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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT00484185

Enrollment

183

Registered

2007-06-08

Start date

2007-08-31

Completion date

2012-06-30

Last updated

2013-08-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia B

Keywords

Hemophilia B, BeneFIX, Safety, Efficacy

Brief summary

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

Detailed description

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

Interventions

DRUGBeneFIX (coagulation factor IX (recombinant))

BeneFIX will be administered according to physician's discretion.

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Number of Participants With Unexpected Adverse Events (AEs)	Baseline up to 6 months	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.
Number of Participants With Adverse Events (AEs) by Relationship	Baseline up to 6 months	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).
Number of Participants With Adverse Events (AEs) According to Baseline Characteristics	Baseline up to 6 months	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.
Number of Participants With Adverse Events (AEs) According to Severity	Baseline up to 6 months	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).
Number of Participants With Action Taken in Response to Adverse Events (AEs)	Baseline up to 6 months	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.
Number of Participants With Adverse Events (AEs) According to Seriousness	Baseline up to 6 months	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.
Number of Participants With Outcome in Response to Adverse Events (AEs)	Baseline up to 6 months	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'.

Secondary

Measure	Time frame	Description
Percentage of Participants With Efficacy Evaluation	Baseline up to 6 months	The efficacy of study drug was rated as 'very effective', 'effective', 'slightly ineffective' and 'ineffective'.
Mean Annualized Bleeding Rate (ABR)	Baseline up to 6 months	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.
Number of Responses to On-demand Treatment With Study Medication	Baseline up to 6 months	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).
Mean Number of Infusion of Study Medication	Baseline up to 6 months	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.
Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication	Baseline up to 6 months	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.
Average Infusion Dose of Study Medication	Baseline up to 6 months	Average of dose per infusion per kilogram (kg) body weight was reported for prophylaxis purpose or on-demand therapy and surgery.
Total Infusion of Study Medication	Baseline up to 6 months	Total dose of study drug infused was calculated over the study duration.

Other

Measure	Time frame	Description
Duration of Adverse Events (AEs)	Baseline up to 6 months	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.
Number of Participants Who Discontinued the Study Due to Adverse Events (AEs)	Baseline up to 6 months	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.

Countries

South Korea

Participant flow

Participants by arm

Arm	Count
BeneFIX Participants who received original or reformulated BeneFIX (recombinant coagulation factor IX) infusion intravenously as indicated according to the approved local product document, dosage solely adjusted as per physician's discretion, were observed for a period of 6 months.	178
Total	178

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Death	2
Overall Study	Lost to Follow-up	44
Overall Study	Other	20
Overall Study	Randomized but not treated	5

Baseline characteristics

Characteristic	BeneFIX
Age Continuous	25.1 years STANDARD_DEVIATION 16.1
Sex: Female, Male Female	1 Participants
Sex: Female, Male Male	177 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	5 / 178
serious Total, serious adverse events	4 / 178

Outcome results

Primary

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.