BeneFIX Drug Use Results Survey [All-Case Surveillance]

• ClinicalTrials.gov Identifier: NCT01154231
• Recruitment Status: Completed
• First Posted: June 30, 2010
• Results First Posted: April 11, 2017
• Last Update Posted: October 17, 2018
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment
Hemophilia B	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 Design

• Study Type: Observational
• Actual Enrollment: 314 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Benefix(Registered) Intravenous 500 1000 2000 Drug Use Results Survey (All-case Surveillance)
• Study Start Date: February 2010
• Actual Primary Completion Date: August 2014
• Actual Study Completion Date: February 27, 2017

Groups and Cohorts

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 Name: BeneFIX

Outcome Measures

Primary Outcome Measures :

1. Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy [ Time Frame: 2 years for PTPs, 1 year for PUPs ]
   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. Number of Administrations Required for Hemostasis for Bleeding Events [ Time Frame: 2 years for PTPs, 1 year for PUPs ]
   Mean number of administrations for hemostasis in replacement therapy for bleeding events.

Secondary Outcome Measures :

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

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.

Criteria

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.

Contacts and Locations

Locations

Japan

Tokyo Medical University Hospital

Shinjuku-ku, Tokyo, Japan

Sponsors and Collaborators

Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

More Information

Additional Information:

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

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT01154231
• Other Study ID Numbers: 3090X1-4415; B1821004; B1821009 ( Other Identifier: Alias Study Number )
• First Posted: June 30, 2010
• Results First Posted: April 11, 2017
• Last Update Posted: October 17, 2018
• Last Verified: September 2018

Keywords provided by Pfizer:

BeneFIX Regulatory Post Marketing Commitment Plan

Additional relevant MeSH terms:

Hemophilia B; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked