Clinical Trial: Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B

Brief Summary: To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of IB1001 in subjects with hemophilia B.

Detailed Summary:

Primary Objectives:

  • to evaluate safety of IB1001 within the first 50 exposure days,
  • to determine IB1001 pharmacokinetics (PK), and
  • to assess efficacy of IB1001 prophylaxis with respect to breakthrough bleeding and with respect to control of hemorrhaging in subjects with severe hemophilia B within the first 50 exposure days

Secondary Objectives:

  • to evaluate long-term safety of IB1001; and
  • to evaluate long term efficacy of IB1001.

Exploratory Objectives:

  • to evaluate markers of thrombogenicity during the first 24 hours post-infusion [thrombogenicity markers will include at a minimum D-dimer test; however should there be a clinical reason (e.g., three consecutive elevations in D-dimer levels, a possible clinical thrombogenic episode), sufficient samples will be collected to also evaluate levels of fragment 1+2 (F1+2) and thrombin-antithrombin III complex (TAT)]
  • to evaluate IB1001 immunogenicity response (development of inhibitory and non-inhibitory factor IX binding antibodies and antibodies to host cell proteins)

Sponsor: Cangene Corporation

Current Primary Outcome: Number of study subjects with adverse events [ Time Frame: Within 6 months ]

Information on adverse events is collected after each infusion of study drug by a study subject. Assessment of adverse events is then performed by an investigator after 5 infusions of study drug, 1 month, 2 months, 3 months and 6 months of study drug treatment.


Original Primary Outcome: Same as current

Current Secondary Outcome: Number of bleeding episodes divided by number of months of observation [ Time Frame: Within 12 months ]

Original Secondary Outcome: Same as current

Information By: Cangene Corporation

Dates:
Date Received: November 14, 2013
Date Started: March 2014
Date Completion: November 2015
Last Updated: August 19, 2016
Last Verified: August 2016