Hemophilia B Clinical Trial
Official title:
Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B
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.
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)
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care
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