Phase I/II/III Pharmacokinetic and outcome study of Inspiration`s Recombinant Factor IX product, IB1001, in subjects with Hemophilia B - ND

• Conditions: Prophylaxis and treatment of bleeding in patients with Hemophilia B; MedDRA version: 12.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)

• Registration Number: EUCTR2007-005100-41-IT
• Lead Sponsor: INSPIRATION BIOPHARMACEUTICALS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-07-20
• Last Update Posted: 9 October 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 75

Inclusion Criteria

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 ≤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 >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] ≤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 up to 12-15 months 10. 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; 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 11. Hemoglobin ≥7 g/dL at the time of the blood draw
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified