A Phase 3, single-arm, multicenter, multinational, open-label, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged ≥ 12 years with severe hemophilia A or B with or without inhibitory antibodies to factor VIII or IX

Conditions

Hemophilia

Brief summary

Annualized Bleeding Rate (ABR) in the fitusiran primary efficacy period

Detailed description

Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on prophylaxis standard of care (SOC) in the SOC period, Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on on-demand standard of care (SOC) in the SOC period, Annualized spontaneous bleeding rate in the fitusiran primary efficacy period and in the SOC period, Annualized joint bleeding rate in the fitusiran primary efficacy period and in the SOC period, Change in Haem-A-QOL physical health score and total score during SOC and during fitusiran prophylaxis, Annualized bleeding rate (ABR) in the fitusiran 18-month efficacy period, Annualized bleeding rate (ABR) in the fitusiran 36-month treatment period, Annualized weight-adjusted consumption of CFC/BPA, Number of participants with adverse events

Related papers

No related papers found yet.

Papers published before 2008-02-04 may not appear yet. Historical indexing is in progress.

Interventions

DRUGFACTOR VIII INHIBITOR BYPASSING ACTIVITY

DRUGANTITHROMBIN III

DRUGCOAGULATION FACTOR IX

DRUGSAR439774

DRUGCOAGULATION FACTOR VIII

DRUGEPTACOG ALFA (ACTIVATED)

Eligibility

Sex/Gender

Male

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Annualized Bleeding Rate (ABR) in the fitusiran primary efficacy period	—

Secondary

Measure	Time frame
Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on prophylaxis standard of care (SOC) in the SOC period, Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on on-demand standard of care (SOC) in the SOC period, Annualized spontaneous bleeding rate in the fitusiran primary efficacy period and in the SOC period, Annualized joint bleeding rate in the fitusiran primary efficacy period and in the SOC period, Change in Haem-A-QOL physical health score and total score during SOC and during fitusiran prophylaxis, Annualized bleeding rate (ABR) in the fitusiran 18-month efficacy period, Annualized bleeding rate (ABR) in the fitusiran 36-month treatment period, Annualized weight-adjusted consumption of CFC/BPA, Number of participants with adverse events	—

Measure

Time frame

Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on prophylaxis standard of care (SOC) in the SOC period, Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on on-demand standard of care (SOC) in the SOC period, Annualized spontaneous bleeding rate in the fitusiran primary efficacy period and in the SOC period, Annualized joint bleeding rate in the fitusiran primary efficacy period and in the SOC period, Change in Haem-A-QOL physical health score and total score during SOC and during fitusiran prophylaxis, Annualized bleeding rate (ABR) in the fitusiran 18-month efficacy period, Annualized bleeding rate (ABR) in the fitusiran 36-month treatment period, Annualized weight-adjusted consumption of CFC/BPA, Number of participants with adverse events

—

Countries

France, Germany, Greece, Italy, Poland, Spain

Outcome results

None listed