BAX 326 (recombinant coagulation factor nine): Continuation of a study of the safety and effectiveness of the product 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: 13.1Level: 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: EUCTR2010-022726-33-DE
• Lead Sponsor: Baxter Innovations GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-03-24
• Last Update Posted: 11 September 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

•The subject and/or legal representative has/have voluntartily provided signed informed consent.
•Subject has completed Baxter clinical study 250901.
•Subject was 12 to 65 years old at the time of screening for 250901 study.
•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 has not developed an inhibitory FIX antibody during Baxter clinical study 250901.
•Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/µL ~ <400,000 copies/mL
•Subject is immunocompetent as evidenced by a CD4 count = 200 cells/mm3.
•If female of childbearing potential, subject presents with a negative pregnancy test and agrees to continue employing 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: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0
;
•The subject and/or legal representative has/have voluntartily provided signed informed consent.
•Subject has completed Baxter clinical study 250901.
•Subject was 12 to 65 years old at the time of screening for 250901 study.
•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 has not developed an inhibitory FIX antibody during Baxter clinical study 250901.
•Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/µL ~ <400,000 copies/mL
•Subject is immunocompetent as evidenced by a CD4 count = 200 cells/mm3.
•If female of childbearing potential, subject presents with a negative pregnancy test and agrees to continue employing 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: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

•The subject received factor IX product(s) other than BAX 326 upon completion of Baxter pivotal study 250901.
•The subject has been diagnosed with an acquired hemostatic defect other than hemophilia B.
•The subject’s weight is < 35 kg or > 120 kg.
•The subject’s platelet count is < 100,000/mL.
•The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
•The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels = 5 times the upper limit of normal.
•The subject 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.
•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 planned to take part in any other clinical study, with the exception of BAX 326 Surgery study as described in this protocol, during the course of the Continuation Study.
•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.
;
•The subject received factor IX product(s) other than BAX 326 upon completion of Baxter pivotal study 250901.
•The subject has been diagnosed with an acquired hemostatic defect other than hemophilia B.
•The subject’s weight is < 35 kg or > 120 kg.
•The subject’s platelet count is < 100,000/mL.
•The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
•The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels = 5 times the upper limit of normal.
•The subject 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.
•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 planned to take part in any other clinical study, with the exception of BAX 326 Surgery study as described in this protocol, during the course of the Continuation Study.
•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