Type of information: update on patient enrollment
Company: Roche (Switzerland)
Product: emicizumab (ACE910 - anti-factor IXa x anti-factor X humanized bispecific antibody)
Action mechanism: bispecific antibody. Emicizumab is a bispecific antibody that mimics coagulation factor VIII. It is currently investigated as a therapy for people with hemophilia A. Emicizumab was designated as a Breakthrough Therapy by the FDA in September 2015.
Disease: hemophilia A
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country: Australia, Costa Rica, France, Germany, Italy, Japan, Republic of Korea, New Zealand, Poland, South Africa, Spain, Taiwan, UK, USA
Trial details: HAVEN 1 is a randomized, multi-center, open-label phase III clinical trial to evaluate the efficacy, safety and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in hemophilia A patients with inhibitors previously treated with episodic or prophylactic bypassing agents. Episodic bypassing agent patients will be randomized in a 2:1 fashion to receive emicizumab prophylaxis (Arm A) versus no prophylaxis (Arm B) and will be stratified across Arms A and B according to the number of bleeds they experienced over the last 24 weeks prior to study entry (less than [<] 9 or greater than or equal to [>=] 9 bleeds); Arm B patients will have the opportunity to switch to emicizumab prophylaxis after 24 weeks on-study. Prophylactic bypassing agent patients will switch to emicizumab prophylaxis (Arm C) from the start of the trial; enrollment will be extended for 24 weeks after the last patient has enrolled in Arms A or B or until approximately 50 patients have enrolled in Arm C, whichever occurs first. Episodic bypassing agent patients who previously participated in a Non-Interventional Study BH29768 but were unable to enroll in Arms A or B prior to their closure will have the opportunity to enroll in Arm D until 24 weeks after the last patient has enrolled in Arms A or B or until approximately 35 patients have enrolled in Arm D, whichever occurs first. Like patients in Arms A and C, Arm D patients will receive emicizumab prophylaxis from the start of the trial. All patients will continue to receive standard of care/background treatment with their usual episodic bypassing agent therapy to treat breakthrough bleeds, as needed. (NCT02622321)