Table 1. Participant Demographics and Baseline Disease Characteristics in the Clinical Trial of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Using BIIB112 (XIRIUS) and Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS) Studies.
| Parameter | XIRIUS (cohorts 3-6) (n = 12) | XOLARIS subgroup meeting criteria for XIRIUS (n = 103) |
|---|---|---|
| Age, mean (range), y | 30.6 (20.7-50.7) | 30.8 (18.0-84.0) |
| Race, No. (%) | ||
| Asian | 0 | 4 (3.9) |
| Black or African American | 0 | 1 (1.0) |
| Native Hawaiian or other Pacific Islander | 0 | 1 (1.0) |
| White | 12 (100) | 96 (93.2) |
| Ethnicity, No. (%) | ||
| Hispanic or Latino | 1 (8.3) | 4 (3.9) |
| Not reported | 0 | 1 (1.0) |
| Retinal sensitivity, mean (SD), dB (16 central loci) | ||
| Study eyea | 7.8 (5.9) | 8.8 (6.7) |
| Untreated fellow eye | 9.1 (6.2) | 9.1 (6.5) |
| Retinal sensitivity, mean (SD), dB (68 central loci) | ||
| Study eyea | 3.9 (6.4) | 4.4 (4.5) |
| Untreated fellow eye | 4.6 (7.2) | 4.7 (4.6) |
| BCVA, ETDRS letters, mean (SD) [approximate Snellen equivalent] b | ||
| Study eyea | 66.5 (11.8) [20/40 to 20/50] | 63.2 (10.9) [20/50 to 20/63] |
| Untreated fellow eye | 69.3 (11.2) [20/40 to 20/50] | 67.5 (10.1) [20/40 to 20/50] |
| Ellipsoid zone width, mean (SD), μm | ||
| Study eyea | 966.8 (1623.0) | 543.3 (680.8) |
| Untreated fellow eye | 1379.1 (2462.7) | 611.1 (701.5) |
| Ellipsoid zone area, mean (SD), mm2 | ||
| Study eyea | 2.9 (7.2) | 0.5 (0.8) |
| Untreated fellow eye | 5.4 (13.0) | 0.6 (0.9) |
Abbreviations: BCVA, best-corrected visual acuity; ETDRS, Early Treatment Diabetic Retinopathy Study.
a
In the XIRIUS trial part 1, the study eye was the dosed eye.
b
Snellen equivalent approximate is given as ranges on the basis of previously published conversion tables.12