Table 2 Vector- and surgery-related AEs by dose and severity
From: Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial
AEs | Grade 1 | Grade 2 | Grade 3 | Total | Description |
|---|---|---|---|---|---|
Vector-related | |||||
Starting dose | 0 | 0 | 0 | 0 | |
Low | 1 | 2 | 1 | 4 | LFT (2, 1 with +ELISpot), movement disorder (2) |
Mid | 0 | 5 | 1 | 6 | LFT (3), +ELISpot (2), worsening dystonia (1) |
High | 1 | 4 | 0 | 5 | LFT (2), +ELISpot (2), transient abnormal movements |
Total | 2 | 11 | 2 | 15 | |
Surgery-related | |||||
Starting dose | 1 | 1 | 0 | 2 | Fever, rigors |
Low | 1 | 1 | 2 | 4 | Congestion, wound dehiscence (2), unexpected progression |
Mid | 0 | 3 | 1 | 4 | Wound dehiscence (2), other wound issues (2) |
High | 2 | 4 | 0 | 6 | Other wound issues (2) |
Total | 4 | 9 | 3 | 16 |
