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RIXUBIS PMS India (RIXUBIS PMS)

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ClinicalTrials.gov Identifier: NCT03565237
Recruitment Status : Recruiting
First Posted : June 21, 2018
Last Update Posted : September 5, 2018
Sponsor:
Collaborator:
Baxalta Innovations GmbH, now part of Shire
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

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

Condition or disease Intervention/treatment Phase
Hemophilia B Biological: RIXUBIS: On-Demand Biological: RIXUBIS: Prophylaxis Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: A Post Marketing Surveillance (PMS) Study of RIXUBIS in India
Actual Study Start Date : August 28, 2018
Estimated Primary Completion Date : April 21, 2021
Estimated Study Completion Date : April 21, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: All Study Participants
Study participants with Hemophilia B in India receiving Rixubis
Biological: RIXUBIS: On-Demand
RIXUBIS used under standard clinical practice in India: On-Demand treatment.
Other Names:
  • BAX326
  • Recombinant Factor IX
  • rFIX
  • Coagulation Factor IX [Recombinant]
  • rFactor IX
  • BAX 326

Biological: RIXUBIS: Prophylaxis
RIXUBIS used under standard clinical practice in India: Prophylaxis treatment.
Other Names:
  • BAX326
  • Recombinant Factor IX
  • rFIX
  • Coagulation Factor IX [Recombinant]
  • rFactor IX
  • BAX 326




Primary Outcome Measures :
  1. Incidence of serious adverse events (SAEs) [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Incidence of SAEs (including FIX inhibitors) possibly or probably related to RIXUBIS


Secondary Outcome Measures :
  1. Incidence of adverse events (AEs) [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Incidence of AEs possibly or probably related to RIXUBIS

  2. Clinically significant changes in clinical laboratory parameters [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Clinical laboratory assessments include: hematology and clinical chemistry

  3. Incidence of binding IgG antibodies [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Incidence of binding IgG antibodies to factor IX (FIX)

  4. Incidence of binding IgM antibodies [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Incidence of binding immunoglobulin M (IgM) antibodies to factor IX (FIX)

  5. Incidence of antibodies to CHO proteins [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Incidence of antibodies to Chinese hamster ovary (CHO) proteins

  6. Incidence of antibodies to rFurin [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Incidence of antibodies to recombinant Furin (rFurin)

  7. Annualized bleeding rate [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Annualized bleeding rate with prophylactic use of RIXUBIS

  8. Rate of success of RIXUBIS [ Time Frame: Throughout the study period of approximately 6 months per patient. ]
    Rate of success of RIXUBIS for treatment of bleeding episodes. Based on a four-point scale (Excellent-Good-Moderate-None)



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  1. The participant or legally authorized representative (in case of study participants <18 years of age) gave written informed consent to participate in the study.
  2. Participant has hemophilia B.
  3. 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.
  4. Participant has no evidence of a history of FIX inhibitors.
  5. 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.
  6. 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.
  7. The participant is willing and able to comply with the requirements of the protocol.

Exclusion Criteria

  1. Participant has known hypersensitivity or presence of any contraindication to RIXUBIS or its excipients including hamster protein.
  2. Participant has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
  3. 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.
  4. 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.
  5. 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.
  6. 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].
  7. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
  8. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B.
  9. Participant's platelet count is < 100,000/mL.
  10. 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.
  11. 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.
  12. 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.
  13. Participant is a family member or employee of the investigator.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03565237


Contacts
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Contact: Thomas Soucek, PhD +43-1-20-100-247-3473 thomas.soucek@shire.com
Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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India
Sahyadri Super Speciality Hospital Recruiting
Pune, Maharashtra, India, 411004
Sponsors and Collaborators
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT03565237     History of Changes
Other Study ID Numbers: 251602
First Posted: June 21, 2018    Key Record Dates
Last Update Posted: September 5, 2018
Last Verified: August 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:
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Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked