This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
Patients with AHA who are eligible will receive two loading doses of the study drug, emicizumab (6mg/kg on day 1 and 3 mg/kg on day 2) followed by once weekly subcutaneous emicizumab (1.5 mg/kg). Immunosuppression will be given concurrently as per investigator discretion. The primary endpoint (bleed rate) will be assessed after 12 weeks on study drug. If partial remission of the AHA has not been achieved, an additional 12 weeks of study drug may be given. A historical cohort and a study conducted in parallel in Germany (NCT04188639) will serve as control groups for evaluation of secondary endpoints provided the study cohort are comparable.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
51
This study design uses emicizumab as a prophylaxis treatment to prevent bleeding for all participants, bypassing agents (with the exception of aPCC) and treatment of concomitant diseases will be given as clinically indicated. All eligible subjects with AHA will receive the same study medication consisting of: two loading doses of the emicizumab on day 1 and 2 followed by once weekly subcutaneous emicizumab injections. Immunosuppressive therapy (IST) will be given concurrently as per investigator discretion.
UCSD Hemophilia and Thrombosis Treatment Center
San Diego, California, United States
Georgetown University
Washington D.C., District of Columbia, United States
Primary Outcome Meassure
Number of clinically significant bleeds after 12 weeks of study drug (emicizumab)
Time frame: 12 weeks
Incidence and severity of adverse events
Incidence and severity of adverse events, including thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab treatment; mortality and cause of death in the 24 weeks after starting emicizumab treatment.
Time frame: duration of entire study
Days of treatment with additional hemostatic agent
Days of treatment with and total dose of bypassing agents (recombinant factor VIIa, activated prothrombin complex concentrate) or recombinant porcine factor VIII (susoctocag alfa) or other factor VIII concentrates; specifics (drug, amount and timing) of IST started during the 12 weeks of emicizumab prophylaxis
Time frame: 12 weeks
Remission Rate
Number of patients achieving partial remission (PR) and complete remission (CR) over 12 and 24 weeks after starting emicizumab prophylaxis
Time frame: 1 to 24 weeks
Hospitalizations
Days in hospital during week 12 of emicizumab treatment
Time frame: 12 weeks
Total dose of additional hemostatic agent
Total dose of bypassing agents (recombinant factor VIIa, activated prothrombin complex concentrate) or recombinant porcine factor VIII (susoctocag alfa) or other factor VIII concentrates; specifics (drug, amount and timing) of IST started during the 12 weeks of emicizumab prophylaxis
Time frame: 12 weeks
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Emory University
Atlanta, Georgia, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States
Indiana Hemophilia and Thrombosis Center, Inc.
Indianapolis, Indiana, United States
Tulane University
New Orleans, Louisiana, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University
St Louis, Missouri, United States
University of North Carolina
Chapel Hill, North Carolina, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
...and 6 more locations