RIXUBIS Drug Use-Result Survey (Japan)

The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS.

1. Unexpected adverse drug reactions
2. Occurrence of adverse drug reactions in the actual clinical use
3. Factors that may affect safety and effectiveness
4. Occurrence of Factor IX (FIX) inhibitor development in patients with coagulation FIX deficiency
5. Safety and effectiveness for hemophilia B patients who received routine prophylactic therapy, on-demand therapy and perioperative therapy

Study Overview

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

• Study Type: Observational
• Enrollment (Actual): 6

Contacts and Locations

Study Locations

• Japan
  • Gifu Prefecture
    • Seki-shi, Gifu Prefecture, Japan, 501-3802
      • Seki-shi, Japan
  • Gunma Prefecture
    • Maebashi-shi, Gunma Prefecture, Japan, 371-8511
      • Maebashi-shi, Japan
  • Miyazaki Prefecture
    • Nobeoka-shi, Miyazaki Prefecture, Japan, 882-0835
      • Nobeoka-shi, Japan
  • Niigata Prefecture
    • Niigata-shi, Niigata Prefecture, Japan, 950-0862
      • Niigata-shi, Japan
  • Osaka Prefecture
    • Osaka-shi, Osaka Prefecture, Japan, 543-0001
      • Osaka-shi, Japan

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who receive RIXUBIS in the actual clinical setting

Description

Inclusion Criteria:

• Patients with hemophilia B scheduled to receive treatment with RIXUBIS

Exclusion Criteria:

• Patients not administered RIXUBIS

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
All Study Participants	Biological: RIXUBIS; Other Names: Recombinant factor IX; BAX326; Coagulation Factor IX [Recombinant]; BAX 326

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination)	Number of participants who discontinued the use of Nonacog Gamma (Genetical Recombination) was reported in this outcome measure.	Throughout the study period, approximately 4 ½ years
Number of Participants Who Developed a Factor IX (FIX) Inhibitor	Number of participants who developed a Factor IX (FIX) Inhibitor was reported in this outcome measure.	Throughout the study period, approximately 4 ½ years
Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study	Annual bleed rate (ABR) was defined as the number of times of bleeding during the study. ABR was reported in this outcome measure.	Throughout the study period, approximately 4 ½ years
Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen	Number of doses to treat a bleed of participants on an on-demand regimen was reported in this outcome measure.	At bleed resolution throughout the study period of approximately 4 ½ years
Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor)	Number of participants in hemostatic effectiveness of Rixubis with a 4-point ordinal scale (Excellent, Moderate, Good, Poor) for an on-demand regimen was reported in this outcome measure. The definition of each scale was following: Excellent; After a single infusion, complete disappearance of pain and objective decrease of bleeding symptom (swelling, tenderness, and increase in range of motion in musculoskeletal bleeding case) were observed. Good; After a single infusion, there were definitive relief of pain and improvement of bleeding symptom. Fair; After a single infusion, there were a probable or slight relief of pain and a mild improvement of bleeding signs. Poor; Improvement was not observed or symptom was aggravated.	At bleed resolution throughout the study period of approximately 4 ½ years
Hemostatic Effectiveness of Rixubis in Surgery-Perioperative and Postoperative Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) for Participants Who Received Perioperative Therapy During the Study	Number of participants in hemostatic effectiveness of Rixubis with a 4-point ordinal scale (Excellent, Moderate, Good, Poor) for perioperative therapy was reported in this outcome measure. The definition of each scale was following: Excellent; Amount of bleeding is smaller than expected. Good; Amount of bleeding is within the expected range. Fair; Amount of bleeding is greater than expected, with use of additional concomitant medication. Poor; Hemostasis difficulty.	Assessed at the time of discharge from recovery room; and at 24 to 72 hours postoperatively

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Who Experienced Adverse Events of Shock or Anaphylaxis	Number of participants who experienced adverse events of shock or anaphylaxis was reported in this outcome measure.	Throughout the study period, approximately 4 ½ years
Number of Participants Who Experienced Adverse Events of Thromboembolism	Number of participants who experienced adverse events of thromboembolism was reported in this outcome measure.	Throughout the study period, approximately 4 ½ years

Collaborators and Investigators

• Sponsor: Baxalta now part of Shire
• Collaborators: Takeda
• Investigators: Study Director: Study Director, Shire

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): November 16, 2016
• Primary Completion (Actual): May 11, 2022
• Study Completion (Actual): May 11, 2022

Study Registration Dates

• First Submitted: October 5, 2016
• First Submitted That Met QC Criteria: October 17, 2016
• First Posted (Estimated): October 19, 2016

Study Record Updates

• Last Update Posted (Actual): July 22, 2024
• Last Update Submitted That Met QC Criteria: February 8, 2024
• Last Verified: February 1, 2024

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 B
• Other Study ID Numbers: 251601

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites).
• 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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No