Open label study of ATX-F8-17 in male subjects with Haemophilia A
Research type
Research Study
Full title
Safety and proof of principle study of ATX-F8-17 in male subjects with Haemophilia A with a history of high responding inhibitor titre to factor VIII who have either failed or did not receive Immune Tolerance Induction therapy (ITI): An open label study, with an upward titration over 5 dose levels administered by intradermal injection.
IRAS ID
216808
Contact name
Gerard Dolan
Contact email
Sponsor organisation
Apitope NV international
Eudract number
2016-002488-32
Duration of Study in the UK
0 years, 10 months, 1 days
Research summary
This study is to help develop a drug called ATX-F8-17. ATX-F8-17 has not previously been tested in humans. This is a proof of principle study whereby the safety and tolerability of ATX-F8-17 will be assessed in haemophilia A subjects previous high inhibitor titre to factor VIII who have either failed or did not receive Immune Tolerance Induction therapy (ITI).
Briefly, haemophilia A is a clotting disorder which prevents normal bleeding. Patients with haemophilia A can develop an immune response to the standard treatment (Replacement factor VIII therapy). The patient’s body starts to mistake the factor VIII for a foreign object and develop antibodies 'inhibitors'. This makes Standard of Care treatment of haemophilia A ineffective. ATX-F8-17 aims to restore the patient’s immune response so that they can effectively treated with factor VIII.
This particular study aims to recruit 12 subjects. The study will involve regular visits, mostly every 2 weeks, to the clinic where patients will be administered the test product (ATX-F8-17) at increasing doses.
The key objective of the study is to look at the safety and tolerability of ATX-F8-17 and to look at exploratory aspects to determine if the drug may be efficacious. This will include the administration of factor VIII replacement therapy at weeks 19 and possibly 21, in order to see how the patient’s body responds.
The population eligible to take part in the study are males with haemophilia A, aged between 18 and 65 years, who are known to have previously produced inhibitors in response to factor VIII therapy at a high level but at inclusion, a low level. Several screening tests will be conducted to ensure the subject is suitable for the study.
Participation in the trial will last for about 30 weeks (from first screening visit to final end of study visit).
REC name
London - City & East Research Ethics Committee
REC reference
16/LO/2039
Date of REC Opinion
16 Jan 2017
REC opinion
Further Information Favourable Opinion