NCT02048111

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

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

• Status: Withdrawn
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Cangene Corporation · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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 description

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)

Conditions

• Hemophilia B

Interventions

Type	Name	Description
BIOLOGICAL	IB1001	Prophylaxis (during Treatment and Continuation phases): 40 - 75 IU/kg twice weekly. The starting dose for prophylaxis may be based on previous recombinant factor IX product use. The recommended starting prophylaxis dose is 40 - 60 IU/kg twice weekly, however, the investigator may prescribe up to 75 IU/kg twice weekly at their discretion (after clinically assessing the subject) and discretion of the subject. The dose or the frequency of IB1001 prophylaxis may be adjusted at the discretion of the investigator.

Timeline

• Start date: 2014-03-01
• Primary completion: 2015-10-01
• Completion: 2015-11-01
• First posted: 2014-01-29
• Last updated: 2016-08-23

Locations

2 sites across 2 countries: United States, United Kingdom