and quadruple masking are listed, but the analysis plan, missing-data approach, multiplicity strategy and original sample-size justification are not reported. These details are necessary to judge whether the available comparisons follow prespecified methods and how secondary findings should be weighted. The planning figures can illustrate the sample sizes associated with particular effect assumptions, but they do not reveal the trial’s original design or its probability of success.

Finally, the trial combines several medicines and includes outcomes with different meanings and scales. Clear statistical interpretation requires distinguishing the primary question from secondary exploration, descriptive safety counts from causal conclusions, and posted summary statistics from sponsor-reported analyses. Those distinctions make the evidence easier to assess without overstating what the record establishes.

18. 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 primary endpoint is reported for 43 participants in each arm, but enrollment is listed as 97. The registry record does not explain the difference, identify reasons for missing primary measurements or describe the analysis population. Without those details, potential effects of attrition or missingness cannot be assessed. The record also does not provide baseline characteristics that would help readers understand the enrolled population or assess the observed groups.

The primary assessment covers baseline to 16 weeks, so the posted endpoint does not describe longer-term outcomes. The multidrug regimen includes levofloxacin, ethambutol, azithromycin and rifampin or rifabutin. Because these drugs were assigned together, this comparison cannot separate their individual contributions. The record does not report adherence, treatment discontinuations or further exposure details that might help interpret the observed summaries.

The available statistical information is limited. Sponsor-reported analyses are not posted in the registry record; the original analysis plan, sample-size justification, multiplicity strategy, interim analyses and missing-data methods are not reported. The calculations from summary statistics are unadjusted and should not be mistaken for a documented adjusted primary analysis. The secondary outcomes add information, but their methods and inferential status are not described sufficiently to support strong conclusions across outcomes.

The safety record gives counts and a list of serious-event terms, but it does not provide enough detail about event timing, duration, severity or attribution for a comprehensive comparative assessment. These limitations concern what is reported in the registry record; they do not establish that information was not collected or that a particular method was not used.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: Phase II Investigation of Antimycobacterial Therapy on Progressive, Pulmonary Sarcoidosis — NCT02024555.
• Registry record last updated 2020-07-09; status last verified 2020-06.
• Results first posted 2020-07-09.
• 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.

22. Record Summary

The registry record describes a completed, randomized, quadruple-masked, parallel, placebo-controlled phase II trial sponsored by Vanderbilt University. It lists enrollment of 97 and primary completion on 2019-04-01. The primary question was change in percent predicted absolute FVC from baseline to 16 weeks among participants with progressive pulmonary sarcoidosis.

The primary outcome was completed by 43 participants in each arm. The unadjusted mean difference was 0.18, with a confidence interval from -2.772 to 3.132 and an unadjusted p value of 0.905. The interval includes zero and is broad, leaving substantial uncertainty about the size and direction of the between-arm difference.

The record also posts secondary outcome and adverse-event summaries, but does not report sponsor analyses, the original statistical plan, missing-data methods or a multiplicity approach. The posted comparisons are best interpreted as descriptive evidence with important uncertainty, not as a definitive statement about efficacy or safety.