BAX 326 (Recombinant factor nine): A clinical study evaluating pharmacokinetics, efficacy, safety, and immunogenicity in previously treated pediatric patients with severe or moderately severe hemophilia B.

• Conditions: Pediatric previously treated patients (PTPs) with severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B.; MedDRA version: 14.1Level: 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: EUCTR2011-002437-19-BG
• Lead Sponsor: Baxter Innovations GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-03-29
• Last Update Posted: 16 September 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

•Subject and/or legal representative has/have voluntarily 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 determined by the central laboratory
•Subject is < 12 years old at the time of screening
•Subject 6 to <12 years of age 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 the following criteria are met:
1) there are an estimated 100 - 150 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), prothrombin complex concentrate ?PCC? or fresh frozen plasma ?FFP? that are not fully documented (assumption based on the severity of disease and treatment history), and
2) s/he has participated in Immunine Study 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 < 6 years of age is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for > 50 EDs (based on the subject’s medical records).
If a subject does not have a verifiable, documented history of >50 EDs, s/he can be enrolled if the following criteria are met:
1) there are approximately 20 - 50 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), PCC or FFP) that are not fully documented, and
2) s/he has participated in Immunine Study 050901 and accumulated a minimum of 30 EDs to Immunine or a total of > 50 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 30 EDs (< 6 years of age) or at least 50 EDs (6 to < 12 years of age) to Immunine prior to enrollment
•Subject and/or legal representative accepts prophylactic treatment over a period of 6 months
•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
•Subject and/or the legal representative 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: 24
F.1.2 Adults (18-64 years) no
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

•Subject has a history of FIX inhibitors with a titer = 0.6 BU (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory with the corresponding detection limit) at any time prior to screening
•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
•Subject has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
•Subject has a known hypersensitivity to hamster proteins or rFurin
•Subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
•Subject has an abnormal renal function (serum creatinine >1.5 times the upper limit of normal)
•Subject has an International Normalized Ratio (INR) >1.4
•Subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels > 5 times the upper limit of normal
•Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B
•Subject’s platelet count is < 100,000/mL
•Subject has a clinically significant medical, psychiatric, or cognitive illness, that, in the opinion of the Investigator, would affect subject’s safety or compliance
•Subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or a-interferon) other than anti-retroviral chemotherapy
•Subject has participated in another investigational study within 30 days of enrollment. However, participation in Study 050901 with Immunine is allowed.

Study & Design

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