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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00484185
First received: June 7, 2007
Last updated: May 15, 2013
Last verified: May 2013
  Purpose

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


Condition Intervention
Hemophilia B
Drug: BeneFIX (coagulation factor IX (recombinant))

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Post Marketing Surveillance To Observe Safety And Efficacy Of BeneFIX In Patients With Hemophilia B

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Number of Participants With Adverse Events (AEs) According to Baseline Characteristics [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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) [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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) [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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'.

  • Number of Participants With Adverse Events (AEs) by Relationship [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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 Unexpected Adverse Events (AEs) [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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.


Secondary Outcome Measures:
  • Mean Annualized Bleeding Rate (ABR) [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    Total dose of study drug infused was calculated over the study duration.

  • Percentage of Participants With Efficacy Evaluation [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    The efficacy of study drug was rated as 'very effective', 'effective', 'slightly ineffective' and 'ineffective'.


Other Outcome Measures:
  • Duration of Adverse Events (AEs) [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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) [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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.


Enrollment: 183
Study Start Date: August 2007
Study Completion Date: June 2012
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
1 Drug: BeneFIX (coagulation factor IX (recombinant))
BeneFIX will be administered according to physician's discretion.

Detailed Description:

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

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

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

Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00484185

Locations
Korea, Republic of
Pfizer Investigational Site
Seoul, Korea, Republic of, 137-882
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00484185     History of Changes
Other Study ID Numbers: 3090X1-4403, B1821005
Study First Received: June 7, 2007
Results First Received: May 15, 2013
Last Updated: May 15, 2013
Health Authority: Korea: Food and Drug Administration

Keywords provided by Pfizer:
Hemophilia B
BeneFIX
Safety
Efficacy

Additional relevant MeSH terms:
Hemophilia B
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on July 31, 2014