Brief Title
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Official Title
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Brief Summary
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects
associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and
control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric
subjects with hemophilia B.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of Infusions Required for Bleed Control
Secondary Outcome
Area Under the Curve (0-inf)
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
9
Start Date
November 2010
Completion Date
December 2016
Primary Completion Date
December 2016
Eligibility Criteria
Inclusion Criteria:
1. Subject's parent or legal guardian must give written Institutional Review Board
(IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to
make the required study visits and follow instructions while enrolled in the study.
For subjects ≥7 years of age, assent will be obtained if required by the institution.
For subjects < 7 years of age, legal assent is not reasonable to obtain.
2. Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy
with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6
months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis
with a bleeding pattern similar to that above demonstrated prior to starting
prophylaxis
3. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or
chemotherapeutic agents
4. Previously treated patients with a minimum of 50 exposure days to a factor IX
preparation
5. Platelet count at least 150,000/mm3
6. Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times
the upper limit of the normal range
7. Total bilirubin ≤1.5 times the upper limit of the normal range
8. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
9. Willingness to participate in the trial for approximately 6 months (50 exposures)
10. Age ≤12 years
11. Hemoglobin ≥7 g/dL at the time of the blood draw
Exclusion Criteria:
1. History of factor IX inhibitor ≥0.6 Bethesda units (BU)
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. On medications that could impact hemostasis, such as aspirin
6. 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
7. 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
N/A - 12 Years
Accepts Healthy Volunteers
No
Contacts
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Location Countries
India
Location Countries
India
Administrative Informations
NCT ID
NCT01271868
Organization ID
IB1001-02
Responsible Party
Sponsor
Study Sponsor
Medexus Pharma, Inc.
Study Sponsor
, ,
Verification Date
March 2021