Table 3.
Primary efficacy outcomes
| DS-TB | RR-TB | |||||
|---|---|---|---|---|---|---|
| Total* | HRZE | 4Pa100MZ† | 4Pa200MZ† | 6Pa200MZ† | 6Pa200MZ† | |
| Modified intention-to-treat analysis | ||||||
| Total randomised | 271 | 68 | 65 | 71 | 67 | 13 |
| Unassessable | 37 | 8 | 8 | 10 | 11 | 2 |
| Assessable | 234 | 60 | 57 | 61 | 56 | 11 |
| Unfavourable, n (%) | 55 (23.5) | 8 (13.3) | 19 (33.3) | 15 (24.6) | 13 (23.2) | 1 (9.1) |
| Adverse event | 17 | 2 | 3 | 3 | 9 | 1 |
| Withdrawal (culture-positive)‡ | 16 | 0 | 9 | 6 | 1 | 0 |
| Confirmed relapse | 4 | 0 | 2 | 1 | 1 | 0 |
| Death (not violent or accidental)§ | 4 | 0 | 1 | 1 | 2 | 0 |
| Non-adherence to study protocol | 6 | 4 | 1 | 1 | 0 | 0 |
| Physician decision | 4 | 2 | 0 | 2 | 0 | 0 |
| Death (TB-related)¶ | 2 | 0 | 1 | 1 | 0 | 0 |
| Clinical deterioration in follow-up | 1 | 0 | 1 | 0 | 0 | 0 |
| Lost to follow-up (during treatment) | 1 | 0 | 1 | 0 | 0 | 0 |
| Favourable, n (%) (95% CI) | 179 (76.5) | 52 (86.7) | 38 (66.7) | 46 (75.4) | 43 (76.8) | 10 (90.9) |
| (70.5 to 81.8) | (75.4 to 94.1) | (52.9 to 78.6) | (62.7 to 85.5) | (63.6 to 87.0) | (58.7 to 99.8) | |
| Difference from HRZE in unfavourable, % (95% CI) | — | — | 20.0 (−5.0 to 35.0) | 11.3 (−2.6 to 25.1) | 9.9 (−4.1 to 23.9) | — |
| Per-protocol analysis | ||||||
| Total randomised | 271 | 68 | 65 | 71 | 67 | 13 |
| Unassessable | 62 | 15 | 13 | 14 | 20 | 3 |
| Assessable | 209 | 53 | 52 | 57 | 47 | 10 |
| Unfavourable, n (%) | 30 (14.4) | 1 (1.9) | 14 (26.9) | 11 (19.3) | 4 (8.5) | 0 (0.0) |
| Withdrawal (culture-positive)‡ | 16 | 0 | 9 | 6 | 1 | — |
| Confirmed relapse | 4 | 0 | 2 | 1 | 1 | — |
| Death (not violent or accidental)§ | 4 | 0 | 1 | 1 | 2 | — |
| Death (TB-related)¶ | 2 | 0 | 1 | 1 | 0 | — |
| Treatment failure | 2 | 1 | 0 | 1 | 0 | — |
| Other | 2 | 0 | 1 | 1 | 0 | — |
| Favourable, n (%) (95% CI) | 179 (85.6) | 52 (98.1) | 38 (73.1) | 46 (80.7) | 43 (91.5) | 10 (100.0) |
| (80.1 to 90.1) | (89.9 to 100.0) | (59.0 to 88.4) | (68.1 to 90.0) | (79.6 to 97.6) | (69.2 to 100.0) | |
| Difference from HRZE in unfavourable, % (95% CI) | — | — | 25.0 (−12.4 to 37.6) | 17.4 (−6.5 to 28.3) | 6.6 (−2.2 to 15.4) | — |
* Total includes control arm but does not include the (non-randomised) MDR-TB patients.
† 200 mg pretomanid (Pa, PMD) daily, 400 mg moxifloxacin (M) and 1500 mg pyrazinamide (Z) for 6 months (6Pa200MZ) or 4 months (4Pa200MZ); 100 mg pretomanid daily for 4 months in the same combination (4Pa100MZ).
‡ Patients who failed to achieve sustained culture-negative status and were withdrawn based on clinical assessment of treatment failure.
§ During treatment.
¶ During follow-up.
DS-TB = drug-susceptible TB; RR-TB = rifampicin-resistant TB; H = isoniazid; R = rifampicin; Z = pyrazinamide; E = ethambutol; M = moxifloxacin; Pa = pretomanid; CI = confidence interval; MDR-TB = multidrug-resistant TB.