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A Post-marketing study receiving RIXUBIS as on-demand or Prophylaxis under standard clinical practice in patients with Hemophilia B

Phase 4
Conditions
Health Condition 1: null- Hemophilia B
Registration Number
CTRI/2018/07/014754
Lead Sponsor
Baxalta Innovations GmbH
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Closed to Recruitment of Participants
Sex
Not specified
Target Recruitment
0
Inclusion Criteria

1 The subject or legally authorized representative (in case of study participants <18 years of age) gave written informed consent to participate in the study.

2 Subject has Hemophilia B.

3 Subject is defined as previously-treated patient (PTP):

�Subject aged greater than equal to 6 years that has been previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 150 EDs

�Subject aged less than years that has been previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 50 EDs

4 Subject has no evidence of a history of FIX inhibitors.

5 Subject is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count � 200 cells/mm3, as confirmed by central laboratory at screening.

6 Subject is hepatitis C virus negative (HCV-) by antibody or PCR testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.

7 The subject is willing and able to comply with the requirements of the protocol

Exclusion Criteria

1.Subject has known hypersensitivity or presence of any contraindication to RIXUBIS or its excipients including hamster protein.

2.Subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).

3.Subject 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.Subject 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.Subject 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.Subject 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.Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.

8.Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B.

9.Subjectââ?¬•s platelet count is < 100,000/mL.

10.Subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subjectââ?¬•s safety or compliance.

11.Subject 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 �±-interferon) other than antiretroviral chemotherapy.

12.Subject has participated in another clinical study involving an 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.Subject is a family member or employee of the investigator.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
The primary outcome measure is incidence of SAEs (including FIX inhibitors) possibly or probably related to RIXUBIS.Timepoint: <br/ ><br>Throughout the study period of approximately 6 months per patient.
Secondary Outcome Measures
NameTimeMethod
Annualized bleeding rate with prophylactic use of RIXUBISTimepoint: Throughout the study period of approximately 6 months per patient.;Clinically significant changes in clinical laboratory parameters (hematology and clinical chemistry)Timepoint: <br/ ><br>Throughout the study period of approximately 6 months per patient.;Incidence of AEs possibly or probably related to RIXUBISTimepoint: <br/ ><br>Throughout the study period of approximately 6 months per patient.;Incidence of antibodies to CHO proteins and rFurinTimepoint: <br/ ><br>Throughout the study period of approximately 6 months per patient.;Incidence of binding IgG and IgM antibodies to Factor IX (FIX)Timepoint: Throughout the study period of approximately 6 months per patient.;Rate of success of RIXUBIS for treatment of bleeding episodesTimepoint: <br/ ><br>Throughout the study period of approximately 6 months per patient.
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