Clinical Trial: Study of rFVIIIFc for ITI in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional
Official 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 P
Brief Summary: The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at tolerization.
Detailed Summary: This is an open-label, single-arm, interventional multi-center study designed to explore ITI performed with recombinant coagulation factor VIII Fc fusion protein (rFVIIIFc) within a timeframe of 60 weeks in patients with severe haemophilia A, who have failed previous attempts at tolerization including use of immunosuppressants.
Sponsor: Swedish Orphan Biovitrum
Current Primary Outcome: ITI success [ Time Frame: up to 60 weeks ]
Number of patients who achieve ITI success where ITI success is defined as achieving all 3 of the following criteria:
- Negative titer for inhibitor (<0.6 Bethesda units/mL by the Nijmegen-modified Bethesda assay) at 2 consecutive visits
- FVIII incremental recovery (IR) >66% of the expected IR at 2 consecutive visits
- FVIII half-life (t½) ≥7 hours
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Time to ITI success [ Time Frame: up to 60 weeks ]Time to the patient reaches ITI success according to the pre-defined criteria
- Occurrence of relapse during a 48-week period following successful ITI treatment [ Time Frame: Up to 108 weeks ]Relapse classified according to pre-defined criteria
- Number of bleedings during ITI treatment [ Time Frame: up to 60 weeks ]Only bleeds requiring treatment with rFVIIIFc or bypassing agents should be registered. A bleeding episode starts from the first sign of a bleed and ends no more than 72 hours after the last injection of bypassing agents or rFVIIIFc to treat the bleeding episode.
- Bleeding rate during a 48-week period following successful ITI treatment [ Time Frame: up to 108 weeks ]Only bleeds requiring treatment with rFVIIIFc or bypassing agents should be registered. A bleeding episode starts from the first sign of a bleed and ends no more than 72 hours after the last injection of bypassing agents or rFVIIIFc to treat the bleeding episode.
- Adverse events (AEs) [ Time Frame: Up to 114 weeks ]AEs and Serious Adverse Events as a measure of tolerability
- Consumption of rFVIIIFc [ Time Frame: Up to 108 weeks ]Consumption will be assessed based on amount of administered study treatment.
- Number of days missed school or work during ITI treatment [ Time Frame: up to 60 weeks ]Days missed school or work will be registered by the patients in an electronic diary
- Number of days missed school or work during a 48-week period following successful ITI treatment [ Time Frame: up to 108 weeks ]Days missed school or work will be registered by the patients in an electronic diary
- Number of hospitalizations during ITI treatment [ Time Frame: up to 60 weeks ]Days of hospitalization will be collected by the Investigator at the study visits
- Number of hospitalizations during a 48-week period following successful ITI treatment [ Time Frame: Up to 108 weeks ]Days of hospitalization will be collected by the Investigator at the study visits
- Adherence [ Time Frame: up to 108 weeks ]Defined as percentage of administered doses versus planned doses
Original Secondary Outcome: Same as current
Information By: Swedish Orphan Biovitrum
Dates:
Date Received: March 31, 2017
Date Started: June 1, 2017
Date Completion: April 1, 2020
Last Updated: March 31, 2017
Last Verified: March 2017