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| Name | Class |
|---|---|
| Baxalta Innovations GmbH, now part of Shire | INDUSTRY |
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The purpose of this study is to characterize the safety and describe the effectiveness of RIXUBIS in routine clinical practice.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Study Participants | Experimental | Study participants with Hemophilia B in India receiving Rixubis |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RIXUBIS: On-Demand | Biological | RIXUBIS used under standard clinical practice in India: On-Demand treatment. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs) Related to RIXUBIS | TEAE was defined as any event not presented prior to the initiation of the treatments or any event already present that worsens in either intensity or frequency following exposure to the treatments. A SAE was defined as any untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/resulted in death, life-threatening, required in-patient hospitalization or resulted in prolongation of an existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event that was not immediately life-threatening or resulted in death or required hospitalization but jeopardize the participant or required medical or surgical intervention to prevent any of the above outcomes. Relatedness to study drug was based on physician discretion. Number of participants with serious TEAEs related to RIXUBIS were reported. | From start of study drug administration up to End of treatment (EOT) (up to 6 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With TEAEs Related to RIXUBIS | TEAE was defined as any event not presented prior to the initiation of the treatments or any event already present that worsens in either intensity or frequency following exposure to the treatments. Relatedness to study drug was based on physician discretion. Number of participants with TEAEs related to RIXUBIS were reported. | From start of study drug administration up to EOT (up to 6 months) |
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Inclusion Criteria
The participant or legally authorized representative (in case of study participants <18 years of age) gave written informed consent to participate in the study.
Participant has hemophilia B.
Participant is defined as previously-treated patient (PTP):
Participant has no evidence of a history of FIX inhibitors.
Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.
Participant is hepatitis C virus negative (HCV-) by antibody or PCR testing (if positive, antibody titer will be confirmed by polymerase chain reaction (PCR)), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
The participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Sahyadri Super Speciality Hospital | Pune | Maharashtra | 411004 | India |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
A total 31 participants were screened, of which 25 participants were enrolled and received RIXUBIS treatment regimen (either on-demand or prophylaxis) based on discretion of the physician choice under standard clinical practice. No participants were enrolled in the on-demand treatment of RIXUBIS. Hence, no data collection and analysis were done during on-demand treatment of this study.
The study was conducted at 8 study sites in India from 07 December 2018 to 11 August 2021.
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| ID | Title | Description |
|---|---|---|
| FG000 | RIXUBIS: Prophylaxis Treatment | Participants received intravenous bolus of RIXUBIS prophylaxis treatment at a maximum infusion rate of 10 milliliter (mL) per (/) minute based on discretion of the physician for up to 6 months as standard clinical practice. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
SAS included all enrolled participants having received RIXUBIS at any time during the study.
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| ID | Title | Description |
|---|---|---|
| BG000 | RIXUBIS: Prophylaxis Treatment | Participants received intravenous bolus of RIXUBIS prophylaxis treatment at a maximum infusion rate of 10 mL/minute based on discretion of the physician for up to 6 months as standard clinical practice. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 With Serious Treatment-emergent Adverse Events (TEAEs) Related to RIXUBIS | TEAE was defined as any event not presented prior to the initiation of the treatments or any event already present that worsens in either intensity or frequency following exposure to the treatments. A SAE was defined as any untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/resulted in death, life-threatening, required in-patient hospitalization or resulted in prolongation of an existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event that was not immediately life-threatening or resulted in death or required hospitalization but jeopardize the participant or required medical or surgical intervention to prevent any of the above outcomes. Relatedness to study drug was based on physician discretion. Number of participants with serious TEAEs related to RIXUBIS were reported. | SAS included all enrolled participants having received RIXUBIS at any time during the study. | Posted | Count of Participants | Participants | From start of study drug administration up to End of treatment (EOT) (up to 6 months) |
From start of study drug administration up to EOT (up to 6 months)
SAS included all enrolled participants having received RIXUBIS at any time during the study.
