Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With 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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
|Official Title:||Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B|
- Number of study subjects with adverse events [ Time Frame: Within 6 months ] [ Designated as safety issue: Yes ]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.
- Number of bleeding episodes divided by number of months of observation [ Time Frame: Within 12 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||March 2014|
|Estimated Study Completion Date:||November 2015|
|Estimated Primary Completion Date:||October 2015 (Final data collection date for primary outcome measure)|
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.
- 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
- to evaluate long-term safety of IB1001; and
- to evaluate long term efficacy of IB1001.
- 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)
Please refer to this study by its ClinicalTrials.gov identifier: NCT02048111
|Contact: Fran Yadao, Manager Clinical Operationsfirstname.lastname@example.org|
|United States, Illinois|
|Not yet recruiting|
|Chicago, Illinois, United States|
|Not yet recruiting|
|Manchester, United Kingdom|