997HA402 - ITI in Severe Haemophilia A Subjects with Inhibitors
Research type
Research Study
Full title
A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects with Inhibitors Undergoing the First ITI Treatment
IRAS ID
234047
Contact name
Jim Luther
Contact email
Sponsor organisation
Bioverativ
Eudract number
2017-000373-36
Duration of Study in the UK
3 years, 1 months, 9 days
Research summary
Summary of Research
The 997HA402 clinical trial will treat up to 30 male patients in the USA, EU, Japan and Canada with severe Haemophilia A, who have developed inhibitors to haemophilia medication.
The study drug is a Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc), the brand name is ELOCATE® in Europe and ELOCTA® in the USA. The study drug will be given daily via a slow push injection into a vein. The first study drug administration will be at the study centre and then at home thereafter.
The aim of the study is stop the patients producing inhibitors to the haemophilia medication, the treatment is called Immune Tolerance Induction (ITI).The main purposes of this study is see how long this takes for patients to tolerate haemophilia medication. It is expected patients will be involved in the study for about 2 years (Screening 2 -4 weeks, Treatment 48 weeks, Tapering Treatment 16 weeks, Follow-up 32 Weeks).
This trial is “open label” meaning the participant and doctor will know the treatment that is what being received. All patients will get study drug there is no dummy drug called a “placebo”. It is a Phase 4 clinical trial, this means study drug has already been tested in patients but further information will be collected.
Summary of Results
The efficacy and safety data support a positive risk-benefit profile for rFVIIIFc in ITI treatment of patients with severe hemophilia A and high-titer inhibitors to FVIII.
REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
17/EE/0459
Date of REC Opinion
22 Nov 2017
REC opinion
Favourable Opinion