Table 2. Summary of Treatment-Emergent Adverse Events (TEAEs) in Part 1 of the Clinical Trial of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Using BIIB112 (XIRIUS) Study.
| Parameter | All participants (n = 18) | Cohorts 3-6 (n = 12) | ||
|---|---|---|---|---|
| Participants, No. (%) | Events, No. | Participants, No. (%) | Events, No. | |
| All TEAEs | 18 (100) | 109 | 12 (100) | 84 |
| Non-ocular TEAEs | 9 (50) | 30 | 5 (42) | 23 |
| Ocular TEAEs | 18 (100) | 79 | 12 (100) | 61 |
| Dosed eye | 18 (100) | 73 | 12 (100) | 58 |
| Untreated fellow eye | 5 (28) | 6 | 3 (25) | 3 |
| Most common ocular TEAEs in the dosed eyea | ||||
| Increased intraocular pressure | 6 (33) | 10 | 5 (42) | 9 |
| Noninfective retinitis | 5 (28) | 8 | 5 (42) | 8 |
| Eye inflammation | 4 (22) | 5 | 4 (33) | 5 |
| Anterior chamber inflammation | 3 (17) | 3 | 1 (8) | 1 |
| Conjunctival hemorrhage | 3 (17) | 3 | 0 | 0 |
| Vision blurred | 3 (17) | 3 | 3 (25) | 3 |
| Severity of TEAEs in the dosed eye | ||||
| Mild | 10 (56) | 55 | 6 (50) | 42 |
| Moderate | 4 (22) | 14 | 4 (33) | 14 |
| Severe | 3 (17) | 4 | 1 (8) | 2 |
| All withdrawals related to TEAEs | 0 | 0 | 0 | 0 |
| All TEAEs related to treatment | 18 (100) | 64 | 12 (100) | 52 |
| TEAEs related to study drug in the dosed eye | 6 (33) | 16 | 6 (50) | 16 |
| TEAEs related to procedure in the dosed eye | 17 (94) | 35 | 11 (92) | 25 |
| Most common TEAEs related to treatment in the dosed eyeb | ||||
| Noninfective retinitis | 3 (17) | 5 | 3 (25) | 5 |
| Corneal deposits | 2 (11) | 2 | 2 (17) | 2 |
| All serious TEAEs | 5 (28) | 6 | 3 (25) | 4 |
| Serious TEAEs in the dosed eye | 5 (28) | 5 | 3 (25) | 3 |
| Visual acuity reduced | 2 (11) | 2 | 2 (17) | 2 |
| Noninfective retinitis | 1 (6) | 1 | 1 (8) | 1 |
| Retinal detachment | 1 (6) | 1 | 0 | 0 |
| Visual impairment | 1 (6) | 1 | 0 | 0 |
a
Occurring in more than 15% of all participants.
b
Occurring in more than 10% of all participants.