Status:
COMPLETED
Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
Lead Sponsor:
Medexus Pharma, Inc.
Conditions:
Hemophilia B
Eligibility:
All Genders
5+ years
Phase:
PHASE2
PHASE3
Brief Summary
Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophy...
Eligibility Criteria
Inclusion
- Patient must be willing to give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent, make the required study visits, and follow instructions while enrolled in the study
- Severe (factor IX activity ≤2 U/dL) hemophilia B subjects on demand therapy with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern as above demonstrated prior to starting prophylaxis
- Immunocompetent (CD4 count \>400/mm3) and not receiving immune modulating or chemotherapeutic agents
- Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
- Platelet count at least 150,000/mm3
- Liver function: alanine transaminase \[ALT\] and aspartate transaminase \[AST\] ≤2 times the upper limit of the normal range
- Total bilirubin ≤1.5 times the upper limit of the normal range
- Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
- Willingness to participate in the trial for up to 12-15 months
- European Union (EU), Israel, and Canada: Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study \[the Surgical Sub-study does not apply to the UK\]; age of at least 12 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study
- United States (US): Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study; age of at least 5 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study
- Hemoglobin ≥7 g/dL at the time of the blood draw
Exclusion
- History of factor IX inhibitor ≥0.6 Bethesda units (BU)
- Existence of another coagulation disorder
- Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC)
- Use of an investigational drug within 30 days prior to study entry
- On medications that could impact hemostasis, such as aspirin
- 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
- 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
Key Trial Info
Start Date :
January 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2016
Estimated Enrollment :
77 Patients enrolled
Trial Details
Trial ID
NCT00768287
Start Date
January 1 2009
End Date
December 1 2016
Last Update
April 6 2021
Active Locations (23)
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1
City of Hope
Duarte, California, United States, 91010
2
The Hemophilia Treatment Center of Orthopaedic Hospital
Los Angeles, California, United States, 90007
3
Emory University School of Medicine Pediatric Hematology
Atlanta, Georgia, United States, 30322
4
Rush University Medical Center-Pediatric Hematology Oncology
Chicago, Illinois, United States, 60612