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| Name | Class |
|---|---|
| Takeda | INDUSTRY |
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The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Study Participants |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RIXUBIS | Biological |
|
|
| 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. |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who receive RIXUBIS in the actual clinical setting
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Shire | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Seki-shi, Japan | Seki-shi | Gifu | 501-3802 | Japan | ||
| Maebashi-shi, Japan |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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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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Participants with a historical diagnosis of hemophilia B were enrolled. Participants received Nonacog Gamma (Genetical Recombination) as part of a routine medical care.
Participants took part in the survey at 2 investigative sites in Japan, from November 16, 2016 to May 11, 2022.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. | Posted | Count of Participants | Participants | Throughout the study period, approximately 4 ½ years |
|
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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 1, 2020 | Apr 6, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 1, 2018 | Apr 6, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D005164 | Factor IX |
| ID | Term |
|---|---|
| D004792 | Enzyme Precursors |
| D045762 | Enzymes and Coenzymes |
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
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| 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 |
Number of participants who experienced adverse events of thromboembolism was reported in this outcome measure. |
| Throughout the study period, approximately 4 ½ years |
| Maebashi |
| Gunma |
| 371-8511 |
| Japan |
| Nobeoka-shi, Japan | Nobeoka-shi | Miyazaki | 882-0835 | Japan |
| Niigata-shi, Japan | Niigata | Niigata | 950-0862 | Japan |
| Osaka-shi, Japan | Osaka | Osaka | 543-0001 | Japan |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
|
|
| Primary | 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. | Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. | Posted | Count of Participants | Participants | Throughout the study period, approximately 4 ½ years |
|
|
|
| Primary | 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. | 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. | Posted | Mean | Standard Deviation | bleeds | Throughout the study period, approximately 4 ½ years |
|
|
|
| Primary | 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. | 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. | Posted | Number | doses | At bleed resolution throughout the study period of approximately 4 ½ years |
|
|
|
| Primary | 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. | 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. | Posted | Count of Participants | Participants | At bleed resolution throughout the study period of approximately 4 ½ years |
|
|
|
| Primary | 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. | 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). | Posted | Assessed at the time of discharge from recovery room; and at 24 to 72 hours postoperatively |
|
|
| Secondary | 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. | Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. | Posted | Count of Participants | Participants | Throughout the study period, approximately 4 ½ years |
|
|
|
| Secondary | Number of Participants Who Experienced Adverse Events of Thromboembolism | Number of participants who experienced adverse events of thromboembolism was reported in this outcome measure. | Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. | Posted | Count of Participants | Participants | Throughout the study period, approximately 4 ½ years |
|
|
|
| 0 |
| 2 |
| 0 |
| 2 |
| 1 |
| 2 |
| Gastroenteritis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
|
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.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D011506 |
| Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
| None |
|