Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B

Post Marketing Surveillance To Observe Safety And Efficacy Of BeneFIX In Patients With Hemophilia B

To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including:

1) Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses.

3) Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug

Study Overview

• Status: Completed
• Conditions: Hemophilia B
• Intervention / Treatment: Drug: BeneFIX (coagulation factor IX (recombinant))

Detailed Description

The patients who meet the inclusion criteria will be enrolled consecutively.

• Study Type: Observational
• Enrollment (Actual): 183

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 137-882
    • Pfizer Investigational Site

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Hemophilia B (congenital factor IX deficiency or Christmas disease).

Description

Inclusion Criteria:

• Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement).
• Pediatric and adult patients who have been treated with original or reformulated BeneFIX for hemophilia B (congenital factor IX deficiency or Christmas disease) from first approved date by KFDA, or who are planned to be newly prescribed BeneFIX (for example, patients switching from pdFIX to BeneFIX).

Exclusion Criteria:

• Patients with a known history of hypersensitivity to original or reformulated BeneFIX or any component of the product.
• Patients with a known history of hypersensitivity to hamster protein.
• Patients participating in an interventional trial of any investigational drug or device.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
1	Drug: BeneFIX (coagulation factor IX (recombinant)); BeneFIX will be administered according to physician's discretion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events (AEs) According to Baseline Characteristics	AE: any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. AE assessed by baseline characteristics (chr) included age, gender, pediatric/geriatric status, liver disorder, BeneFIX treatment (previously/newly), factor nine (FIX) gene mutation, prior exposure to plasma-derived FIX products, prior FIX regimen(s) utilized, personal history of FIX inhibitor, family history of hemophilia B, severity of bleeding, medical history, concomitant medication and therapy.	Baseline up to 6 months
Number of Participants With Adverse Events (AEs) According to Severity	AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AE was assessed according to severity; mild (not causing any significant problem, dose adjustment not required), moderate (caused problem that does not interfere significantly with usual activities or the clinical status, dose adjustment needed due to adverse event) and severe (caused problem that interferes significantly with usual activities or the clinical status, study drug stopped due to adverse event).	Baseline up to 6 months
Number of Participants With Action Taken in Response to Adverse Events (AEs)	AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. After an onset of an AE, relevant actions were undertaken on the study drug or the participant. Actions related to study drug included: dosage reduced, dosage increased, stopped temporarily or permanently, no action taken; actions related to participants included: withdrawal from the study, concomitant medication, no action taken or any other as per physician's discretion.	Baseline up to 6 months
Number of Participants With Adverse Events (AEs) According to Seriousness	AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Seriousness of an AE was assessed under the criteria of serious adverse event (SAE). An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.	Baseline up to 6 months
Number of Participants With Outcome in Response to Adverse Events (AEs)	AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Outcome of an AE was assessed based on response to a question 'Is the adverse event still present?' as 'yes', 'unknown' or 'no-resolved'.	Baseline up to 6 months
Number of Participants With Adverse Events (AEs) by Relationship	AE: untoward medical occurrence in participant who received study drug without regard to causal relationship. All causalities and drug-related AEs reported. Drug-related AEs based on physician's discretion: certain (AE after drug intake, not explained by other drugs, reaction on drug cessation [DC], relapse on re-intake of drug), probable/likely (AE after drug intake, not explained by other drugs, reaction on DC, no information on re-intake), possible (AE after drug intake, explained by other drugs, no information on DC), unlikely (not related to drug intake time, explained by other drugs).	Baseline up to 6 months
Number of Participants With Unexpected Adverse Events (AEs)	AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Unexpected AEs were those that were not included in precaution of local product document.	Baseline up to 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Annualized Bleeding Rate (ABR)	An annualized bleeding rate (ABR) was calculated as the number of bleeds requiring administration of BeneFIX (for on-demand therapy and surgery), divided by total period of bleeding multiplied by 365.25. Total period of bleeding is the number of days on treatment for prophylaxis purpose and on-demand therapy and surgery.	Baseline up to 6 months
Number of Responses to On-demand Treatment With Study Medication	Responses to on-demand treatment were rated by participant/caregiver or physician each time the drug was administered, on 4-point scale. Score 1=excellent (definite pain relief [PR] and improvement [imp] within 8 hours [h] of infusion [inf], no additional inf); score 2=good (definite PR and imp within 8h of inf, at least 1 additional inf for complete resolution [CR] of bleeding or starting after 8h of inf, no additional inf); score 3=moderate (probable or slight imp starting after 8h of inf, at least 1 additional inf for CR of bleeding); score 4=no imp at all, or condition worsens).	Baseline up to 6 months
Mean Number of Infusion of Study Medication	Mean frequency of BeneFIX administration of each participant was calculated from number of BeneFIX infusions which each participant received for treatment of each new bleed. Mean frequency of BeneFIX administration for total participants was summarized.	Baseline up to 6 months
Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication	Mean frequency of breakthrough (spontaneous/non-traumatic) bleeds of each participant within 48 hours of a preventive/prophylaxis dose of BeneFIX was calculated from number of irregular bleeding which occurred in each participant. Mean frequency breakthrough bleeds for total participants within 48 hours of a preventive/prophylaxis dose of BeneFIX was summarized.	Baseline up to 6 months
Average Infusion Dose of Study Medication	Average of dose per infusion per kilogram (kg) body weight was reported for prophylaxis purpose or on-demand therapy and surgery.	Baseline up to 6 months
Total Infusion of Study Medication	Total dose of study drug infused was calculated over the study duration.	Baseline up to 6 months
Percentage of Participants With Efficacy Evaluation	The efficacy of study drug was rated as 'very effective', 'effective', 'slightly ineffective' and 'ineffective'.	Baseline up to 6 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Duration of Adverse Events (AEs)	Total time from onset of adverse event till the event is resolved. Duration of AE per event = AE stop date minus AE start date plus 1.	Baseline up to 6 months
Number of Participants Who Discontinued the Study Due to Adverse Events (AEs)	AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Number of participants who discontinued the study due to AEs was reported.	Baseline up to 6 months

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: August 1, 2007
• Primary Completion (Actual): June 1, 2012
• Study Completion (Actual): June 1, 2012

Study Registration Dates

• First Submitted: June 7, 2007
• First Submitted That Met QC Criteria: June 7, 2007
• First Posted (Estimate): June 8, 2007

Study Record Updates

• Last Update Posted (Estimate): August 12, 2013
• Last Update Submitted That Met QC Criteria: May 15, 2013
• Last Verified: May 1, 2013

More Information

Terms related to this study

• Keywords: Safety; Hemophilia B; Efficacy; BeneFIX
• 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 A; Hemophilia B
• Other Study ID Numbers: 3090X1-4403; B1821005