Clinical Trials

Date: 2017-02-21

Type of information: update on patient enrollment

phase: 3

Announcement: update

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)

Latest news:

  • • On February 21, 2017, the European Haemophilia Consortium (EHC) announced that the consortium has been informed of the death of a person with haemophilia with inhibitors, who was on the clinical trial HAVEN 1 with emicizumab. The HEC requested information from Roche about this case. The company answered that it has  received two new reports of Serious Adverse Events (SAE) in a patient who was enrolled in the HAVEN 1 study. This patient experienced a serious rectal haemorrhage (first reported SAE) and received bypassing agents, including repeated doses of activated prothrombin complex (aPCC), after which the patient developed signs of Thrombotic Microangiopathy (TMA) (second SAE). The preliminary assessment is that the clinical and laboratory characteristics of this case of TMA are consistent with what was observed in the two previously reported cases; however, our evaluation of the available information is ongoing.After aPCC was discontinued, laboratory values associated with TMA (platelet count and LDH) were improving. According to the report, treatment of the haemorrhage was complicated because the patient declined blood transfusions. The investigator’s assessment is that the cause of death was the rectal haemorrhage, and that this is not related to emicizumab. Roche is continuing to investigate the details of this patient case.
  • • On December 2, 2015, a Phase 3 trial sponsored by Roche was published on the NIH website for emicizumab and is currently recruiting participants.

Is general: Yes