Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea

Primary: To characterize the safety of RIXUBIS when used under normal clinical care in South Korea.

Secondary: To describe hemostatic effectiveness in subjects receiving RIXUBIS under normal clinical care in South Korea.

Study Overview

• Status: Completed
• Conditions: Hemophilia B
• Intervention / Treatment: Biological: RIXUBIS

• Study Type: Observational
• Enrollment (Actual): 57

Contacts and Locations

Study Locations

• Korea, Republic of
  • Daegu, Korea, Republic of, 42472
    • Daegu Catholic University Medical Center
  • Daejeon, Korea, Republic of, 35233
    • Daejeon Eulji University Hospital
  • Gyeonggi-do, Korea, Republic of, 13316
    • Chung Hospital
  • Seoul, Korea, Republic of, 05538
    • Kim Hugh Chul Internal Medicine
  • Seoul, Korea, Republic of, 06641
    • Korea Hemophilia Foundation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients with hemophilia B in South Korea who have been prescribed RIXUBIS.

Description

Inclusion Criteria:

1. Participants with congenital hemophilia B (FIX level ≤5%)
2. Participant or the participant's legally authorized representative has provided signed informed consent.
3. Participant is indicated for treatment according to the RIXUBIS Korean product leaflet.

Exclusion Criteria:

1. Participants with known hypersensitivity or presence of any contraindication to RIXUBIS or its excipients including hamster protein
2. Participants with Disseminated Intravascular Coagulation (DIC)
3. Participants with signs of fibrinolysis

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
All Study Participants; Participants with congenital hemophilia B (FIX level ≤5%)	Biological: RIXUBIS; Recombinant Factor IX (rFIX) for intravenous use; Other Names: BAX326; Coagulation Factor IX [Recombinant]; rFIX; BAX 326

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse events (AEs)	Seriousness and severity of AEs, including any inhibitory antibody development and/or anaphylactic reactions	Throughout the study period of approximately 2 years and 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Physician rated effectiveness of RIXUBIS for prophylactic treatment in participants <12 years old	Rated on a 4-point ordinal scale: Excellent, Good, Moderate, or none	Up to 6 months from baseline while on treatment
Participant rated effectiveness of RIXUBIS for prophylactic treatment in participants ≥12 years old	Rated on a 4-point ordinal scale: Excellent, Good, Moderate, or none	Up to 6 months from baseline while on treatment
Physician rated effectiveness of RIXUBIS for on-demand treatment in participants <12 years old	Rated on a 4-point ordinal scale: Excellent, Good, Moderate, or none	Up to 6 months from baseline while on treatment
Participant rated effectiveness of RIXUBIS for on-demand treatment in participants ≥12 years old	Rated on a 4-point ordinal scale: Excellent, Good, Moderate, or none	Up to 6 months from baseline while on treatment

Collaborators and Investigators

• Sponsor: Baxalta now part of Shire

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 6, 2017
• Primary Completion (ACTUAL): April 4, 2019
• Study Completion (ACTUAL): April 4, 2019

Study Registration Dates

• First Submitted: September 30, 2016
• First Submitted That Met QC Criteria: September 30, 2016
• First Posted (ESTIMATE): October 4, 2016

Study Record Updates

• Last Update Posted (ACTUAL): May 5, 2021
• Last Update Submitted That Met QC Criteria: April 30, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: 251501

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR