IMMUNE TOLERANCE INDUCTION, BY FACTOR VIII CONCENTRATE CONTAINING VON WILLEBRAND FACTOR, IN SEVERE OR MODERATE HAEMOPHILIA A PATIENTS WITH INHIBITORS (ITI)

NCT02479087 Phase 4 UNKNOWN

The purpose of this study is to assess the role of a FVIII/VWF complex concentrate (Emoclot) in successfully inducing immune tolerance (I.T.I.) in patients with Haemophilia A with inhibitors, including patients at high risk of failure.

Details

Lead sponsor	Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Phase	Phase 4
Status	UNKNOWN
Enrolment	20
Start date	2015-01
Completion	2020-01

Conditions

• Hemophilia A

Interventions

• Plasma-derived FVIII/VWF concentrate

Primary outcomes

• Efficacy: evaluation of the success of IT induction — Up to33 months
  Success:Inhibitor disappearance/reduction to \<0.6 BU/ml with FVIII activity recovery of at least ≥ 66% within 33 months of treatment ; Partial Success: inhibitor reduction to \<5 BU/ml with clinical response to FVIII treatment, not followed by an inhibitor increase to values \>5 BU/ml for a treatment period of 6 months on demand, or for 12 months of prophylactic treatment; No Response (Failure):Failure in relation to the above criteria defining complete response and partial response within 33 months, OR a reduction of the concentration of the inhibitor, less than 20%, compared to the peak of inhibitor in the IT, in every period of 6 months after the first 3 months of treatment. This implies that 9 months represents the minimum period of treatment and 33 months the maximum possible duration of ITI without success, OR patient withdrawal from the study for any reason.

Countries

Egypt, India