Trial Outcomes & Findings for RIXUBIS Drug Use-Result Survey (Japan) (NCT NCT02937831)

Results Overview

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

Recruitment status

COMPLETED

Target enrollment

6 participants

Primary outcome timeframe

Throughout the study period, approximately 4 ½ years

Results posted on

2024-07-22

Participant Flow

Participants took part in the survey at 2 investigative sites in Japan, from November 16, 2016 to May 11, 2022.

Participants with a historical diagnosis of hemophilia B were enrolled. Participants received Nonacog Gamma (Genetical Recombination) as part of a routine medical care.

Participant milestones

Participant milestones
Measure	Nonacog Gamma (Genetical Recombination) Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Overall Study STARTED	6
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Nonacog Gamma (Genetical Recombination) Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Overall Study Protocol Violation	4

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nonacog Gamma (Genetical Recombination) n=2 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Age, Categorical <=18 years	0 Participants n=2 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=2 Participants
Age, Categorical >=65 years	0 Participants n=2 Participants
Sex: Female, Male Female	0 Participants n=2 Participants
Sex: Female, Male Male	2 Participants n=2 Participants
Region of Enrollment Japan	2 Participants n=2 Participants

PRIMARY outcome

Timeframe: Throughout the study period, approximately 4 ½ years

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

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

Outcome measures

Outcome measures
Measure	Nonacog Gamma (Genetical Recombination) n=2 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination)	1 Participants

PRIMARY outcome

Timeframe: Throughout the study period, approximately 4 ½ years

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

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

Outcome measures

Outcome measures
Measure	Nonacog Gamma (Genetical Recombination) n=2 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Number of Participants Who Developed a Factor IX (FIX) Inhibitor	0 Participants

PRIMARY outcome

Timeframe: Throughout the study period, approximately 4 ½ years

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received routine prophylactic therapy.

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

Outcome measures

Outcome measures
Measure	Nonacog Gamma (Genetical Recombination) n=1 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study	0 bleeds Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.

PRIMARY outcome

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

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received an on-demand regimen.

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

Outcome measures

Outcome measures
Measure	Nonacog Gamma (Genetical Recombination) n=1 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen	1 doses

PRIMARY outcome

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

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received an on-demand regimen.

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.

Outcome measures

Outcome measures
Measure	Nonacog Gamma (Genetical Recombination) n=1 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) Excellent	1 Participants
Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) Good	0 Participants
Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) Moderate	0 Participants
Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) None	0 Participants

PRIMARY outcome

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

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received perioperative therapy. Number of participants who received perioperative therapy during this study was zero (N=0).

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Throughout the study period, approximately 4 ½ years

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

Number of participants who experienced adverse events of shock or anaphylaxis was reported in this outcome measure.

Outcome measures

Outcome measures
Measure	Nonacog Gamma (Genetical Recombination) n=2 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Number of Participants Who Experienced Adverse Events of Shock or Anaphylaxis	0 Participants

SECONDARY outcome

Timeframe: Throughout the study period, approximately 4 ½ years

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

Number of participants who experienced adverse events of thromboembolism was reported in this outcome measure.

Outcome measures

Outcome measures
Measure	Nonacog Gamma (Genetical Recombination) n=2 Participants Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Number of Participants Who Experienced Adverse Events of Thromboembolism	0 Participants

Adverse Events

Nonacog Gamma (Genetical Recombination)

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Nonacog Gamma (Genetical Recombination) n=2 participants at risk Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care.
Infections and infestations Upper respiratory tract infection	50.0% 1/2 • Throughout the study period, approximately 4 ½ years At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Gastroenteritis	50.0% 1/2 • Throughout the study period, approximately 4 ½ years At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.

Additional Information

Study Director

Takeda

Phone: +1-877-825-3327

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Research Organization shall not publish any articles or papers nor make any presentations, nor assist any other person in publishing any articles or papers or in making any presentations relating or referring to the Study or any results, data or insights from or any data, information or materials obtained or generated in the performance of its obligations without the prior written consent of Takeda, which consent may be granted or withheld in Takeda's sole discretion.
Publication restrictions are in place

Restriction type: OTHER