BAX 326 (recombinant Factor Nine): A Phase 1/3 Prospective, Controlled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, Immunogenicity in Previously Treated Patients with Severe or Moderately Severe Hemophilia B

• Conditions: Previously treated patients (PTPs) with severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B.; MedDRA version: 15.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2009-016720-31-CZ
• Lead Sponsor: Baxter Innovations GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-04-19
• Last Update Posted: 16 October 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

•Subject is 12 to 65 years old at the time of screening. Subject and/or legal representative has/have provided signed informed consent.
•Subject has severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory.
•Subject is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 150 EDs (based on the subject’s medical records).
•If a subject does not have a verifiable, documented history of 150 EDs, s/he can be enrolled if
1) there are 100 - 150 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), cryoprecipitate, or fresh frozen plasma) that are not fully documented, and
2) s/he has participated in the Immunine protocol 050901 and accumulated either at least 50 EDs to Immunine or a total of at least 150 EDs to a plasma-derived and/or recombinant FIX concentrate prior to enrollment.
•Subject has no evidence of a history of FIX inhibitors (based on the subject’s medical records). If a verifiable, documented history is unavailable, the subject can be enrolled if s/he has participated in Study 050901 for at least 50 EDs to Immunine prior to enrollment.
•If the subject is willing to receive prophylactic treatment over a period of 6 months.
•If the subject is willing to receive on-demand treatment, the subject has = 12 documented bleeding episodes requiring treatment within 12 months prior to enrollment and is willing to receive on-demand treatment for the duration of participation in this study.
•Subject is immunocompetent as evidenced by a CD4 count = 200 cells/mm3.
•Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/µL ~ <400,000 copies/mL
•If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
•Subject is willing and able to comply with the requirements of the protocol.

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•The subject has a history of FIX inhibitors with a titer = 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory) at any time prior to screening.
•The subject has a detectable FIX inhibitor at screening, with a titer =0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory.
•The subject's weight is < 35kg or > 120 kg.
•The subject has a history of allergic reaction, eg, anaphylaxis, following exposure to FIX concentrate(s).
•The subject has a known hypersensitivity to hamster proteins or rFurin.
•The subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
•The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
•The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4 hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
•The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels > 5 times the upper limit of normal.
•The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B.
•The subject’s platelet count is < 100,000/mL.
•The subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject’s safety or compliance.
•The subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or a-interferon) other than anti-retroviral chemotherapy.
•The subject has participated in another investigational study within 30 days of enrollment. Participation in study 050901 with Immunine is allowed.
•The subject is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (i.e., children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.

Study & Design

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