RIXUBIS Drug Use-Result Survey (Japan)

Study at a glance

From the registry record, last updated by the sponsor on 22 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

RIXUBIS Drug Use-Result Survey (Japan)

ClinicalTrials.gov ID

NCT02937831

Acronym / sponsor ID

251601

Conditions

Hemophilia B

Study type

Observational

Recruitment status

Completed

Enrolment

6 participants (actual)

Lead sponsor

Baxalta now part of ShireIndustry (pharma, biotech, devices)

Collaborators

Takeda

Study start

16 Nov 2016 (actual)

Primary completion

11 May 2022 (actual)

Study completion

11 May 2022 (actual)

First posted

19 Oct 2016 (estimated)

Results

Posted 22 Jul 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model

Cohort

Time perspective

Retrospective

Number of arms

1

Who may qualify

Sex

All

Healthy volunteers

Not accepted

Age groups

Child, Adult, Older adult

Study population

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination)

   Time frame: Throughout the study period, approximately 4 ½ years

   Number of participants who discontinued the use of Nonacog Gamma (Genetical Recombination) was reported in this outcome measure.

2. Number of Participants Who Developed a Factor IX (FIX) Inhibitor

   Time frame: Throughout the study period, approximately 4 ½ years

   Number of participants who developed a Factor IX (FIX) Inhibitor was reported in this outcome measure.

3. Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study

   Time frame: Throughout the study period, approximately 4 ½ years

   Annual bleed rate (ABR) was defined as the number of times of bleeding during the study. ABR was reported in this outcome measure.

4. Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen

   Time frame: At bleed resolution throughout the study period of approximately 4 ½ years

   Number of doses to treat a bleed of participants on an on-demand regimen was reported in this outcome measure.

5. Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor)

   Time frame: At bleed resolution throughout the study period of approximately 4 ½ years

   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.

6. 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

   Time frame: Assessed at the time of discharge from recovery room; and at 24 to 72 hours postoperatively

   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.

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2024.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
• To obtain more information on the study, click here/on this linkLink provided by the sponsor.
• Individual participant dataSharing planned: No. 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).

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

5 sites in 1 country: see every site and its status.