Table 1.
Overview of the octanate® clinical trial programme.
| Parameter | AVI-401 | AVI-402 | AVI-403 | AVI-406 | AVI-407 | AVI-408 |
|---|---|---|---|---|---|---|
| Phase | II/III | II/III | III | II | III | III |
| Study period | December 1997 – July 1998 | December 1997 – October 1998 | February 2000 – December 2015 | August 1999 – February 2000 | February 2000 – October 2000 | December 2000 – June 2003 |
| Primary objective(s) | Pharmacokinetics | Pharmacokinetics (phase I); efficacy in prevention/treatment of bleeds and surgical prophylaxis (phase II) | Immunogenicity | Pharmacokinetics | Immunogenicity | Immunogenicity |
| Secondary objectives | Efficacy in prevention/treatment of bleeds and surgical prophylaxis, immunogenicity, viral safety, tolerability | Immunogenicity, viral safety, tolerability | Efficacy in prevention/treatment of bleeds and surgical prophylaxis, IVR (optional), viral safety, tolerability | Efficacy in prevention/treatment of bleeds, immunogenicity, viral safety, tolerability | Efficacy in prevention/treatment of bleeds, IVR, viral safety, tolerability | Efficacy in prevention/treatment of bleeds and surgical prophylaxis, IVR, viral safety, tolerability |
| Patients | PTPs | PTPs | PUPs | PTPs | PTPs | PTPs |
| N | 10 + 2a | 22 + 6a | 51 | 12 | 18 | 15 |
| Age, years | ⩾16 years | ⩾12 years | 0.6 (0.01–5.6) yearsb | ⩾12 years | ⩾12 and ⩽65 years | <6 years |
| FVIII:C | <2% | <2% | <2% | ⩽1% | ⩽2% | <2% |
| Previous EDs to FVIII | ⩾100 | ⩾100 | 0 | ⩾100 | ⩾150 | 60 (12–100)b |
a
Additional patients who participated in the AVI-401/402 surgery study.
b
Median (range) provided as no cut-off for this parameter was specified by the inclusion criteria.
ED, exposure day; FVIII, coagulation factor VIII; FVIII:C, FVIII coagulant activity; IVR, in vivo recovery; PTP, previously treated patient; PUP, previously untreated patient.