Table 5.
Efficacy Analysis by Cohort, Final Dose Population, Week 24 and Week 48, Observed Failure Approach
| Parameter | Cohort I (n = 59) |
Cohort IIA (n = 4) |
Cohort IIB (n = 13) |
Cohort III (n = 20) |
Total (N = 96) |
|||||
|---|---|---|---|---|---|---|---|---|---|---|
| no./No. | % (95% CI) | no./No. | % (95% CI) | no./No. | % (95% CI) | no./No. | % (95% CI) | no./No. | % (95% CI) | |
| Proportion with HIV RNA with ≥1 log10 decline or <400 copies/mL | ||||||||||
| Week 24 | 42/58 | 72.4 (59.1, 83.3) | 2/4 | 50 (6.8, 93.2) | 10/13 | 76.9 (46.2, 95) | 14/20 | 70 (45.7, 88.1) | 68/95 | 71.6 (61.4, 80.4) |
| Week 48 | 42/56 | 75 (61.6, 85.6) | 3/4 | 75 (19.4, 99.4) | 10/11 | 90.9 (58.7, 99.8) | 17/20 | 85 (62.1, 96.8) | 72/91 | 79.1 (69.3, 86.9) |
| Proportion with HIV RNA <50 copies/mL | ||||||||||
| Week 24 | 32/58 | 55.2 (41.5, 68.3) | 2/4 | 50 (6.8, 93.2) | 7/13 | 53.8 (25.1, 80.8) | 10/20 | 50 (27.2, 72.8) | 51/95 | 53.7 (43.2, 64) |
| Week 48 | 32/56 | 57.1 (43.2, 70.3) | 2/4 | 50 (6.8, 93.2) | 6/11 | 54.5 (23.4, 83.3) | 12/20 | 60 (36.1, 80.9) | 52/91 | 57.1 (46.3, 67.5) |
| Proportion with HIV RNA <400 copies/mL | ||||||||||
| Week 24 | 40/58 | 69 (55.5, 80.5) | 2/4 | 50 (6.8, 93.2) | 9/13 | 69.2 (38.6, 90.9) | 12/20 | 60 (36.1, 80.9) | 63/95 | 66.3 (55.9, 75.7) |
| Week 48 | 39/56 | 69.6 (55.9, 81.2) | 2/4 | 50 (6.8, 93.2) | 10/11 | 90.9 (58.7, 99.8) | 16/20 | 80 (56.3, 94.3) | 67/91 | 73.6 (63.3, 82.3) |
| Change from baseline in CD4 cell count, cells/µL | Mean | (95% CI) | Mean | (95% CI) | Mean | (95% CI) | Mean | (95% CI) | Mean | (95% CI) |
| Week 24 | 114.4 | (73.7, 155.1) | −35.8 | (−348.8, 277.3) | 143.4 | (−12.9, 299.6) | 147.2 | (−2.7, 297.1) | 119.0 | (74.9, 163.1) |
| Week 48 | 168.2 | (117.5, 218.9) | 189.5 | (−154.2, 533.2) | 76.8 | (−85.3, 238.9) | 158.1 | (11.7, 304.4) | 155.7 | (108.3, 203.1) |
| Change from baseline in CD4 percentage | ||||||||||
| Week 24 | 4.1 | (2.8, 5.3) | 2.2 | (−7.2, 11.5) | 0.8 | (−3.6, 5.2) | 5.3 | (2.9, 7.7) | 3.8 | (2.7, 4.9) |
| Week 48 | 5.2 | (3.9, 6.6) | 6.0 | (−2.6, 14.6) | 1.6 | (−2.7, 5.9) | 4.3 | (1, 7.6) | 4.6 | (3.4, 5.8) |
For binary endpoints: no./No. with % (95% CI) was reported for each cohort, where no./No. = number of responders/number of patients. For continuous endpoints: mean change with (95% CI) was reported. Normal distributions were assumed for continuous endpoints. Observed failure approach for handling missing data: for binary endpoints, missing values were considered as failures for patients missing data due to discontinuation of study treatment for lack of efficacy or for non-treatment-related reasons with last available HIV RNA value <1 log10 drop from baseline and ≥400 copies/mL; otherwise patients with missing values were excluded. For continuous endpoints (eg, change from baseline in CD4 cell counts and percent), baseline values were carried forward for patients missing data due to discontinuation of study treatment for lack of efficacy or for non-treatment-related reasons with last available HIV RNA value <1 log10 drop from baseline and ≥400 copies/mL; otherwise patients with missing values were excluded.
Abbreviations: CI, confidence interval; HIV, human immunodeficiency virus.