Hemophilia BPhase 4NCT03565237
RIXUBIS PMS India (RIXUBIS PMS)
A Phase 4 study testing RIXUBIS: On-Demand and RIXUBIS: Prophylaxis in people with Hemophilia B, run by Baxalta now part of Shire at 1 site in India.
Trial summary
The purpose of this study is to characterize the safety and describe the effectiveness of RIXUBIS in routine clinical practice. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia B
- What is being tested?
- RIXUBIS: On-Demand and RIXUBIS: Prophylaxis
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteIndia
- Study dates
- 7 Dec 2018to 11 Aug 2021
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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 aged ≥ 6 years that has been previously treated with plasma-derived and/or recombinant factor IX (FIX) concentrate(s) for a minimum of 150 exposure days (EDs).
- Participant aged < 6 years that has been previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 50 EDs.
- Participant has no evidence of a history of FIX inhibitors.
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- 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.
Who cannot join
- Participant has known hypersensitivity or presence of any contraindication to RIXUBIS or its excipients including hamster protein.
- Participant has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
- Participant has a history of FIX inhibitors with a titer ≥ 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay, employed in the respective local laboratory) at any time prior to screening.
- Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory.
- Participant has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4 hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
- Participant has severe chronic hepatic dysfunction [eg, ≥ 5 times upper limit of normal alanine aminotransferase (ALT), as confirmed by central laboratory at screening, or a documented INR > 1.5].
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- Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B.
- Participant's platelet count is < 100,000/mL.
- Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant's safety or compliance.
- Participant is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than antiretroviral chemotherapy.
- Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
- Participant is a family member or employee of the investigator.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase IV Multi-Center, Prospective, Interventional, Post-Marketing Study in Hemophilia B Patients in India Receiving RIXUBIS as On-demand or Prophylaxis Under Standard Clinical Practice
The purpose of this study is to characterize the safety and describe the effectiveness of RIXUBIS in routine clinical practice.
- Conditions
- Hemophilia B
- Being tested
- RIXUBIS: On-Demand, RIXUBIS: Prophylaxis
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RIXUBIS: On-DemandBiological
Also called BAX326, Recombinant Factor IX, rFIX, Coagulation Factor IX [Recombinant]
RIXUBIS used under standard clinical practice in India: On-Demand treatment.
RIXUBIS: ProphylaxisBiological
Also called BAX326, Recombinant Factor IX, rFIX, Coagulation Factor IX [Recombinant]
RIXUBIS used under standard clinical practice in India: Prophylaxis treatment.
| Study group | Role | What they receive |
|---|---|---|
| All Study Participants | Experimental | Study participants with Hemophilia B in India receiving Rixubis Receives: Biological: RIXUBIS: On-Demand; Biological: RIXUBIS: Prophylaxis |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From start of study drug administration up to End of treatment (EOT) (up to 6 months)
Secondary outcomes (6)
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.
Measured: From start of study drug administration up to EOT (up to 6 months)
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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: From screening up to EOT (up to 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2018
- Study start7 Dec 2018
- Primary completion11 Aug 2021
- Study completion11 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03565237
- Sponsor's protocol ID
- 251602
- Status last verified
- Aug 2022
- First submitted
- 11 Jun 2018
- Last updated
- 30 Aug 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IV Multi-Center, Prospective, Interventional, Post-Marketing Study in Hemophilia B Patients in India Receiving RIXUBIS as On-demand or Prophylaxis Under Standard Clinical Practice
- ClinicalTrials.gov ID
- NCT03565237
- Acronym / sponsor ID
- 251602
- Conditions
- Hemophilia B
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Study start
- 7 Dec 2018 (actual)
- Primary completion
- 11 Aug 2021 (actual)
- Study completion
- 11 Aug 2021 (actual)
- First posted
- 21 Jun 2018 (actual)
- Results
- Posted 30 Aug 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to characterize the safety and describe the effectiveness of RIXUBIS in routine clinical practice.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Study Participants | Experimental | Biological: RIXUBIS: On-Demand, Biological: RIXUBIS: ProphylaxisStudy participants with Hemophilia B in India receiving Rixubis |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (13 listed)
- Participant has known hypersensitivity or presence of any contraindication to RIXUBIS or its excipients including hamster protein.
- Participant has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
- Participant has a history of FIX inhibitors with a titer ≥ 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay, employed in the respective local laboratory) at any time prior to screening.
- Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory.
- Participant has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4 hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RIXUBIS: On-DemandAlso: BAX326, Recombinant Factor IX, rFIX, Coagulation Factor IX [Recombinant]RIXUBIS used under standard clinical practice in India: On-Demand treatment. | Biological | All Study Participants |
| RIXUBIS: ProphylaxisAlso: BAX326, Recombinant Factor IX, rFIX, Coagulation Factor IX [Recombinant]RIXUBIS used under standard clinical practice in India: Prophylaxis treatment. | Biological | All Study Participants |
Primary outcome
Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs) Related to RIXUBIS
Time frame: From start of study drug administration up to End of treatment (EOT) (up to 6 months)
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.
6 secondary outcomes
Number of Participants With TEAEs Related to RIXUBIS
Time frame: From start of study drug administration up to EOT (up to 6 months)
Number of Participants With Clinically Significant Laboratory Abnormalities
Time frame: 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)
Time frame: 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
Time frame: From start of study drug administration up to EOT (up to 6 months)
Annualized Bleeding Rate (ABR) With Prophylactic Use of RIXUBIS
Time frame: From start of study drug administration up to EOT (up to 6 months)
Rate of Success of RIXUBIS for Treatment of Bleeding Episodes
Time frame: From screening up to EOT (up to 6 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Aug 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing RIXUBIS: On-Demand and RIXUBIS: Prophylaxis in people with Hemophilia B, run by Baxalta now part of Shire at 1 site in India.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in India. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Aug 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs) Related to RIXUBIS (time frame: From start of study drug administration up to End of treatment (EOT) (up to 6 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Aug 2022; AzTrials last refreshed its copy on 29 Oct 2026.