BeneFIX Drug Use Results Survey [All-Case Surveillance]

Benefix(Registered) Intravenous 500 1000 2000 Drug Use Results Survey (All-case Surveillance)

The survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch.

1. Occurrence status of adverse events
2. Factors that may influence the safety
3. Efficacy In addition, the following occurrence statuses will be investigated as priority items of the survey: Incidence rate of inhibitor, reduction in drug, efficacy, Allergic reaction, and Thrombosis.

Study Overview

• Status: Completed
• Conditions: Hemophilia B
• Intervention / Treatment: Drug: Nonacog Alfa (Genetical Recombination)

Detailed Description

Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug.

Patients with hemophilia B are either patients for whom treatment was already started with a blood coagulation factor IX product ("Previously treated patients": hereinafter, "PTPs") or patients who have no history of treatment with a blood coagulation factor IX product in the past and for whom treatment will be started with this drug for the first time ("Previously untreated patients": hereinafter, "PUPs").

The definition of PUPs in this survey is "Patients in whom the total number of days of administration of a blood coagulation factor IX product other than this drug before administration of this drug was 3 exposure days (actual number of days of administration: hereinafter, "EDs") or less."

• Study Type: Observational
• Enrollment (Actual): 314

Contacts and Locations

Study Locations

• Japan
  • Tokyo
    • Shinjuku-ku, Tokyo, Japan
      • Tokyo Medical University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug.

Description

Inclusion Criteria:

• All patients with hemophilia B scheduled to receive treatment with BeneFIX will be eligible for the surveillance.
• No patient will be excluded because of prior inhibitor history; however, complete patient history and demographics will be collected.

Exclusion Criteria:

• Patients not administered BeneFIX.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Nonacog Alfa (Genetical Recombination)

Drug: Nonacog Alfa (Genetical Recombination); Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug. Patients with hemophilia B are either patients for whom treatment was already started with a blood coagulation factor IX product ("Previously treated patients": hereinafter, "PTPs") or patients who have no history of treatment with a blood coagulation factor IX product in the past and for whom treatment will be started with this drug for the first time ("Previously untreated patients": hereinafter, "PUPs"). The definition of PUPs in this survey is "Patients in whom the total number of days of administration of a blood coagulation factor IX product other than this drug before administration of this drug was 3 exposure days (actual number of days of administration: hereinafter, "EDs") or less."; Other Names: BeneFIX

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy	Annual bleeding rate (ABR) was calculated as the number of total bleeding events occured during prophylaxis period devided by the period (events/year). ABR for other replacement treatment period was also calculated to evaluate the differences between types of treatment. If the period used for ABR calculation was less or equal than 7 days, the relevant data was regarded as missing.	2 years for PTPs, 1 year for PUPs
Number of Administrations Required for Hemostasis for Bleeding Events	Mean number of administrations for hemostasis in replacement therapy for bleeding events.	2 years for PTPs, 1 year for PUPs

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes	Propotion of subjects whose physicians evaluated the effect of BeneFIX for bleeding episodes as excellent or good was calculated.	2 years for PTPs, 1 year for PUPs

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start: February 1, 2010
• Primary Completion (Actual): August 1, 2014
• Study Completion (Actual): February 27, 2017

Study Registration Dates

• First Submitted: June 28, 2010
• First Submitted That Met QC Criteria: June 29, 2010
• First Posted (Estimate): June 30, 2010

Study Record Updates

• Last Update Posted (Actual): October 17, 2018
• Last Update Submitted That Met QC Criteria: September 18, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Keywords: BeneFIX Regulatory Post Marketing Commitment Plan
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia B
• Other Study ID Numbers: 3090X1-4415; B1821004; B1821009 (Other Identifier: Alias Study Number)