RIXUBIS PMS India (RIXUBIS PMS)

NCT03565237 | Completed Phase 4 Results FDA Drug

• 1 other identifier: 251602
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to characterize the safety and describe the effectiveness of RIXUBIS in routine clinical practice.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• 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)

Secondary Outcomes (6)

• Number of Participants With TEAEs Related to RIXUBIS

  From start of study drug administration up to EOT (up to 6 months)

• Number of Participants With Clinically Significant Laboratory Abnormalities

  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)

  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

  From start of study drug administration up to EOT (up to 6 months)

• Annualized Bleeding Rate (ABR) With Prophylactic Use of RIXUBIS

  From start of study drug administration up to EOT (up to 6 months)

Study Arms (1)

All Study Participants

EXPERIMENTAL

Study participants with Hemophilia B in India receiving Rixubis

Biological: RIXUBIS: On-Demand; Biological: RIXUBIS: Prophylaxis

Interventions

RIXUBIS: On-Demand BIOLOGICAL

RIXUBIS used under standard clinical practice in India: On-Demand treatment.

Also known as: BAX326, Recombinant Factor IX, rFIX, Coagulation Factor IX [Recombinant], rFactor IX, BAX 326

All Study Participants

RIXUBIS: Prophylaxis BIOLOGICAL

RIXUBIS used under standard clinical practice in India: Prophylaxis treatment.

Also known as: BAX326, Recombinant Factor IX, rFIX, Coagulation Factor IX [Recombinant], rFactor IX, BAX 326

All Study Participants

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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 \[eg, ≥ 5 times upper limit of normal alanine aminotransferase (ALT), as confirmed by central laboratory at screening, or a documented INR \> 1.5\].
• 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.

Sponsors & Collaborators

• Baxalta now part of Shire: lead
• Baxalta Innovations GmbH, now part of Shire: collaborator

Study Sites (1)

Sahyadri Super Speciality Hospital

Pune, Maharashtra, 411004, India

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Limitations and Caveats

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.

Results Point of Contact

• Title: Study Director
• Organization: Takeda

ClinicalTransparency@takeda.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2018
• First Posted: June 21, 2018
• Study Start: December 7, 2018
• Primary Completion: August 11, 2021
• Study Completion: August 11, 2021
• Last Updated: August 30, 2022
• Results First Posted: August 30, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: 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.

Locations

IN (1)