Table 1.
Demographics and Baseline Characteristics
| Characteristic | All Participants (n = 12) | Dose Group |
|||
|---|---|---|---|---|---|
| 50 μg (n = 3) | 100 μg (n = 3) | 250 μg (n = 3) | 500 μg (n = 3) | ||
| Age (yrs) | |||||
| Mean ± SD | 81.5 ± 6.2 | 84.7 ± 8.3 | 79.7 ± 2.1 | 77.0 ± 7.0 | 84.7 ± 5.0 |
| Range | 70–94 | 78–94 | 78–84 | 70–84 | 80–90 |
| Female sex, no. (%) | 9 (75.0) | 2 (66.7) | 3 (100.0) | 3 (100.0) | 1 (33.3) |
| Tobacco amount (pack-years), no. (%) | |||||
| Former | 7 (58.3) | 1 (33.3) | 2 (66.7) | 2 (66.7) | 2 (66.7) |
| Never | 5 (41.7) | 2 (66.7) | 1 (33.3) | 1 (33.3) | 1 (33.3) |
| Genetics, no. (%) | |||||
| Y402H/Y402H | 6 (50) | 2 (67) | 2 (67) | 2 (67) | 0 (0) |
| Y402H/Y402Y | 2 (17) | 0 (0) | 1 (33) | 1 (33) | 0 (0) |
| Y402Y/Y402Y | 4 (33) | 1 (33) | 0 (0) | 0 (0) | 3 (100) |
| GA lesion size (mm2), study eye (fellow eye)∗ | |||||
| Mean ± SD | 9.5 ± 11.8 (7.1 ± 9.3) | 6.7 ± 4.3 (5.8 ± 8.0) | 14.3 ± 18.2 (12.3 ± 15.6) | 13.2 ± 17.2 (5.0 ± 3.5) | 3.7 ± 0.9 (2.8 ± 1.7) |
| Range | 1.7–35.3 (0.2–29.9) | 2.2–10.8 (0.2–11.4) | 2.6–35.3 (0.2–29.9) | 1.7–33.0 (2.5–7.5) | 2.9–4.7 (1.6–4.0) |
| BCVA (ETDRS letters), study eye (fellow eye) | |||||
| Mean ± SD | 39.7 ± 6.0 (54.5 ± 23.1) | 44.0 ± 6.1 (53.0 ± 29.6) | 35.7 ± 9.3 (52.0 ± 25.2) | 40.3 ± 3.1 (58.7 ± 32.6) | 38.7 ± 3.5 (54.3 ± 17.9) |
| Range | 28–51 (21–84) | 40–51 (25–84) | 28–46 (35–81) | 37–43 (21–78) | 35–42 (43–75) |
| LLVA (ETDRS), study eye (fellow eye) | |||||
| Mean ± SD | 29.3 ± 7.7 (38.2 ± 16.4) | 34.0 ± 4.6 (37.0 ± 14.1) | 32.0 ± 12.1 (34.3 ± 18.9) | 27.7 ± 2.5 (43.3 ± 26.6) | 23.3 ± 7.0 (38.0–12.1) |
| Range | 16–43 (14–66) | 30–39 (22–50) | 19–43 (18–55) | 25–30 (14–66) | 16–30 (27–51) |
| AH CFH (ng/ml)† | |||||
| Mean ± SD | 134.3 ± 97.0 | 102.1 ± 24.8 | 161.0 ± 100.4 | 89.4 ± 61.7 | 210.1 ± 205.7 |
| Range | 50–356 | 76–126 | 56–256 | 50–161 | 65–356 |
| Plasma CFH (ng/ml) | |||||
| Mean ± SD | 284 252.6 ± 53 681.5 | 304 275.0 ± 51 570.0 | 228 260.2 ± 23 392.9 | 318 468.1 ± 27 152.3 | 286 007.3 ± 69 574.9 |
| Range | 204 793–363 540 | 269 622–363 540 | 204 793–251 578 | 290 996–345 289 | 208 507–343 087 |
| AH Ba (ng/ml)‡ | |||||
| Mean ± SD | 21.6 ± 10.6 | 26.5 ± 10.7 | 25.4 ± 13.8 | 17.8 ± 9.7 | 12.2 ± NA |
| Range | 8–41 | 19–34 | 16–41 | 8–28 | 12 |
| AH C3a (ng/ml)† | |||||
| Mean ± SD | 5.4 ± 2.4 | 4.6 ± 2.2 | 5.2 ± 1.3 | 5.2 ± 3.5 | 7.6 ± 2.9 |
| Range | 1.5–9.6 | 2.2–6.6 | 4.4–6.9 | 1.5–8.6 | 5.6–9.6 |
| AH CFB (ng/ml)† | |||||
| Mean ± SD | 545.7 ± 305.4 | 447.2 ± 311.9 | 558.7 ± 223.1 | 500.1 ± 405.1 | 742.4 ± 443.8 |
| Range | 166–1056 | 169–785 | 301–694 | 166–951 | 429–1056 |
| AH C3 (ng/ml)† | |||||
| Mean ± SD | 598.8 ± 414.3 | 926.9 ± 272.6 | 611.4 ± 510.7 | 216.3 ± 76.0 | 661.6 ± 513.9 |
| Range | 131–1181 | 617–1129 | 196–1181 | 131–277 | 298–1025 |
AH = aqueous humor; BCVA = best-corrected visual acuity; CFB = complement factor B; CFH = complement factor H; C3(a) = complement component 3(a); GA = geographic atrophy; LLVA = low-luminance visual acuity; NA = not applicable; SD = standard deviation.
All participants were White; 8% were Hispanic/Latino. All AH biomarker data are in relationship to the study eye. Data are in relationship to all participants unless otherwise stated. Demographic information and patient characteristics at baseline show a total of 12 participants, 3 in each of the following dose groups: 50 μg, 100 μg, 250 μg, or 500 μg. Characteristics include age, sex, tobacco history, genetic information, GA lesion size, visual acuity, and biomarker measurements at baseline.
Data were not available for 1 participant each in the 50-μg, 250-μg, and 500-μg dose groups.
Data were not available for 1 participant in the 500-μg dose group.
Data were not available for 1 participant in the 50-μg dose group and 2 participants in the 500-μg dose group.