A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Hemophilia B With Inhibitors (PRESent-3)

Conditions

Hemophilia B

Brief summary

Study Endpoint: Treated bleeds (expressed as annualized bleeding rate [ABR]) in the observation period and during the first 24 weeks under SerpinPC

Detailed description

• Treated bleeds (expressed as ABR) other than those defined by the primary endpoint (eg, during the first 48 weeks), • Treated spontaneous bleeds (expressed as ABR), • Treated spontaneous joint bleeds (expressed as ABR), • All bleeds requiring treatment (expressed as ABR; ie, all bleeds that would ordinarily be treated with factor concentrate/bypass agent if therapy were available), • Total coagulation factor and/or bypass product consumption during SerpinPC treatment, • PK concentrations of SerpinPC throughout the study, • Haemophilia Quality-of-Life Questionnaire for Adults (Haem-A-QoL) Physical Health scale in subjects aged 17 to ≤65 years

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 during the first 24 weeks under SerpinPC	—

Secondary

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

Measure

Time frame

• Treated bleeds (expressed as ABR) other than those defined by the primary endpoint (eg, during the first 48 weeks), • Treated spontaneous bleeds (expressed as ABR), • Treated spontaneous joint bleeds (expressed as ABR), • All bleeds requiring treatment (expressed as ABR; ie, all bleeds that would ordinarily be treated with factor concentrate/bypass agent if therapy were available), • Total coagulation factor and/or bypass product consumption during SerpinPC treatment, • PK concentrations of SerpinPC throughout the study, • Haemophilia Quality-of-Life Questionnaire for Adults (Haem-A-QoL) Physical Health scale in subjects aged 17 to ≤65 years

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Countries

France, Germany, Italy, Spain

Outcome results

None listed