Table 1.
Characteristics of patients at baselinea
| Placebo | MAPTRx | |||||
|---|---|---|---|---|---|---|
| Characteristic | (N = 12) | MAPTRx groups (N = 34) | 10 mg monthly (N = 6) | 30 mg monthly (N = 6) | 60 mg monthly Q4W (N = 9) | 115 mg quarterly (N = 13) |
| Age, years | 66 ± 4.6 | 66 ± 6.1 | 64 ± 5.2 | 65 ± 6.1 | 66 ± 6.8 | 67 ± 6.3 |
| Age at diagnosis, years | 65 ± 4.6 | 64 ± 6.4 | 62 ± 5.2 | 64 ± 6.9 | 64 ± 6.6 | 65 ± 6.9 |
| Female, no. (%) | 6 (50) | 17 (50) | 2 (33) | 4 (67) | 5 (56) | 6 (46) |
| Race—white, no. (%) | 12 (100) | 34 (100) | 6 (100) | 6 (100) | 9 (100) | 13 (100) |
| MMSE Total Score | 24.2 ± 1.7 | 23.5 ± 2.4 | 21.5 ± 1.6 | 24.5 ± 1.4 | 24.6 ± 2.5 | 23.2 ± 2.5 |
| RBANS Total Score | 64.9 ± 10.2 | 68.2 ± 12.1 | 58.8 ± 11.2 | 69.2 ± 12.1 | 69.9 ± 9.1 | 70.9 ± 13.4 |
| CDR Global Score, no. (%) | ||||||
| 0.5 | 7 (58) | 23 (68) | 0 (0) | 3 (50) | 9 (100) | 11 (85) |
| 1 | 5 (42) | 11 (32) | 6 (100) | 3 (50) | 0 (0) | 2 (15) |
| CDR Sum of Boxes | 4.1 ± 1.3 | 3.7 ± 1.1 | 4.8 ± 0.5 | 4.7 ± 1.0 | 2.9 ± 0.6 | 3.3 ± 1.1 |
| Concomitant medications, no. (%) | ||||||
| Anticholinesterases | 7 (58) | 21 (62) | 4 (67) | 5 (83) | 4 (44) | 8 (62) |
| Memantine | 1 (8) | 7 (21) | 2 (33) | 0 (0) | 3 (33) | 2 (15) |
| Estrogen replacement | 0 (0) | 3 (9) | 1 (17) | 0 (0) | 1 (11) | 1 (8) |
| APOE4 carrier (%) | 8 (67) | 25 (74) | 5 (83) | 3 (50) | 6 (67) | 11 (85) |
| Homozygous | 2 (16.7) | 8 (23.5) | 2 (33.3) | 0 (0) | 3 (33.3) | 3 (23.1) |
| Heterozygous | 6 (50) | 17 (50) | 3 (50) | 3 (50) | 3 (33.3) | 8 (61.5) |
| CSF t-tau (λpg ml−1) | 387.3 ± 120.9 | 405.6 ± 132.7 | 364.6 ± 98.1 | 386.4 ± 152.3 | 391.0 ± 111.8 | 443.4 ± 153.8 |
| p-tau181 (pg ml−1) | 38.7 ± 13.0 | 40.7 ± 14.2 | 39.1 ± 13.0 | 38.6 ± 16.6 | 39.5 ± 12.6 | 43.2 ± 15.9 |
| t-tau/Aβ42 | 0.6 ± 0.2 | 0.6 ± 0.2 | 0.6 ± 0.2 | 0.6 ± 0.1 | 0.5 ± 0.1 | 0.6 ± 0.2 |
aPlus–minus values are mean ± standard deviation. Patients were assigned to receive either placebo or ascending doses of the ASO MAPTRx. Percentages may not total 100 because of rounding. RBANS, Repeatable Battery for the Assessment of Neuropsychological Status; APOE4, apolipoprotein epsilon 4.