A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)

Conditions

Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Brief summary

Study Endpoint: Treated bleeds (expressed as annualized bleeding rate [ABR]) in the observation period and under SerpinPC at the end of Part 2 Week 24 in subjects with HemB previously receiving “on-demand” treatment

Detailed description

Treated bleeds (expressed as ABR) other than those defined by the primary endpoint (eg, bleeds during the first 48 weeks, and in those subjects receiving prior “prophylaxis” treatment), Treated spontaneous bleeds (expressed as ABR), Treated spontaneous joint bleeds (expressed as ABR), All bleeds requiring treatment (expressed as ABR, ie, all treated bleeds and all bleeds that would ordinarily be treated with factor concentrate/bypass agent if therapy were available), Total coagulation factor and/or bypass product consumption, PK concentrations of SerpinPC throughout the study, Haemophilia-specific QoL Instrument for Adults (Haem-A-QoL) Physical Health scale in subjects aged 17 to ≤65 years with hemophilia and the end of Part 3 Week 24).

Related papers

No related papers found yet.

Interventions

DRUGSerpinPC

Eligibility

Sex/Gender

Male

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Study Endpoint: Treated bleeds (expressed as annualized bleeding rate [ABR]) in the observation period and under SerpinPC at the end of Part 2 Week 24 in subjects with HemB previously receiving “on-demand” treatment	—

Secondary

Measure	Time frame
Treated bleeds (expressed as ABR) other than those defined by the primary endpoint (eg, bleeds during the first 48 weeks, and in those subjects receiving prior “prophylaxis” treatment), Treated spontaneous bleeds (expressed as ABR), Treated spontaneous joint bleeds (expressed as ABR), All bleeds requiring treatment (expressed as ABR, ie, all treated bleeds and all bleeds that would ordinarily be treated with factor concentrate/bypass agent if therapy were available), Total coagulation factor and/or bypass product consumption, PK concentrations of SerpinPC throughout the study, Haemophilia-specific QoL Instrument for Adults (Haem-A-QoL) Physical Health scale in subjects aged 17 to ≤65 years with hemophilia and the end of Part 3 Week 24).	—

Measure

Time frame

Treated bleeds (expressed as ABR) other than those defined by the primary endpoint (eg, bleeds during the first 48 weeks, and in those subjects receiving prior “prophylaxis” treatment), Treated spontaneous bleeds (expressed as ABR), Treated spontaneous joint bleeds (expressed as ABR), All bleeds requiring treatment (expressed as ABR, ie, all treated bleeds and all bleeds that would ordinarily be treated with factor concentrate/bypass agent if therapy were available), Total coagulation factor and/or bypass product consumption, PK concentrations of SerpinPC throughout the study, Haemophilia-specific QoL Instrument for Adults (Haem-A-QoL) Physical Health scale in subjects aged 17 to ≤65 years with hemophilia and the end of Part 3 Week 24).

—

Countries

Belgium, Bulgaria, France, Germany, Italy, Poland, Spain

Outcome results

None listed