IMMUNINE Pre-Treatment Study

NCT01128881 | Completed Phase 4 DMC

• 2 other identifiers: 0509012009-016719-39
• Study Type: interventional
• Target: 57
• Locations: 10 countries
• Sites: 18

Brief 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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Hemostatic Efficacy

  * Number of IMMUNINE infusions required to achieve adequate hemostasis for each bleeding episode * Overall hemostatic efficacy rating of IMMUNINE for all bleeding episodes (scale of excellent, good, fair, none) * Annualized bleeding rate * Consumption of IMMUNINE * Number of infusions per month and per year (prophylaxis and on-demand) * Weight-adjusted consumption of IMMUNINE per event (prophylaxis, on-demand), per month and per year

  28 months

Study Arms (1)

IMMUNINE

EXPERIMENTAL

Biological: Factor IX Concentrate (purified, virus-inactivated)

Interventions

Factor IX Concentrate (purified, virus-inactivated) BIOLOGICAL

Intravenous injection/infusion; dose for prophylaxis: 20-40 IU/kg bodyweight, twice weekly (which may be adjusted to the subject´s bleeding pattern and lifestyle); dose for bleeding episodes and surgery: according to the Summary of Product Characteristics and the Product Information Leaflet of the respective country.

Also known as: IMMUNINE

IMMUNINE

Eligibility Criteria

• Age: Up to 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (18)

Hospital JR Vidal (Servicio de Hemotologie - Area de Investiagacion

Corrientes, 3400, Argentina

Location

Instituto de Hemofilia y Medicina Clinica Rubén Dávoli

Rosario, 2000, Argentina

Location

Hospital do apoio de Brasilia

Distrito Federal, 72620-000, Brazil

Location

UNIFESP - Universidade Estadual de Sáo Paulo

Sáo Paulo, 040024-002, Brazil

Location

Specialized Haematological Hospital "Joan Pavel"

Sofia, 1233, Bulgaria

Location

Hospital Dr. Sotero del Rio

Santiago, Chile

Location

Hospital san José, Centro de Hemofilia

Santiago, Chile

Location

Centro Medico Imbanaco

Cali, Colombia

Location

Klinika detske hematologie a onkologie UK

Prague, 150 06, Czechia

Location

Hematology and Transplantology Clinic, University Clinic Centre - Medical University Hospital

Gdansk, 80-952, Poland

Location

Medical College of the Jagiellonian University

Krakow, 31-501, Poland

Location

Medical University Lodz, Department of Hematology

Lodz, 93-510, Poland

Location

Institute of Haematology and Transfusion

Warsaw, 02-776, Poland

Location

Prof. Dr. C. T. Nicolau National Institute for Transfusional Hematology

Bucharest, 11156, Romania

Location

Louis Turcanu Emergency Clinical Children´s Hospital

Timișoara, Romania

Location

Hematology Research Center RAMS, Department of Hemophilia and Other Coagulopathies

Moscow, 125157, Russia

Location

Republican Center for Hemophilia Treatment, Outpatient Clinic No. 37

Saint Petersburg, 195213, Russia

Location

State Institution "Institute of Blood Pathology and Transfusion Medicine of Academy of Medical Sciences of Ukraine"

Lviv, 79044, Ukraine

Location

MeSH Terms

Conditions

Hemophilia B

Interventions

prothrombin complex concentrates

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 7, 2010
• First Posted: May 24, 2010
• Study Start: May 31, 2010
• Primary Completion: August 28, 2012
• Study Completion: August 28, 2012
• Last Updated: April 23, 2021

Record last verified: 2021-04

Locations

CL (2); CZ (1); RO (2); CO (1); RU (2); AR (2); BR (2); UA (1); BU (1); PL (4)