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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 | RIXUBIS: Prophylaxis Treatment | Participants received intravenous bolus of RIXUBIS prophylaxis treatment at a maximum infusion rate of 10 mL/minute based on discretion of the physician for up to 6 months as standard clinical practice. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Non-systematic Assessment |
No participants were enrolled in the on-demand treatment of RIXUBIS. Hence, no data collection and analysis was done during on-demand treatment of this study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1 866 842 5335 | ClinicalTransparency@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 | Sep 27, 2017 | Aug 3, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 29, 2021 | Aug 3, 2022 | 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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| RIXUBIS: Prophylaxis | Biological | RIXUBIS used under standard clinical practice in India: Prophylaxis treatment. |
|
|
| Number of Participants With Clinically Significant Laboratory Abnormalities | Clinical laboratory evaluations included clinical chemistry (biochemistry and endocrinology), hematology and urinalysis. Any change in clinical laboratory abnormalities which were deemed clinically significant by the investigator were recorded as TEAEs (defined as any event not presented prior to the initiation of the treatments or any event already present that worsens in either intensity or frequency following exposure to the treatments). | From start of study drug administration up to EOT (up to 6 months) |
| Number of Participants Who Developed Binding Antibodies (Immunoglobulin G [IgG] and Immunoglobulin M [IgM]) to Factor IX (FIX) | Binding antibodies (IgG and IgM) to FIX was determined using validated enzyme-linked immunosorbent assays (ELISAs) employing polyclonal anti-human IgG and IgM antibodies. Number of participants who developed binding antibodies (IgG and IgM) combined data to FIX were reported. | From start of study drug administration up to EOT (up to 6 months) |
| Number of Participants Who Developed Binding Antibodies to Chinese Hamster Ovary (CHO) Proteins and rFurin | Citrated plasma was assayed for the presence of antibodies to CHO protein and rFurin, derived from cultures of un-transfected cells. Testing for binding anti-CHO protein and rFurin antibodies was done on citrate-anti-coagulated plasma using an ELISA employing polyclonal anti-human IgG antibodies. Number of participants who developed binding antibodies to CHO proteins and rFurin were reported. | From start of study drug administration up to EOT (up to 6 months) |
| Annualized Bleeding Rate (ABR) With Prophylactic Use of RIXUBIS | The ABR was defined as the total number of unique bleeding episodes by participants reported during RIXUBIS treatment for prophylaxis, divided by the RIXUBIS treatment duration for prophylaxis and multiplied by 365.25. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not be associated to a trauma event (spontaneous bleeding). Bleeding occurring at multiple locations related to the same injury (e.g., knee and ankle bleed following a fall) was counted as a single bleeding episode. | From start of study drug administration up to EOT (up to 6 months) |
| Rate of Success of RIXUBIS for Treatment of Bleeding Episodes | The success of RIXUBIS for treatment of bleeding episodes was defined by grouping the categories of "Excellent"/"Good" of the corresponding hemostatic effectiveness ratings of a 4 point Likert scale ("Excellent", "Good", "Moderate" and "None") by the participants/legally authorized representative (LAR) (participants less than (<) 12 years: LAR, participants greater than or equal to (>=) 12 years: self-assessment) for treatments given at home, or by the investigator for treatments given in the hospital/clinic. The rate of success of RIXUBIS for treatment of bleeding episodes was defined as: The number of successful bleeding episodes/total number of bleeding episodes where the treatment of the bleeding was rated *100. Rate of success of RIXUBIS for treatment of bleeding episodes, 95% confidence interval (CIs) was calculated using the exact Binomial CI (Clopper-Pearson method). | From screening up to EOT (up to 6 months) |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | RIXUBIS: Prophylaxis Treatment | Participants received intravenous bolus of RIXUBIS prophylaxis treatment at a maximum infusion rate of 10 mL/minute based on discretion of the physician for up to 6 months as standard clinical practice. |
|
|
| Secondary | Number of Participants With TEAEs Related to RIXUBIS | TEAE was defined as any event not presented prior to the initiation of the treatments or any event already present that worsens in either intensity or frequency following exposure to the treatments. Relatedness to study drug was based on physician discretion. Number of participants with TEAEs related to RIXUBIS were reported. | SAS included all enrolled participants having received RIXUBIS at any time during the study. | Posted | Count of Participants | Participants | From start of study drug administration up to EOT (up to 6 months) |
