Table 2.
Summary of TEAEsa by treatment group at week 52 in the phase 2/3 study (safety population).
| Overall safety population |
Patients in the safety population aged <6 years at baseline with missense IDS variants |
|||
|---|---|---|---|---|
| Idursulfase-IT (n = 33) | No idursulfase-IT (n = 15) | Idursulfase-IT (n = 12) | No idursulfase-IT (n = 6) | |
| At least one AE | 33 (100.0) | 14 (93.3) | 12 (100) | 6 (100) |
| At least one severe AE | 3 (9.1) | 0 | 0 | 0 |
| At least one SAE | 12 (36.4) | 2 (13.3) | 4 (33.3) | 1 (16.7) |
| At least one life-threatening AE | 0 | 0 | 0 | 0 |
| Discontinuation due to an AE | 0 | 0 | 0 | 0 |
| Deaths | 0 | 0 | 0 | 0 |
| At least one AE related to IV idursulfase infusion | 8 (24.2) | 6 (40.0) | 3 (25.0) | 3 (50.0) |
| At least one AE related to idursulfase-IT | 25 (75.8) | – | 10 (83.3) | – |
| At least one IDDD surgical procedure related AE | 29 (87.9) | – | 12 (100) | – |
| At least one IDDD-related AE | 22 (66.7) | – | 9 (75.0) | – |
| At least one IT administration process-related AE | 22 (66.7) | – | 10 (83.3) | – |
| Associated with administration via IDDD | 18 (54.5) | – | 9 (75.0) | – |
| Associated with administration via lumbar puncture | 9 (27.3) | – | 5 (41.7) | – |
| Most common TEAEs by preferred term | ||||
| Vomiting | 25 (75.8) | 3 (20.0) | 9 (75.0) | 2 (33.3) |
| Pyrexia | 20 (60.6) | 7 (46.7) | 6 (50.0) | 3 (50.0) |
| Cough | 15 (45.5) | 4 (26.7) | 6 (50.0) | 3 (50.0) |
| Procedural pain | 14 (42.4) | – | 5 (41.7) | – |
| Nasopharyngitis | 13 (39.4) | 5 (33.3) | 5 (41.7) | 4 (66.7) |
| Headache | 12 (36.4) | 1 (6.7) | 4 (33.3) | 1 (16.7) |
| Diarrhea | 10 (30.3) | 2 (13.3) | 4 (33.3) | 1 (16.7) |
| Ear infection | 9 (27.3) | 4 (26.7) | 3 (25.0) | 1 (16.7) |
| Heart rate increased | 7 (21.2) | 4 (26.7) | 3 (25.0) | 2 (33.3) |
Data are shown as number of patients (%) reporting at least one AE.
AE, adverse event; EOS, end of study; IDDD, intrathecal drug delivery device; IDS, iduronate-2-sulfatase gene; IT, intrathecal; IV, intravenous; SAE, serious adverse event; TEAE, treatment-emergent AE.
TEAEs for the no idursulfase-IT group are defined as all AEs occurring on or after the date of randomization and on or before the EOS visit TEAEs for the idursulfase-IT group are defined as all AEs occurring on or after the date of the first IDDD implantation surgery or first dose (whichever is earlier) and on or before the EOS visit (+ 30 days) or 2 weeks after the removal of the last IDDD (whichever is later).