Clinical Trial: Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Patients With Congenital Hemophilia A With Factor VIII Inhibitors

Brief Summary: This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital hemophilia A.

Detailed Summary:
Sponsor: Baxalta US Inc.

Current Primary Outcome: Proportion of Serious Bleeding Episodes Responsive to OBI-1 [ Time Frame: 24 hours after initiation of treatment ]

This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, the study results are not posted. The decision to terminate this study was not related to any safety and/or efficacy concern of OBI-1 in the indication described within the OBI-1-302 study (Congenital Hemophilia A).


Original Primary Outcome: Proportion of Serious Bleeding Episodes Responsive to OBI-1 [ Time Frame: 24 hours after initiation of treatment ]

Current Secondary Outcome:

  • Overall Proportion of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Proportion of Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Total Dose of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Total Number of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Correlation Between Response to OBI-1 Therapy at Specified Time Points and Eventual Control of Serious Bleeding Episodes. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers, the Total Dose of OBI-1, the Outcome at 24 Hours and the Eventual Control of the Bleeding Episode. [ Time Frame: Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers and the Recovery of OBI-1. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Recovery and Elimination Rate Parameters of OBI-1 in Subjects With Inhibitors Treated With OBI-1 Therapy. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Efficacy Assessment of OBI-1 in Participants With Anti-human Factor VIII Titers >30 Bethesda Units (BU) [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Anti-human Factor VIII Antibody Titer. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Anti-OBI-1 Antibody Titer. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]
  • Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ]


Original Secondary Outcome:

Information By: Baxalta US Inc.

Dates:
Date Received: September 13, 2011
Date Started: August 2011
Date Completion:
Last Updated: June 26, 2015
Last Verified: December 2014