ITI rFVIIIFc study in HaemophiliaA Pts who failed previous ITI therapy
Research type
Research Study
Full title
A Non-Controlled, Open-Label, Multicenter, Study of Immune Tolerance Induction Performed with rFVIIIFc within a Timeframe of 60 Weeks in Severe Haemophilia A Patients with Inhibitors who have Failed Previous Immune Tolerance Induction Therapies
IRAS ID
226627
Contact name
Jessica Bainbridge
Contact email
Sponsor organisation
Swedish Orphan Biovitrum AB (publ)
Eudract number
2017-000065-73
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 6 months, 5 days
Research summary
Haemophilia A is a genetic disorder which is caused by deficiency of a clotting protein called factor VIII (FVIII). The main treatment for haemophilia A is the replacement of FVIII. Some patients develop antibodies towards FVIII, so called inhibitors, that interfere with and may completely inhibit the FVIII. A patient who has developed inhibitors does not respond properly to FVIII replacement therapy. The standard treatment to overcome inhibitors of FVIII is called immune tolerance induction (ITI). This treatment uses high doses of FVIII and sometimes additional medications over a period of time. While the majority of patients with haemophilia A respond favourably to the ITI treatment, a significant number of patients do not respond or have a recurrence of inhibitors.
The study medication, a factor VIII product called recombinant human coagulation factor VIII fusion protein (rFVIIIFc / ELOCTA®), has been approved for the treatment of haemophilia A by the Medicines & Healthcare Products Regulatory Agency (MHRA) and the European Medicines Agency (EMA) and is already available on prescription.
The main purpose of the study is to describe the outcome of ITI treatment performed with the study medication within a timeframe of 60 weeks in patients who failed previous ITI treatments attempts.
This study is “open label,” which means that both participant and the study doctor will know that the study medication given will be rFVIIIFc.
There will be approximately 20 male participants with haemophilia A, who will participate in this study in centres in North America and Europe.
This clinical research study is being sponsored by Swedish Orphan Biovitrum.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
17/NW/0511
Date of REC Opinion
6 Oct 2017
REC opinion
Favourable Opinion