Characteristics of the 6 clinical trials included in the data analysis
| Study number . | Description . | Patient age∗ range (y) . | Number of patients† . | Rurioctocog alfa pegol treatment . | Reference . |
|---|---|---|---|---|---|
| NCT01599819 | Phase 1: safety and PK | 18-65 | 19 | 30 ± 3 IU/kg and 60 ± 6 IU/kg for PK | 25 |
| NCT01736475 | Phase 2/3: efficacy, safety, and PK | 12-65 | 137 | 45 ± 5 IU/kg twice weekly for prophylaxis and PK 10-60 ± 5 IU/kg for on-demand treatment | 25 |
| NCT02210091 | Phase 3: PK, safety, efficacy, and immunogenicity | <12 | 66 | 50 ± 10 IU/kg twice weekly for prophylaxis 60 ± 5 IU/kg for PK | 34 |
| NCT01913405 | Phase 3: safety and efficacy in surgery | 12-75 (newly recruited patients)‡ | 22 | Tailored dose to achieve FVIII target levels of:
| 35 |
| NCT01945593 | Phase 3b: long-term safety and efficacy | ≤75 | 216 | Fixed-dose prophylaxis twice weekly, with option to switch to either every 5 or every 7 d§; or to PK-tailored prophylaxis maintaining a FVIII trough level ≥3%|| | 36 |
| NCT02585960 | Phase 3: safety and efficacy following PK-guided prophylaxis | 12-65 | 120 | Dosing based on PK to maintain FVIII trough levels of either:
| 37 |
| Study number . | Description . | Patient age∗ range (y) . | Number of patients† . | Rurioctocog alfa pegol treatment . | Reference . |
|---|---|---|---|---|---|
| NCT01599819 | Phase 1: safety and PK | 18-65 | 19 | 30 ± 3 IU/kg and 60 ± 6 IU/kg for PK | 25 |
| NCT01736475 | Phase 2/3: efficacy, safety, and PK | 12-65 | 137 | 45 ± 5 IU/kg twice weekly for prophylaxis and PK 10-60 ± 5 IU/kg for on-demand treatment | 25 |
| NCT02210091 | Phase 3: PK, safety, efficacy, and immunogenicity | <12 | 66 | 50 ± 10 IU/kg twice weekly for prophylaxis 60 ± 5 IU/kg for PK | 34 |
| NCT01913405 | Phase 3: safety and efficacy in surgery | 12-75 (newly recruited patients)‡ | 22 | Tailored dose to achieve FVIII target levels of:
| 35 |
| NCT01945593 | Phase 3b: long-term safety and efficacy | ≤75 | 216 | Fixed-dose prophylaxis twice weekly, with option to switch to either every 5 or every 7 d§; or to PK-tailored prophylaxis maintaining a FVIII trough level ≥3%|| | 36 |
| NCT02585960 | Phase 3: safety and efficacy following PK-guided prophylaxis | 12-65 | 120 | Dosing based on PK to maintain FVIII trough levels of either:
| 37 |
Patient age per the study protocol.
Number of patients enrolled in each study who received ≥1 infusion of rurioctocog alfa pegol. Patients could participate in ≥1 of the 6 studies.
Per protocol, patients <12 years of age who were either participating in NCT02210091 or had completed NCT02210091 and transitioned into NCT01945593 could also be enrolled.
Patients aged ≥12 years who had no spontaneous bleeds for 6 months had the option to switch to fixed-dose prophylaxis every 5 days and subsequently to fixed-dose prophylaxis every 7 days if they had no spontaneous bleeds for a further 6 months.
Option to choose fixed-dose or PK-tailored prophylaxis at patient or physician discretion.