Study of a new medicinal product, IB1001, to treat patients with severe hemophilia B

• Conditions: Severe Hemophilia B; MedDRA version: 16.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2013-003742-16-GB
• Lead Sponsor: Cangene Europe Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-09-18
• Last Update Posted: 4 August 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 18

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 (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 subjects with 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
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

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. 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

Study & Design

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