RIXUBIS Drug Use-Result Survey (Japan)

NCT02937831 | Completed Results

• 1 other identifier: 251601
• Study Type: observational
• Target: 6
• Locations: 1 country
• Sites: 5

Brief Summary

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

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

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (6)

• 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 Outcomes (2)

• Number of Participants Who Experienced Adverse Events of Shock or Anaphylaxis

  Throughout the study period, approximately 4 ½ years

• Number of Participants Who Experienced Adverse Events of Thromboembolism

  Throughout the study period, approximately 4 ½ years

Study Arms (1)

All Study Participants

Biological: RIXUBIS

Interventions

RIXUBIS BIOLOGICAL

Also known as: Coagulation Factor IX [Recombinant], BAX 326, Recombinant factor IX, BAX326

All Study Participants

Eligibility Criteria

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

You may qualify if:

• Patients with hemophilia B scheduled to receive treatment with RIXUBIS

You may not qualify if:

• Patients not administered RIXUBIS

Sponsors & Collaborators

• Baxalta now part of Shire: lead
• Takeda: collaborator

Study Sites (5)

Seki-shi, Japan

Seki-shi, Gifu, 501-3802, Japan

Location

Maebashi-shi, Japan

Maebashi, Gunma, 371-8511, Japan

Location

Nobeoka-shi, Japan

Nobeoka-shi, Miyazaki, 882-0835, Japan

Location

Niigata-shi, Japan

Niigata, Niigata, 950-0862, Japan

Location

Osaka-shi, Japan

Osaka, Osaka, 543-0001, Japan

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

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

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 5, 2016
• First Posted: October 19, 2016
• Study Start: November 16, 2016
• Primary Completion: May 11, 2022
• Study Completion: May 11, 2022
• Last Updated: July 22, 2024
• Results First Posted: July 22, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

JP (5)