Davide Martino, MD, McMaster University Department of Medicine, discusses the BASIS clinical trial, a phase 3 study testing marstacimab in patients with hemophilia without inhibitors.
Hemophilia is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding following insults, such as an injury, surgery, or tooth extraction. In severe cases, heavy bleeding occurs after minor trauma or in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. The major types of this disorder are Hemophilia A and Hemophilia B. Although the two types have very similar signs and symptoms, they are caused by genetic changes in different genes. Hemophilia A, which is known as classical hemophilia, manifests as protracted and excessive bleeding either spontaneously or secondary to trauma. People with an unusual form of Hemophilia B, known as Hemophilia B Leyden, experience episodes of excessive bleeding in childhood, but have few bleeding problems after puberty.
Traditional therapies for hemophilia include factor replacement therapies. Unfortunately, persons can develop inhibitors (i.e., antibodies) to that type of therapy and render them ineffective.
BASIS Study
The BASIS study is a global Phase 3, open-label, multicenter study evaluating annualized bleed rate through 12 months on treatment with marstacimab, an investigational anti-tissue factor pathway inhibitor. The primary outcome measures were the safety and efficacy of marstacimab compared to traditional treatments of factor replacement therapy or bypass therapy. A total of 116 patients were enrolled in this study who had hemophilia A or B without inhibitors. After a six month observational phase, patients received 300 mg of marstacimab, followed by 150 mg subcutaneous weekly injections for twelve months.
As Dr. Martino describes, the trial observed significant reductions in patient’s analyzed bleeding rates after starting treatment. Marstacimab also showed superiority over previous treatments for reducing bleeding episodes. There were also no significant safety concerns during the study including no thromboembolic events.
For more information on hemophilia and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/