Primary, key secondary, and select secondary end points (ITT population)
| End point . | Mavorixafor (n = 14) . | Placebo (n = 17) . | LS mean difference from placebo (SE), LS mean difference 95% CI . | P value . |
|---|---|---|---|---|
| Primary end point | ||||
| Overall TATANC,∗ LS mean (SE), h, 95% CI | 15.0 (1.9) 11.2-18.9 | 2.8 (1.5) 0-5.9 | 12.3 (2.5) 7.2-17.4 | P < .001 |
| Key secondary end point | ||||
| Overall TATALC,† LS mean (SE), h, 95% CI | 15.8 (1.4) 13.0-18.7 | 4.6 (1.1) 2.2-6.9 | 11.3 (1.8) 7.5-15.0 | P < .001 |
| Composite efficacy score at 52 wk, LS mean (SE), 95% CI | n = 11 26.7 (3.5) 19.9-33.5 | n = 17 33.4 (2.8) 27.9-38.8 | −6.6 (4.5) −15.5 to 2.2 | P = .14‡ |
| Total wart change score at 52 wk (based on CGI-C), LS mean (SE), 95% CI | n = 11 −1.1 (0.7) −2.5 to 0.3 | n = 17 −1.2 (0.5) −2.2 to −0.1 | 0.1 (0.9) −1.7 to 1.8 | P = .94‡ |
| Total infection score, LS mean (SE), 95% CI | 7.4 (2.8) 1.6-13.2 | 12.3 (2.4) 7.2-17.3 | −4.9 (3.7) −12.6 to 2.9 | P = .21‡ |
| Select secondary end point | ||||
| Annualized infection rate, LS mean (SE) | 1.7 (0.5) | 4.2 (0.7) | 0.4 (0.2-0.8)§ | P = .007‡ |
| Infection severity based on CTCAE grade, n (%) | — | — | ||
| Grade 1 | 8 (57.1) | 8 (47.1) | — | — |
| Grade 2 | 2 (14.3) | 3 (17.6) | — | — |
| Grade 3 | 1 (7.1) | 4 (23.5) | — | — |
| Grade 4 | 0 | 1 (5.9) | — | — |
| Grade 5 | 0 | 0 | — | — |
| Proportion of participants with infections, n (%) | ||||
| ≥5 infections | 1 (7.1) | 5 (29.4) | — | P = .13‡ |
| <5 infections | 13 (92.9) | 12 (70.6) | — | |
| Infection duration, median (range), d | 8.5 (0-43) | 32.0 (8-134) | — | — |
| Overall vaccine titer (tetanus‖), LS mean (95% CI) | 1.3 (0.8-2.0) | 0.8 (0.5-1.2) | 1.6 (1.0-2.6)¶ | P = .039‡ |
| End point . | Mavorixafor (n = 14) . | Placebo (n = 17) . | LS mean difference from placebo (SE), LS mean difference 95% CI . | P value . |
|---|---|---|---|---|
| Primary end point | ||||
| Overall TATANC,∗ LS mean (SE), h, 95% CI | 15.0 (1.9) 11.2-18.9 | 2.8 (1.5) 0-5.9 | 12.3 (2.5) 7.2-17.4 | P < .001 |
| Key secondary end point | ||||
| Overall TATALC,† LS mean (SE), h, 95% CI | 15.8 (1.4) 13.0-18.7 | 4.6 (1.1) 2.2-6.9 | 11.3 (1.8) 7.5-15.0 | P < .001 |
| Composite efficacy score at 52 wk, LS mean (SE), 95% CI | n = 11 26.7 (3.5) 19.9-33.5 | n = 17 33.4 (2.8) 27.9-38.8 | −6.6 (4.5) −15.5 to 2.2 | P = .14‡ |
| Total wart change score at 52 wk (based on CGI-C), LS mean (SE), 95% CI | n = 11 −1.1 (0.7) −2.5 to 0.3 | n = 17 −1.2 (0.5) −2.2 to −0.1 | 0.1 (0.9) −1.7 to 1.8 | P = .94‡ |
| Total infection score, LS mean (SE), 95% CI | 7.4 (2.8) 1.6-13.2 | 12.3 (2.4) 7.2-17.3 | −4.9 (3.7) −12.6 to 2.9 | P = .21‡ |
| Select secondary end point | ||||
| Annualized infection rate, LS mean (SE) | 1.7 (0.5) | 4.2 (0.7) | 0.4 (0.2-0.8)§ | P = .007‡ |
| Infection severity based on CTCAE grade, n (%) | — | — | ||
| Grade 1 | 8 (57.1) | 8 (47.1) | — | — |
| Grade 2 | 2 (14.3) | 3 (17.6) | — | — |
| Grade 3 | 1 (7.1) | 4 (23.5) | — | — |
| Grade 4 | 0 | 1 (5.9) | — | — |
| Grade 5 | 0 | 0 | — | — |
| Proportion of participants with infections, n (%) | ||||
| ≥5 infections | 1 (7.1) | 5 (29.4) | — | P = .13‡ |
| <5 infections | 13 (92.9) | 12 (70.6) | — | |
| Infection duration, median (range), d | 8.5 (0-43) | 32.0 (8-134) | — | — |
| Overall vaccine titer (tetanus‖), LS mean (95% CI) | 1.3 (0.8-2.0) | 0.8 (0.5-1.2) | 1.6 (1.0-2.6)¶ | P = .039‡ |
CGI-C, Clinical Global Impression of Change; CTCAE, Common Terminology Criteria for Adverse Events; ITT, intent-to-treat.
TATANC of ≥0.5 × 103/μL over a 24-hour period, assessed every 3 months for 12 months.
TATALC of ≥1.0 × 103/μL over a 24-hour period assessed every 3 months for 12 months.
If the analysis for a specific end point was not statistically significant, results for subsequent end points were considered nominal.
Data reported as ratio (95% CI).
Eight participants receiving placebo and 10 participants receiving mavorixafor were vaccinated with tetanus vaccines at week 13. All participants in the ITT population were followed through week 52.
Data reported as LS mean ratio (95% CI).