One of the new prospective treatments for people with haemophilia which are currently undergoing clinical trials is the bi-specific antibody called ‘Emicizumab’. This antibody treatment is designed to be a subcutaneously administered treatment which mimics the effect of factor VIII. It can be used to treat people with factor VIII deficiency or people with factor VIII inhibitors. Clinical trials are currently in Phase 3 and include separate trials for both those with factor VIII deficiency and those with inhibitors. In Ireland, a small number of people with factor VIII deficiency will be participating in the clinical trial. We will not have any participants in the trial for those with factor VIII inhibitors. In the past week, the Society have been informed of the death of a person with haemophilia with inhibitors who was on the clinical trial HAVEN 1 for people with inhibitors. We have requested information from Roche, the company who are carrying out the clinical trial on this case and the information received is as follows;
At Roche, we respect and value the close relationships that exist within the haemophilia community. We also understand the important role patient organisations play within it. As such, we would like to officially update you on recent events in our HAVEN 1 study.
HAVEN 1 is the ongoing phase III study investigating the safety and efficacy of Emicizumab in people 12 years of age or older with haemophilia A and factor VIII inhibitors.
As part of our standard process to receive and expedite the reporting and investigating of adverse drug events (AEs), we received two new reports of Serious Adverse Events (SAE) in a patient who was enrolled in the HAVEN 1 study. Unfortunately, we were informed that the patient passed away. We are deeply saddened by this information.
The HAVEN 1 trial is ongoing, and thus the amount of information we have and can share is limited in order to protect the integrity of an ongoing study. Below is a brief summary of the medical history.
For all of us at Roche, patient safety continues to be of paramount importance. We will continue to work closely with the IDMC, Steering Committee, and our Investigators to evaluate all safety events that have been observed and to ensure the proper measures are in place to assure continued patient safety.
Meantime, we would recommend that all questions related to the above safety events be referred to the Roche affiliate in your respective country. Questions from patients participating in an Emicizumab clinical trial should be referred to the relevant study investigator.
The clinical trial study investigator in Ireland will be Dr. Niamh O Connell in St. James’s hospital. The I.H.S. will post further relevant updates on this and other clinical trials.