BAX 326 (rFIX) Continuation Study

NCT01286779 | Completed Phase 3 Results DMC

• 2 other identifiers: 2510012010-022726-33
• Study Type: interventional
• Target: 117
• Locations: 17 countries
• Sites: 42

Brief Summary

The purpose of this BAX 326 Continuation Study is to further investigate incremental recovery over time, the hemostatic efficacy, the safety, immunogenicity, and health-related quality of life (HR QoL) of BAX 326 in previously treated patients (PTPs) with severe and moderately severe hemophilia B who participated in BAX 326 pivotal study 250901 or BAX 326 pediatric study 251101.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Adverse Events Possibly or Probably Related to the Investigational Product

  Possibly or probably related adverse events that occurred during or after first BAX326 infusion.

  Assessed (based on patient diary) every 3 months until study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).

Secondary Outcomes (21)

• Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Required Until Bleed Resolution

  Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).

• Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed

  Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).

• Annualized Bleed Rate During Prophylaxis Treatment

  For prophylactic treatment the period from first to last prophylactic infusion is considered.

• Consumption of BAX 326: Number of Infusions Per Month and Per Year

  Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).

• Consumption of BAX 326: Weight Adjusted Consumption Per Month and Per Year

  Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).

Study Arms (1)

BAX 326

EXPERIMENTAL

Biological: BAX 326 (Recombinant factor IX)

Interventions

BAX 326 (Recombinant factor IX) BIOLOGICAL

The treatment with BAX 326 will be at the discretion of the investigator and will consist of either twice weekly prophylactic treatment with 50 IU/kg, modified prophylaxis, or on-demand treatment.

BAX 326

Eligibility Criteria

• Age: Up to 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject and/or legal representative has/have voluntarily provided signed informed consent
• Subject has completed Baxter clinical study 250901 (pivotal study) or Baxter clinical study 251101 (pediatric study)
• Subject was 12 to 65 years old at the time of screening for Study 250901 or \< 12 years old at the time of screening for Study 251101
• Subject has severe (FIX level \< 1%) or moderately severe (FIX level 1-2%) 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 Pivotal Study 250901 or Pediatric Study 251101

You may not qualify if:

• Subject received factor IX product(s) other than BAX 326 upon completion of Baxter Pivotal Study 250901 or Pediatric Study 251101
• Subject has been diagnosed with an acquired hemostatic defect other than hemophilia B
• For subjects transferring from Pivotal Study 250901: Subject's weight is \< 35 kg or \> 120 kg
• 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

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (42)

Instituto de Hematología y Medicina Clíncia Rubén Dávoli

Rosario, 2000, Argentina

Location

UNIFESP - Universidade Estadual de Sao Paulo

São Paulo, 040024-002, Brazil

Location

Specialized Haematological Hospital "Joan Pavel"

Sofia, 1233, Bulgaria

Location

Hospital Dr. Sotero del Rio

Santiago, Chile

Location

Hospital de San Jose

Bogotá, Colombia

Location

Centro Medico Imbanaco

Cali, Colombia

Location

Hospital Pablo Tobon Uribe

Medellín, 005543, Colombia

Location

Klinika detské hematologie a onkologie

Prague, 150 06, Czechia

Location

Maulana Azad Medical College and Associated Hospital

New Delhi, 110002, India

Location

St. James's Hospital, National Center for Hereditary Coagulation Disorders

Dublin, 8, Ireland

Location

University Hospital Policlinico Vittorio Emanuele, Hospital Ferrarotto Alessi

Catania, 95124, Italy

Location

University Hospital Careggi, Agency of Hemophilia - Regional Reference Center for Inherited Bleeding

Florence, 50134, Italy

Location

University of Foggia Riuniti Hospital, Department of Clinical and Experimental Medicine

Foggia, 71100, Italy

Location

Hospital San Giovanni Bosco, Center for Hemophilia and Thrombosis, Department of Hematology

Naples, 80144, Italy

Location

Padova University Hospital, Medical Clinic II, Center for Hemophilia

Padua, 35128, Italy

Location

Nara Medical University, Department of Pediatrics

Nara, 634-8251, Japan

Location

Tokyo Medical University

Tokyo, 160-0023, Japan

Location

Ogikubo Hospital

Tokyo, 167-0035, Japan

Location

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

Gdansk, 80-952, Poland

Location

University Pediatric Hospital in Cracow

Krakow, 30-663, Poland

Location

Medical College of the Jagiellonian University, Department of Hematology

Krakow, 31-501, Poland

Location

Copernicus Hospital, Medical University in Lodz, Department of Hematology

Lodz, 93-510, Poland

Location

Professor Tadeusz Sokolowski Independent Public Teaching Hospital No. 1 of the Pomeranian Medical University in Szczecin

Szczecin, 71-252, Poland

Location

Klinika Hematologii

Warsaw, 00-579, Poland

Location

Institute of Haematology and Transfusion Medicine

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

Federal State Institution Kirov Hematology and Blood Transfusion Research Institute under the Federal Agency for High-Tech Medical Care

Kirov, 610027, Russia

Location

Pediatric Regional Clinical Hospital, Hematology Department

Krasnodar, 350007, Russia

Location

Hematology Research Center RAMS, Department of Reconstructive Orthopedics for Haemophilia Patients

Moscow, 125167, Russia

Location

Republican Center for Hemophilia Treatment Outpatient Clinic No. 37

Saint Petersburg, 195213, Russia

Location

Regional clinical hospital

Yekaterinburg, 620149, Russia

Location

Malmö University Hospital, Department of Coagulation Disorders

Malmo, 205 02, Sweden

Location

Taipei Medical University Hospital

Taipei City, Taipei, 110, Taiwan

Location

Chung-Ho Memorial Hospital

Kaohsiung City, 807, Taiwan

Location

China Medical University Hospital

Taichung, 40447, Taiwan

Location

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

Location

National Taiwan University Hospital

Taipei, 10002, Taiwan

Location

Tri-Service General Hospital

Taipei, 114, Taiwan

Location

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

Lviv, 79044, Ukraine

Location

Katharine Dormandy Haemophilia Centre and Haemostasis Unit, Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

Related Publications (1)

• Windyga J, Stasyshyn O, Lissitchkov T, Mamonov V, Serban M, Rusen L, Ploder B, Tangada S. Safety, Immunogenicity, and Hemostatic Efficacy of Nonacog Gamma in Patients With Severe or Moderately Severe Hemophilia B: A Continuation Study. Clin Appl Thromb Hemost. 2020 Jan-Dec;26:1076029620950836. doi: 10.1177/1076029620950836.

  PMID: 32866032 DERIVED

MeSH Terms

Conditions

Hemophilia B

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

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2011
• First Posted: January 31, 2011
• Study Start: April 12, 2011
• Primary Completion: June 29, 2017
• Study Completion: June 29, 2017
• Last Updated: May 20, 2021
• Results First Posted: September 10, 2018

Record last verified: 2021-04

Locations

BU (1); CZ (1); IT (5); TW (6); IE (1); RO (2); RU (5); SE (1); GB (2); AR (1); BR (1); CO (3); JP (3); UA (1); IN (1); CL (1); PL (7)