|
|
|
| Secondary | Number of Participants With Clinically Significant Laboratory Abnormalities | Clinical laboratory evaluations included clinical chemistry (biochemistry and endocrinology), hematology and urinalysis. Any change in clinical laboratory abnormalities which were deemed clinically significant by the investigator were recorded as TEAEs (defined as any event not presented prior to the initiation of the treatments or any event already present that worsens in either intensity or frequency following exposure to the treatments). | SAS included all enrolled participants having received RIXUBIS at any time during the study. | Posted | Count of Participants | Participants | From start of study drug administration up to EOT (up to 6 months) |
|
|
|
| Secondary | Number of Participants Who Developed Binding Antibodies (Immunoglobulin G [IgG] and Immunoglobulin M [IgM]) to Factor IX (FIX) | Binding antibodies (IgG and IgM) to FIX was determined using validated enzyme-linked immunosorbent assays (ELISAs) employing polyclonal anti-human IgG and IgM antibodies. Number of participants who developed binding antibodies (IgG and IgM) combined data to FIX were reported. | SAS included all enrolled participants having received RIXUBIS at any time during the study. | Posted | Count of Participants | Participants | From start of study drug administration up to EOT (up to 6 months) |
|
|
|
| Secondary | Number of Participants Who Developed Binding Antibodies to Chinese Hamster Ovary (CHO) Proteins and rFurin | Citrated plasma was assayed for the presence of antibodies to CHO protein and rFurin, derived from cultures of un-transfected cells. Testing for binding anti-CHO protein and rFurin antibodies was done on citrate-anti-coagulated plasma using an ELISA employing polyclonal anti-human IgG antibodies. Number of participants who developed binding antibodies to CHO proteins and rFurin were reported. | SAS included all enrolled participants having received RIXUBIS at any time during the study. | Posted | Count of Participants | Participants | From start of study drug administration up to EOT (up to 6 months) |
|
|
|
| Secondary | Annualized Bleeding Rate (ABR) With Prophylactic Use of RIXUBIS | The ABR was defined as the total number of unique bleeding episodes by participants reported during RIXUBIS treatment for prophylaxis, divided by the RIXUBIS treatment duration for prophylaxis and multiplied by 365.25. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not be associated to a trauma event (spontaneous bleeding). Bleeding occurring at multiple locations related to the same injury (e.g., knee and ankle bleed following a fall) was counted as a single bleeding episode. | The EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. Here "Overall number of participants analyzed" signifies participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | episodes per participant per year | From start of study drug administration up to EOT (up to 6 months) |
|
|
|
| Secondary | Rate of Success of RIXUBIS for Treatment of Bleeding Episodes | The success of RIXUBIS for treatment of bleeding episodes was defined by grouping the categories of "Excellent"/"Good" of the corresponding hemostatic effectiveness ratings of a 4 point Likert scale ("Excellent", "Good", "Moderate" and "None") by the participants/legally authorized representative (LAR) (participants less than (<) 12 years: LAR, participants greater than or equal to (>=) 12 years: self-assessment) for treatments given at home, or by the investigator for treatments given in the hospital/clinic. The rate of success of RIXUBIS for treatment of bleeding episodes was defined as: The number of successful bleeding episodes/total number of bleeding episodes where the treatment of the bleeding was rated *100. Rate of success of RIXUBIS for treatment of bleeding episodes, 95% confidence interval (CIs) was calculated using the exact Binomial CI (Clopper-Pearson method). | The EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. Here "Overall number of participants analyzed" signifies participants who were evaluable for this outcome measure. | Posted | Number | 95% Confidence Interval | percentage of bleeding episodes | From screening up to EOT (up to 6 months) | Bleeding episodes | Bleeding episodes |
|
|
|
| 0 |
| 25 |
| 0 |
| 25 |
| 3 |
| 25 |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Non-systematic Assessment |
|
| Dengue fever | Infections and infestations | MedDRA 24.0 | Non-systematic Assessment |
|
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| 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 |
| Title | Measurements |
|---|---|
|