Table 2.
Screening | Lead-In Phase | Treatment and Maintenance Phase | ||||
---|---|---|---|---|---|---|
Patient | Pegademase regimen, no. of injections/week |
Pegademase, U/kga | Elapegademase, mg/kgb | Treatment duration, years | Elapegademase self-dosingc | |
Start | End | |||||
1d | 2 | 43.9 | 43.4 | 0.28 | 0.1 | No |
2 | 2 | 28.2 | 27.9 | 0.19 | 4.2 | Yes |
3 | 1 | 29.6 | 36.1 | 0.23 | 2.4 | No |
4 | 2 | 7.7 | 30.0 | 0.20 | 4.2 | No |
5 | 1 | 31.3 | 32.2 | 0.21 | 2.4 | No |
6 | 2 | 42.9 | 44.1 | 0.29 | 2.1 | Yes |
7 | 3 | 21.6 | 26.2 | 0.17 | 1.4 | Yes |
aTotal dose for the week
bElapegademase dosage was based on pegademase dosage, using a conversion factor for enzyme equivalent activity
cElapegademase self-dosing at home was allowed during the Treatment Phase from weeks 12, 14, 16, 18, and 20 and during the Maintenance Phase until the end of the study
dPatient 1 received 2 doses of elapegademase and withdrew due to a treatment-emergent adverse effect
1 mg elapegademase = 150 U pegademase