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