Table 2.
Efficacy Outcomes for Maribavir (ITT Population)
| Outcome | Maribavir 400 mg BID (n = 40) |
Maribavir 800 mg BID (n = 40) |
Maribavir 1200 mg BID (n = 40) |
All Doses (N = 120) |
|---|---|---|---|---|
| Primary efficacy endpoint: patients with confirmed undetectable plasma CMV DNA within 6 weeks, n (%)a | ||||
| Yesb | 28 (70.0) | 25 (62.5) | 27 (67.5) | 80 (66.7) |
| No | 12 (30.0) | 15 (37.5) | 11 (27.5) | 38 (31.7) |
| Patients with missing data (no post-baseline CMV measurement within 6 weeks), n (%) |
0 | 0 | 2 (5.0) | 2 (1.7) |
| Treatment effect estimate by group | ||||
| Estimated ratea | 0.70 | 0.63 | 0.68 | 0.67 |
| 95% CI | 0.53–0.83 | 0.46–0.77 | 0.51–0.81 | 0.57–0.75 |
| Secondary efficacy endpoint: CMV recurrence in patients achieving undetectable CMV DNA | ||||
| Patients achieving confirmed undetectable CMV DNA during the study, as defined in the recurrence analysis, n | 29 | 27 | 30 | 86 |
| Patients with CMV recurrence at any time during the study, n (%)c | ||||
| Yesd | 7 (24.1) | 11 (40.7) | 12 (40.0) | 30 (34.9) |
| Noe,f | 22 (75.9) | 14 (51.9) | 17 (56.7) | 53 (61.6) |
| Treatment effect estimate by group | ||||
| Estimated ratec | 0.24 | 0.41 | 0.40 | 0.35 |
| 95% CI | 0.10–0.44 | 0.22–0.61 | 0.23–0.59 | 0.25–0.46 |
| Patients with CMV recurrence on treatment, n (%)c | 6 (20.7) | 9 (33.3) | 10 (33.3) | 25 (29.1) |
| Patients with CMV recurrence off treatment, n (%)c,g | 1 (3.4) | 2 (7.4) | 2 (6.7) | 5 (5.8) |
Abbreviations: AE, adverse event; BID, twice daily; CI, confidence interval; CMV, cytomegalovirus; DNA, deoxyribonucleic acid; ITT, intent-to-treat.
aNumerator is the number of “yes” patients; denominator is the number of ITT patients.
bPatients who died after achieving CMV DNA <200 copies/mL within 6 weeks were included as responders in the primary analysis; 5 patients who achieved CMV DNA <200 copies/mL within 6 weeks died within 42 days of the first dose of the study drug (fatal AEs: pneumonia [bacterial], n = 1; sepsis with multi-organ failure, n = 1; multi-organ failure, n = 2; post-transplant lymphoproliferative disorder, n = 1). None of these deaths was related to the study drug and none of the fatal AEs was likely to be related to CMV.
cNumerator is all recurrences; denominator is the number of patients achieving confirmed undetectable CMV DNA during the study, as specifically defined in the recurrence analysis.
dAny recurrence during the study, including early-withdrawal patients who had a recurrence before withdrawal from the study.
eDid not have recurrence during the study and had data after achieving confirmed undetectable CMV DNA, including early-withdrawal patients who did not have recurrence before withdrawal from the study.
fCalculated using the exact (Clopper-Pearson) confidence limits for the binomial proportion.
gFollow-up assessments through 12 weeks post-treatment.