Trial Outcomes & Findings for RIXUBIS PMS India (RIXUBIS PMS) (NCT NCT03565237)
NCT ID: NCT03565237
Last Updated: 2022-08-30
Results Overview
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.
COMPLETED
PHASE4
25 participants
From start of study drug administration up to End of treatment (EOT) (up to 6 months)
2022-08-30
Participant Flow
The study was conducted at 8 study sites in India from 07 December 2018 to 11 August 2021.
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.
Participant milestones
| Measure |
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.
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|---|---|
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Overall Study
STARTED
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25
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Overall Study
Safety Analysis Set (SAS)
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25
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Overall Study
Effectiveness Full Analysis Set (EFAS)
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25
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|
Overall Study
COMPLETED
|
23
|
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Overall Study
NOT COMPLETED
|
2
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Reasons for withdrawal
| Measure |
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.
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|---|---|
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Overall Study
Non-compliance of Investigational Product
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1
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Overall Study
Physician Decision
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1
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Baseline Characteristics
RIXUBIS PMS India (RIXUBIS PMS)
Baseline characteristics by cohort
| Measure |
RIXUBIS: Prophylaxis Treatment
n=25 Participants
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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|---|---|
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Age, Continuous
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24.6 years
STANDARD_DEVIATION 8.29 • n=5 Participants
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Sex: Female, Male
Female
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0 Participants
n=5 Participants
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Sex: Female, Male
Male
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25 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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25 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
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Race/Ethnicity, Customized
Race · Asian: Indian
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24 Participants
n=5 Participants
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Race/Ethnicity, Customized
Race · More than one race
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1 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: From start of study drug administration up to End of treatment (EOT) (up to 6 months)Population: SAS included all enrolled participants having received RIXUBIS at any time during the study.
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.
Outcome measures
| Measure |
RIXUBIS: Prophylaxis Treatment
n=25 Participants
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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|---|---|
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Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs) Related to RIXUBIS
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0 Participants
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SECONDARY outcome
Timeframe: From start of study drug administration up to EOT (up to 6 months)Population: SAS included all enrolled participants having received RIXUBIS at any time during the study.
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.
Outcome measures
| Measure |
RIXUBIS: Prophylaxis Treatment
n=25 Participants
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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Number of Participants With TEAEs Related to RIXUBIS
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0 Participants
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SECONDARY outcome
Timeframe: From start of study drug administration up to EOT (up to 6 months)Population: SAS included all enrolled participants having received RIXUBIS at any time during the study.
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).
Outcome measures
| Measure |
RIXUBIS: Prophylaxis Treatment
n=25 Participants
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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|---|---|
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Number of Participants With Clinically Significant Laboratory Abnormalities
Hematology
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0 Participants
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Number of Participants With Clinically Significant Laboratory Abnormalities
Clinical chemistry
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0 Participants
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Number of Participants With Clinically Significant Laboratory Abnormalities
Urinalysis
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0 Participants
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SECONDARY outcome
Timeframe: From start of study drug administration up to EOT (up to 6 months)Population: SAS included all enrolled participants having received RIXUBIS at any time during the study.
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.
Outcome measures
| Measure |
RIXUBIS: Prophylaxis Treatment
n=25 Participants
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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|---|---|
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Number of Participants Who Developed Binding Antibodies (Immunoglobulin G [IgG] and Immunoglobulin M [IgM]) to Factor IX (FIX)
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1 Participants
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SECONDARY outcome
Timeframe: From start of study drug administration up to EOT (up to 6 months)Population: SAS included all enrolled participants having received RIXUBIS at any time during the study.
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.
Outcome measures
| Measure |
RIXUBIS: Prophylaxis Treatment
n=25 Participants
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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|---|---|
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Number of Participants Who Developed Binding Antibodies to Chinese Hamster Ovary (CHO) Proteins and rFurin
CHO Proteins
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0 Participants
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Number of Participants Who Developed Binding Antibodies to Chinese Hamster Ovary (CHO) Proteins and rFurin
rFurin
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0 Participants
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SECONDARY outcome
Timeframe: From start of study drug administration up to EOT (up to 6 months)Population: 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.
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.
Outcome measures
| Measure |
RIXUBIS: Prophylaxis Treatment
n=23 Participants
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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|---|---|
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Annualized Bleeding Rate (ABR) With Prophylactic Use of RIXUBIS
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0.914 episodes per participant per year
Standard Deviation 1.6896
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SECONDARY outcome
Timeframe: From screening up to EOT (up to 6 months)Population: 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.
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).
Outcome measures
| Measure |
RIXUBIS: Prophylaxis Treatment
n=6 Bleeding episodes
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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|---|---|
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Rate of Success of RIXUBIS for Treatment of Bleeding Episodes
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100 percentage of bleeding episodes
Interval 54.1 to 100.0
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Adverse Events
RIXUBIS: Prophylaxis Treatment
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
RIXUBIS: Prophylaxis Treatment
n=25 participants at risk
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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|---|---|
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Musculoskeletal and connective tissue disorders
Arthropathy
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4.0%
1/25 • Number of events 1 • 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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Musculoskeletal and connective tissue disorders
Joint swelling
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4.0%
1/25 • Number of events 1 • 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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|
Infections and infestations
Dengue fever
|
4.0%
1/25 • Number of events 1 • 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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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER