A clinical trial to study the effects of Recombinant Factor IX product IB-1001 in patients with Hemophilia-B
- Conditions
- Health Condition 1: null- Hamophilia-B
- Registration Number
- CTRI/2011/04/001676
- Lead Sponsor
- Aptevo Bio Therapeutics LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 80
Criteria for Inclusion
1. 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.
2. Severe (factor IX activity less than or equal to 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.
3. Immunocompetent (CD4 count greater than 400/mm3) and not receiving immune modulating or chemotherapeutic agents.
4. Previously treated patients with a minimum of 150 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] less than or equal to 2 times the upper limit of the normal range.
7. Total bilirubin less than or equal to 1.5 times the upper limit of the normal range.
8. Renal function: serum creatinine less than or equal to 1.25 times the upper limit of the normal range.
9. Willingness to participate in the trial for up to 12-15 months.
10. European Union (EU), Israel, and India: Age of at least 12 years and body weight of greater than or equal to 40 kilograms to participate in any PK Study or the Surgical Sub-study; age of at least 12 years for the prophylaxis and on-demand components of the Treatment Phase and Continuation Study.
Subjects in France less than 18 years of age will not be included in any PK study.
United States (US): Age of at least 12 years and body weight of greater than or equal to 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.
11. Hemoglobin greater than or equal to 7 g/dL at the time of the blood draw.
Criteria for Exclusion
1.History of factor IX inhibitor ¡Ý0.6 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.Hypersensitivity to the active substance or to any of the excipients in the investigational products
7.Known allergic reaction to hamster proteins
8.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
9.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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method