Clinical Trial: A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official 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

Brief Summary: The primary purpose of this study is to describe the time to tolerization with rFVIIIFc in participants within a maximum of 12 months of ITI treatment.

Detailed Summary:
Sponsor: Bioverativ Therapeutics Inc.

Current Primary Outcome: Time to Tolerization With rFVIIIFc [ Time Frame: Up to 12 Months ]

Tolerization is defined as inhibitor titer less than (<) 0.6 Bethesda units per milliliter (BU/mL).


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of Participants With Immune Tolerance Induction (ITI) Success [ Time Frame: Up to 48 Weeks ]
    ITI Success will be defined as negative titer for inhibitor less than (<) 0.6 BU/mL by the Nijmegen-modified Bethesda assay.
  • Number of Participants Who Experience Relapse [ Time Frame: Up to 48 Weeks ]
    Number of Participants with ITI success who reaches the criteria for relapse (defined as inhibitor titer > 0.6 BU/mL or abnormal recovery after tolerance is achieved) will be evaluated.
  • Number of Bleeding Episodes During ITI and During the 48-week Period After Successful ITI performed with rFVIIIFc [ Time Frame: Up to Week 104 ]
    A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode.
  • Number of Participants With Treatment-emergent Adverse Events (AEs) and Treatment-emergent Serious Adverse Events (SAEs) as a Measure of Safety and Tolerability [ Time Frame: Approximately 2 Years ]
    An AE is any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition.
  • Number of Days Away From Work or School [ Time Frame: Up to Week 104 ]
    Number of days missed from school or work will be summarized descriptively.
  • Number of Hospitalization Days [ Time Frame: Up to Week 104 ]
    Number of hospitalization days will be summarized descriptively.
  • Adherence to Treatment Regimen [ Time Frame: Up to Week 104 ]
    Defined as percentage of administered doses versus planned doses.
  • Consumption of rFVIIIFc [ Time Frame: Up to Week 104 ]
    Consumption will be assessed based on amount of administered study treatment.


Original Secondary Outcome: Same as current

Information By: Bioverativ Therapeutics Inc.

Dates:
Date Received: March 10, 2017
Date Started: May 2017
Date Completion: December 2020
Last Updated: March 22, 2017
Last Verified: March 2017