IMMUNINE Pre-Treatment Study
Information source: Baxalta US Inc.
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hemophilia B
Intervention: Factor IX Concentrate (purified, virus-inactivated) (Biological)
Phase: Phase 4
Status: Completed
Sponsored by: Baxalta US Inc. Official(s) and/or principal investigator(s): Brigitt Abbuehl, MD, Study Director, Affiliation: Baxter Healthcare Corporation
Summary
The primary objective of this study is to prospectively document the exposure to IMMUNINE
and to monitor FIX inhibitors over a period of approximately 20 to 50 exposure days while
receiving prophylactic treatment in up to 50 previously treated patients (PTPs) aged 12-64
years and approximately 20 pediatric PTPs up to 11 years of age with severe (FIX level < 1%)
or moderately severe (FIX level <= 2%) hemophilia B who are planned to enter BAX326 study
250901, provided all eligibility criteria are met.
In addition, this study will evaluate the efficacy, safety, immunogenicity, thrombogenicity,
and health-related quality of life (HR QoL) of these subjects.
Clinical Details
Official title: IMMUNINE - Purified Factor IX Concentrate Virus-Inactivated: A Phase 4, Prospective, Open-label Multicenter Study to Prospectively Document the Exposure of IMMUNINE and to Monitor FIX Inhibitors in Previously Treated Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level <= 2%) Hemophilia B Who Are Planned to Enter BAX 326 Study 250901 to Investigate a New Recombinant FIX Concentrate
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Hemostatic Efficacy
Eligibility
Minimum age: N/A.
Maximum age: 64 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Subject is up to 64 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 or recombinant FIX concentrate(s),
cryoprecipitate or fresh frozen plasma (FFP) for approximately 100-150 exposure days
(EDs) if >= 6 years old, or 20-50 EDs if < 6 years old, and is planned to enter
BAX326 study 250901. The number of EDs are derived from the subject's treatment
regimen and his/her bleeding pattern
- Subject is willing to receive prophylactic treatment for the duration of the study
- Subject is immunocompetent as evidenced by a CD4 count >= 200 cells/mm(3)
- Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200
particles/μL ~ < 400,000 copies/mL
- Female subject of childbearing potential, presents with a negative serum 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
Exclusion Criteria:
- The subject has a detectable factor IX inhibitor at screening, with a titer >= 0. 6
Bethesda Units (BU) as determined by the Nijmegen modification of the Bethesda assay
in the central laboratory
- The subject has a history of factor IX 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) at any time prior to screening
- The subject has a history of allergic reaction, eg, anaphylaxis, following exposure
to factor IX concentrate(s)
- The subject has a known hypersensitivity to hamster proteins
- The subject has evidence of a thrombotic disease, fibrinolysis or disseminated
intravascular coagulation (DIC)
- The subject is scheduled for elective surgery, unless the surgery is medically
required within the anticipated study period
- 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) exceeding the upper limit of
normal (ULN), 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. During
the study, subjects with chronic hepatitis B or C may have fluctuations of up to 5
times the upper limit of normal but will not require discontinuation.
- 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 other than anti-retroviral chemotherapy (eg,
α-interferon, corticosteroid agents at a dose equivalent to hydrocortisone greater
than 10 mg/day)
- The subject is unwilling to consider further participation in BAX 326 (rFIX) pivotal
study 250901 or BAX 326 pediatric study
- The subject has participated in another investigational study within 30 days of
enrollment or is scheduled to participate in another clinical study involving an
investigational product (IP) or investigational device during the course of this
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 (ie, children, partner/spouse, siblings, parents) as well as
employees of the investigator or site personnel conducting the study.
Locations and Contacts
Hospital JR Vidal (Servicio de Hemotologie - Area de Investiagacion, Corrientes Capital 3400, Argentina
Instituto de Hemofilia y Medicina Clinica Rubén Dávoli, Rosario 2000, Argentina
Hospital do apoio de Brasilia, Distrito Federal 72620-000, Brazil
UNIFESP - Universidade Estadual de Sáo Paulo, Sáo Paulo 040024-002, Brazil
Specialized Haematological Hospital "Joan Pavel", Sofia 1233, Bulgaria
Hospital Dr. Sotero del Rio, Santiago, Chile
Hospital san José, Centro de Hemofilia, Santiago, Chile
Centro Medico Imbanaco, Cali, Colombia
Klinika detske hematologie a onkologie UK, Prague 150 06, Czech Republic
Hematology and Transplantology Clinic, University Clinic Centre - Medical University Hospital, Gdansk 80-952, Poland
Medical College of the Jagiellonian University, Krakow 31-501, Poland
Medical University Lodz, Department of Hematology, Lodz 93-510, Poland
Institute of Haematology and Transfusion, Warsaw 02-776, Poland
Prof. Dr. C. T. Nicolau National Institute for Transfusional Hematology, Bucharest 11156, Romania
Louis Turcanu Emergency Clinical Children´s Hospital, Timisoara, Romania
Hematology Research Center RAMS, Department of Hemophilia and Other Coagulopathies, Moscow 125157, Russian Federation
Republican Center for Hemophilia Treatment, Outpatient Clinic No. 37, St. Petersburg 195213, Russian Federation
State Institution "Institute of Blood Pathology and Transfusion Medicine of Academy of Medical Sciences of Ukraine", Lviv 79044, Ukraine
Additional Information
Starting date: May 2010
Last updated: June 26, 2015
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