5: Five studies providing efficacy data. Design Patients Primary Secondary objectives objectives Phase II, 22 adult Determine Calculate prospective, PTPs the PKs of incremental randomised, enrolled: all simoctocog recovery; II 22 in ITT alfa and Investigate and SAF compare immunogenicity; open-label, populations with PKs of Assess clinical multi-centre. and 14 in full-length efficacy and safety the PP rFVIII BHK in the treatment of Start: population BEs and in surgical 27/04/2012 prophylaxis. End: 18/09/2012. Phase III, 32 Determine Calculate prospective, PTPs the efficacy incremental multinational, enrolled: all of recovery; multicentre, 32 in ITT simoctocog III Investigate open-label, and SAF alfa during immunogenicity; single-arm. prophylactic Assess safety. treatment, Start: treatment of 22/07/2010. BEs and in End: surgical 31/01/2012. prophylaxis. Phase III, 59 Assess Determine PKs; prospective, paediatric clinical Determine multinational, PTPs efficacy of incremental multicentre, enrolled; all simoctocog III recovery; open-label, 59 patients alfa in terms Investigate were of prevention immunogenicity; analysed & treatment for efficacy; of Assess efficacy in Start: patients surgical 27/12/2010. aged from 2 BEs. procedures; End: to 12 Assess safety by AE 06/11/2012. (inclusive). monitoring. Phase II, 22 adult Determine Calculate • prospective, PTPs the PKs of incremental single-centre, enrolled: all simoctocog recovery. open-label, 22 were alfa and II Investigate randomised included in compare immunogenicity. cross-over PK the with Assess clinical part and analyses previously efficacy and safety uncontrolled (safety, ITT, used during efficacy part. PROPH rFVIII BHK.
5: Five studies providing efficacy data. Design Patients Primary objectives Secondary objectives II Phase II, prospective, randomised, open-label, multi-centre. Start: 27/04/2012 End: 18/09/2012. 22 adult PTPs enrolled: all 22 in ITT and SAF and 14 in the PP population Determine the PKs of simoctocog alfa and compare with PKs of full-length rFVIII BHK Calculate incremental recovery; Investigate immunogenicity; Assess clinical efficacy and safety in the treatment of BEs and in surgical prophylaxis. III Phase III, prospective, multinational, multicentre, open-label, single-arm. Start: 22/07/2010. End: 31/01/2012. 32 PTPs enrolled: all 32 in ITT and SAF Determine the efficacy of simoctocog alfa during prophylactic treatment, treatment of BEs and in surgical prophylaxis. Calculate incremental recovery; Investigate immunogenicity; Assess safety. III Phase III, prospective, multinational, multicentre, open-label, Start: 27/12/2010. End: 06/11/2012. 59 paediatric PTPs enrolled; all 59 patients were analysed for efficacy; patients aged from 2 to 12 (inclusive). Assess clinical efficacy of simoctocog alfa in terms of prevention & treatment of BEs. Determine PKs; Determine incremental recovery; Investigate immunogenicity; Assess efficacy in surgical procedures; Assess safety by AE monitoring. II Phase II, prospective, single-centre, open-label, randomised cross-over PK part and uncontrolled efficacy part. 22 adult PTPs enrolled: all 22 were included in the analyses (safety, ITT, PROPH • Determine the PKs of simoctocog alfa and compare with previously used rFVIII BHK. Calculate incremental recovery. Investigate immunogenicity. Assess clinical efficacy and safety during populations
auspar-simoctocog-alfa-rhu-160607.docx