Fig 1.
CONSORT flow diagram for the C208 Stage II trial [19].
*The modified intent-to-treat population was a subset of the intent-to-treat population that excluded nine patients (6 BDQ and 3 placebo) with Mycobacteria Growth Indicator Tube results that did not allow for primary efficacy evaluation (no evidence of culture positivity prior to first intake of blinded study drug or no results during the first 8 weeks after first intake), seven patients (3 BDQ and 4 placebo) infected with extensively drug-resistant tuberculosis, eight (4 BDQ and 4 placebo) with drug-sensitive tuberculosis, and four patients (0 BDQ and 4 placebo) for whom the multidrug-resistant tuberculosis status could not be confirmed.
Table 1.
Relationship between S8 and T for BDQ and placebo.
Table 2.
Relationship between S24 and T for BDQ and placebo.
Table 3.
Sensitivity, specificity, positive predictive value and negative predictive value of S8 and S24.
Table 4.
Drop-out rate during the trial.
Table 5.
Response rates (M = F) and % missing data during the trial.
Fig 2.
Response rate over time in the missing = failure and multiple imputation analyses.
Table 6.
Average response rate over time in the multiple imputation analysis.
Fig 3.
Densities and distribution of the individual causal association between ΔT and ΔS for S8, S8xS24, S24 based on MGIT, and S24 based on AFB.
Table 7.
Distribution of individual causal association between ΔT and ΔS for S8, S8xS24, S24 based on MGIT, and S24 based on AFB.
Fig 4.
ICA evaluated using the missing = failure and multiple imputation methods at A) Week 8 and B) Week 24.
Fig 5.
Surrogate predictive value for A) S8 and B) S24. The conditional probabilities of (A) ΔT given ΔS8 or (B) ΔT given ΔS24 are shown. For any individual patient, ΔT and ΔS will be -1 (Harm), 0 (Equal), and 1 (Benefit).