Post Marketing Observational Study of Reformulated BeneFIX

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00835068
First received: February 2, 2009
Last updated: September 11, 2014
Last verified: September 2014
  Purpose

The primary objective of this observational study is to collect safety data on reformulated BeneFIX as prescribed in routine clinical practice conditions in France. The secondary objectives are to collect data on the clinical course of individuals treated with reformulated BeneFIX and on the ease of reformulated BeneFIX.


Condition Intervention
Hemophilia B
Other: No intervention

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Post Marketing Observational Study Of Reformulated BeneFIX

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) Prior to Safety Amendment [ Time Frame: Baseline up to Year 3.5 ] [ Designated as safety issue: Yes ]
    A treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. 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. AEs included SAEs as well as non-serious AEs which occurred prior to safety amendment. Prior to safety amendment, only AEs/SAEs deemed related to BeneFIX as per participating physician were collected.

  • Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment [ Time Frame: Year 3.5 up to 4.75 ] [ 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. 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. A treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug as per participating physician. AEs included SAEs as well as non-serious AEs which occurred after the safety amendment. After the safety amendment, all AEs/SAEs were collected irrespective of their relationship to BeneFIX.

  • Number of Participants With Events of Special Interest [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: Yes ]
    Events of special interest included allergic reactions, red blood cell (RBC) agglutination phenomena, lack of efficacy/low recovery, thrombotic events and onset of factor IX (FIX) inhibitor. Participants may be represented in more than 1 category.


Secondary Outcome Measures:
  • Number of Bleeding Episodes [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: No ]
    Number of bleeding episode during prophylaxis and on demand period were reported. All periods with at least one injection per week were considered as prophylaxis period. All prophylaxis periods of less than a month were reviewed and cross-checked with the treatment scheme planned at the previous visit to confirm if they were real prophylaxis periods or preventive injections periods. On demand treatment period included the total duration of follow up excluding duration of both prophylaxis and preventive injection treatment scheme. Efficacy population included only those participants who were previously treated with BeneFIX. Here, 'n' signifies participants evaluable for this measure during the specified treatment period. Participants may be represented in more than 1 category.

  • Number of Bleeding Episodes Requiring Treatment by Injection [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: No ]
    Number of injections (1, 2, 3 or greater than or equal to [>= 4]) required to treat the bleeding episodes during prophylaxis and on demand period were reported. All periods with at least one injection per week were considered as prophylaxis period. All prophylaxis periods of less than a month were reviewed and cross-checked with the treatment scheme planned at the previous visit to confirm if they were real prophylaxis periods or preventive injections periods. On demand treatment period included the total duration of follow up excluding duration of both prophylaxis and preventive injection treatment scheme.

  • Total Consumption of BeneFIX [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: No ]
    Total consumption of BeneFIX included consumption during prophylaxis, on demand, during bleeding episodes, preventive injections, surgeries or immune tolerance.

  • Subjective Assessment of Efficacy by Participant [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: No ]
    Participants assessed efficacy of BeneFIX as very good, good, moderate and bad at each follow-up visit. Results were summarized for the latest (most recent), worst and best assessment of BeneFIX done by the participant.

  • Dose Per Injection of BeneFIX [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: No ]
    Dose per injection during prophylaxis and on demand period were reported. All periods with at least one injection per week were considered as prophylaxis period. All prophylaxis periods of less than a month were reviewed and cross-checked with the treatment scheme planned at the previous visit to confirm if they were real prophylaxis periods or preventive injections periods. On demand treatment period included the total duration of follow up excluding duration of both prophylaxis and preventive injection treatment scheme. Participants may be represented in more than 1 category.

  • Subjective Assessment of Efficacy by Physician [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: No ]
    Participating physician assessed efficacy of BeneFIX as very good, good, moderate and bad at follow-up visit. Results were summarized for the latest (most recent), worst and best assessment of BeneFIX done by the physician.

  • Subjective Assessment of Ease of Use by Participant [ Time Frame: Baseline up to Year 4.75 ] [ Designated as safety issue: No ]
    Participants assessed ease of use of BeneFIX as very good, good, moderate and bad at each follow-up visit. Results were summarized for the latest (most recent), worst and best assessment of BeneFIX done by the participant.


Enrollment: 58
Study Start Date: January 2009
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
BeneFIX Other: No intervention
As it is a non interventional study, patient receives his usual treatment (BeneFIX) as determined by the physician

Detailed Description:

No sampling

  Eligibility

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

Hemophilia B patients already receiving or starting treatment with reformulated BeneFIX

Criteria

Inclusion Criteria:

  • Subjects with Hemophilia B already receiving or starting treatment with reformulated BeneFIX.
  • Subjects who have dated and signed the informed consent form.

Exclusion Criteria:

  • Ongoing treatment of Hemophilia B by a product other than reformulated BeneFIX.
  • Participation in the European prospective registry of patients with Hemophilia B treated with BeneFIX (Wyeth protocol 3090A-101039).
  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: NCT00835068

Locations
France
Pfizer Investigational Site
Chamberry, Cedex, France, 73011
Pfizer Investigational Site
Le Chesnay, Cedex, France, 78157
Pfizer Investigational Site
Caen Cedex 9, France, 14033
Pfizer Investigational Site
Clermont Ferrand, France, 63003
Pfizer Investigational Site
Dijon Cedex, France, 21070
Pfizer Investigational Site
leKremlin-Bicetre, France, 94275
Pfizer Investigational Site
Lyon, France, 69003
Pfizer Investigational Site
Marseille Cedex 05, France, 13385
Pfizer Investigational Site
Montmorency, France, 95160
Pfizer Investigational Site
Montpellier Cedex 5, France, 34 34295
Pfizer Investigational Site
Nantes cedex 1, France, 44093
Pfizer Investigational Site
Paris, France, F-75015
Pfizer Investigational Site
Paris, France, 75014
Pfizer Investigational Site
Rouen, France, 76031
Pfizer Investigational Site
Saint Priest en Jarez, France, 42277
Pfizer Investigational Site
Tours, France, 37044
Pfizer Investigational Site
Vandoeuvre Les Nancy Cedex, France, 54511
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: NCT00835068     History of Changes
Other Study ID Numbers: 3090X1-4409, B1821007
Study First Received: February 2, 2009
Results First Received: September 11, 2014
Last Updated: September 11, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
Hemophilia B
observational
post marketing study

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

ClinicalTrials.gov processed this record on September 18, 2014