Guy Young, MD, Children’s Hospital Los Angeles, discusses the SWITCH clinical trial design for patients with hemophilia A.
Hemophilia A is an inherited bleeding disorder in which the blood does not clot normally. People with hemophilia A will bleed more than normal after an injury, surgery, or dental procedure. Bleeding into the joints, muscles, brain, or organs can cause pain and other serious complications. Hemophilia A is caused by having low levels of factor VIII, needed to form blood clots. The disorder is caused by changes in the F8 gene.
Fitusiran is an antithrombin (AT)-lowering therapeutic that increases thrombin generation to restore hemostasis in people with hemophilia A or B, with or without inhibitors. The SWITCH clinical trial is an exploratory, open-label, single-arm phase 1 study (NCT06145373) investigating the safety and tolerability of switching from emicizumab to fitusiran prophylaxis in patients with severe hemophilia A, with or without inhibitors.
The study expects to enroll 15 to 20 male patients ages 18 years or older with severe hemophilia A, with or without inhibitors. The study will consist of a screening period of about 60 days, a 2 month pre-fitusiran treatment period to allow washout of emicizumab, and an 18 month fitusiran prophylaxis period.
During the fitusiran prophylaxis period, breakthrough bleed management guidelines with reduced dose/frequency of clotting factor concentrates/bypassing agents and regular appropriate laboratory testing will be implemented. Following the final fitusiran dose, antithrombin (AT) activity levels will be monitored at monthly intervals during the 6 month follow-up period.
The primary endpoint of the trial is incidence, severity, and seriousness of adverse events from day one to month four of treatment with fitusiran. Secondary endpoints include laboratory assessments, emicizumab plasma concentrations up to complete washout, health-related quality of life measures from baseline to the end of the study, change in participant joint health, and annualized bleeding rate during the 14 month extension period. Additionally, incidence, severity, and seriousness of adverse events from day 1 to month 18 of treatment will be assessed.
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To learn more about hemophilia and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/
*Video Correction at 4:50: Currently, fitusiran is approved only for patients 12 years and older. So it is being studied in children, but is not yet approved in children.