Brief Title
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
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 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)
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of study subjects with adverse events
Secondary Outcome
Number of bleeding episodes divided by number of months of observation
Condition
Hemophilia B
Intervention
IB1001
Study Arms / Comparison Groups
IB1001
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
0
Start Date
March 2014
Completion Date
November 2015
Primary Completion Date
October 2015
Eligibility Criteria
Inclusion Criteria:
1. Age of at least 12 years
2. Body Mass Index of ≤ 29, with a minimum body weight of 40 kg
3. Written Institutional Review Board (IRB)/ Ethics Committee (EC)-approved informed
consent form (ICF)
4. Willingness to make the required study visits, and follow instructions while enrolled
in the study (up to 12 months)
5. Severe (factor IX activity ≤2 U/dL) hemophilia B patients with a minimum of 3 bleeding
episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12
months or in the event the subject is on prophylaxis, a minimum of 3 bleeding episodes
over the preceding 6 months or 6 bleeding episodes over the preceding 12 months prior
to being placed on prophylaxis
6. Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of
the PK and Treatment/Continuation Phase of the study
7. Previously treated patients with a minimum of 150 exposure days to a factor IX
preparation
8. Willingness to adhere to the 5-day washout of any factor IX replacement therapy prior
to PK evaluations
9. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or
chemotherapeutic agents
10. Platelet count at least 150,000/mm3
11. Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤2 times
the upper limit of the normal range
12. Total bilirubin ≤1.5 times the upper limit of the normal range
13. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
14. Hemoglobin ≥7 g/dL at the time of the blood draw
Exclusion Criteria:
1. History of factor IX inhibitor ≥0.6 BU (Bethesda units)
2. Existence of another coagulation disorder
3. Evidence of thrombotic disease, fibrinolysis or disseminated intravascular coagulation
(DIC)
4. Use of an investigational drug within 30 days prior to study entry
5. Previous use of IB1001
6. Use of medications that could impact hemostasis, such as aspirin
7. Hypersensitivity to the active substance or to any of the excipients in the
investigational products
8. Known allergic reaction to hamster proteins
9. History of poor compliance, a serious medical or social condition, or any other
circumstance that, in the opinion of the investigator, would interfere with
participation or compliance with the study protocol
10. History of adverse reaction to either plasma-derived factor IX or recombinant factor
IX that interfered with the subject's ability to treat bleeding episodes with a factor
IX product
Gender
All
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
, ,
Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT02048111
Organization ID
IB1001-04
Responsible Party
Sponsor
Study Sponsor
Cangene Corporation
Study Sponsor
, ,
Verification Date
August 2016