Clinical trial NCT02937831: RIXUBIS Drug Use-Result Survey (Japan)
Official title: RIXUBIS Drug Use-Result Survey (Japan)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia B
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda
First posted: 2016-10-19
Enrollment: 6
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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

Brief summary: 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


Eligibility criteria: Inclusion Criteria:

* Patients with hemophilia B scheduled to receive treatment with RIXUBIS

Exclusion Criteria:

* Patients not administered RIXUBIS

Primary outcomes:
measure: Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination)
timeFrame: Throughout the study period, approximately 4 ½ years
measure: Number of Participants Who Developed a Factor IX (FIX) Inhibitor
timeFrame: Throughout the study period, approximately 4 ½ years
measure: Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study
timeFrame: Throughout the study period, approximately 4 ½ years
measure: Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen
timeFrame: At bleed resolution throughout the study period of approximately 4 ½ years
measure: Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor)
timeFrame: At bleed resolution throughout the study period of approximately 4 ½ years
measure: 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
timeFrame: Assessed at the time of discharge from recovery room; and at 24 to 72 hours postoperatively
Secondary outcomes:
measure: Number of Participants Who Experienced Adverse Events of Shock or Anaphylaxis
timeFrame: Throughout the study period, approximately 4 ½ years
measure: Number of Participants Who Experienced Adverse Events of Thromboembolism
timeFrame: Throughout the study period, approximately 4 ½ years
Other outcomes:
Locations:
city: Seki-shi
country: Japan
facility: Seki-shi, Japan
state: Gifu Prefecture
city: Maebashi-shi
country: Japan
facility: Maebashi-shi, Japan
state: Gunma Prefecture
city: Nobeoka-shi
country: Japan
facility: Nobeoka-shi, Japan
state: Miyazaki Prefecture
city: Niigata-shi
country: Japan
facility: Niigata-shi, Japan
state: Niigata Prefecture
city: Osaka-shi
country: Japan
facility: Osaka-shi, Japan
state: Osaka Prefecture
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: