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Trial record 33 of 398 for:    bleeding episodes

BeneFIX Drug Use Results Survey [All-Case Surveillance]

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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:
Information provided by (Responsible Party):
Pfizer

Study Type Observational
Study Design Observational Model: Cohort;   Time Perspective: Prospective
Condition Hemophilia B
Intervention Drug: Nonacog Alfa (Genetical Recombination)
Enrollment 314
Recruitment Details  
Pre-assignment Details  
Arm/Group Title BeneFIX (Nonacog Alfa)
Hide Arm/Group Description Participants, who received BeneFIX injection intravenously as indicated in the approved local product document, were observed for periods of 1 year for PTPs and 2 years for PUPs. The dosage can be adjusted as per physician’s discretion.
Period Title: Overall Study
Started 314
Completed 312
Not Completed 2
Reason Not Completed
Safety Unassessable, No Drug Admin.             1
No Info. After 1st Day, No Drug Admin.             1
Arm/Group Title BeneFIX (Nonacog Alfa)
Hide Arm/Group Description Participants, who received BeneFIX injection intravenously as indicated in the approved local product document, were observed for periods of 1 year for PTPs and 2 years for PUPs. The dosage can be adjusted as per physician’s discretion.
Overall Number of Baseline Participants 312
Hide Baseline Analysis Population Description
A total of 314 participants completed the study. Of the 314 participants, 2 participants were excluded from the baseline analysis. One was excluded due to no information after first day of treatment and no drug administration, the other due to safety not assessable and no drug administration.
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 312 participants
<15 years 124
≥15 years <65 years 179
≥65 years 8
Unknown 1
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 312 participants
Female
4
   1.3%
Male
308
  98.7%
Previously Treated History   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 312 participants
PTPs 281
PUPs 28
Others 3
[1]
Measure Description: PTPs refer to previously treated patients. PUPs refer to previously untreated patients.
Severity   [1] 
Measure Type: Number
Unit of measure:  Participans
Number Analyzed 312 participants
Severe 156
Moderate 98
Mild 43
Unknown 15
[1]
Measure Description: The severity of heamophilia B was defined as follows; severe, blood coagulation factor IX activity less than 1%; moderate, 1% to 5%; mild, more than 1%.
Number of Bleeding in the Past 1 Year  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 312 participants
˂10 Times 194
≥10 to ˂50 Times 73
≥50 Times 8
Unknown 37
History of Inhibitor  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 312 participants
Yes 6
No 306
Hemophilic Arthropathy  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 312 participants
Yes 133
No 179
1.Primary Outcome
Title Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy
Hide Description 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.
Time Frame 2 years for PTPs, 1 year for PUPs
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.
Arm/Group Title BeneFIX (Nonacog Alfa)
Hide Arm/Group Description:
Participants, who received BeneFIX injection intravenously as indicated in the approved local product document, were observed for periods of 1 year for PTPs and 2 years for PUPs. The dosage can be adjusted as per physician’s discretion.
Overall Number of Participants Analyzed 178
Median (Inter-Quartile Range)
Unit of Measure: Bleeding events/year
2.00
(0.00 to 5.99)
2.Primary Outcome
Title Number of Administrations Required for Hemostasis for Bleeding Events
Hide Description Mean number of administrations for hemostasis in replacement therapy for bleeding events.
Time Frame 2 years for PTPs, 1 year for PUPs
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.
Arm/Group Title BeneFIX (Nonacog Alfa)
Hide Arm/Group Description:
Participants, who received BeneFIX injection intravenously as indicated in the approved local product document, were observed for periods of 1 year for PTPs and 2 years for PUPs. The dosage can be adjusted as per physician’s discretion.
Overall Number of Participants Analyzed 112
Overall Number of Units Analyzed
Type of Units Analyzed: Dosing for hemostasis
1161
Mean (Full Range)
Unit of Measure: administrations
1.8
(0.31 to 33)
3.Secondary Outcome
Title Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes
Hide Description Propotion of subjects whose physicians evaluated the effect of BeneFIX for bleeding episodes as excellent or good was calculated.
Time Frame 2 years for PTPs, 1 year for PUPs
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.
Arm/Group Title BeneFIX (Nonacog Alfa)
Hide Arm/Group Description:
Participants, who received BeneFIX injection intravenously as indicated in the approved local product document, were observed for periods of 1 year for PTPs and 2 years for PUPs. The dosage can be adjusted as per physician’s discretion.
Overall Number of Participants Analyzed 112
Overall Number of Units Analyzed
Type of Units Analyzed: Evaluations by physicians
671
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage
93.7
(91.6 to 95.5)
Time Frame [Not Specified]
Adverse Event Reporting Description The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
 
Arm/Group Title BeneFIX (Nonacog Alfa)
Hide Arm/Group Description Participants, who received BeneFIX injection intravenously as indicated in the approved local product document, were observed for periods of 1 year for PTPs and 2 years for PUPs. The dosage can be adjusted as per physician’s discretion.
All-Cause Mortality
BeneFIX (Nonacog Alfa)
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
BeneFIX (Nonacog Alfa)
Affected / at Risk (%)
Total   5/312 (1.60%) 
Congenital, familial and genetic disorders   
Pulmonary artery atresia * 1  1/312 (0.32%) 
Gastrointestinal disorders   
Duodenal ulcer * 1  1/312 (0.32%) 
General disorders   
Gait disturbance * 1  1/312 (0.32%) 
Infections and infestations   
Herpes zoster * 1  1/312 (0.32%) 
Investigations   
Anti factor IX antibody positive * 1  1/312 (0.32%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 17.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
BeneFIX (Nonacog Alfa)
Affected / at Risk (%)
Total   35/312 (11.22%) 
Blood and lymphatic system disorders   
Iron deficiency anaemia * 1  2/312 (0.64%) 
Gastrointestinal disorders   
Diarrhoea * 1  2/312 (0.64%) 
Abdominal discomfort * 1  1/312 (0.32%) 
Nausea * 1  1/312 (0.32%) 
Stomatitis * 1  1/312 (0.32%) 
General disorders   
Injection site pruritus * 1  1/312 (0.32%) 
Pyrexia * 1  1/312 (0.32%) 
Infections and infestations   
Nasopharyngitis * 1  2/312 (0.64%) 
Urinary tract infection * 1  2/312 (0.64%) 
Adenovirus infection * 1  1/312 (0.32%) 
Bronchitis * 1  1/312 (0.32%) 
Bronchopneumonia * 1  1/312 (0.32%) 
Gastroenteritis * 1  1/312 (0.32%) 
Pneumonia * 1  1/312 (0.32%) 
Rhinitis * 1  1/312 (0.32%) 
Investigations   
CD4 lymphocytes decreased * 1  1/312 (0.32%) 
Musculoskeletal and connective tissue disorders   
Muscle haemorrhage * 1  1/312 (0.32%) 
Nervous system disorders   
Dizziness * 1  2/312 (0.64%) 
Headache * 1  2/312 (0.64%) 
Epilepsy * 1  1/312 (0.32%) 
Respiratory, thoracic and mediastinal disorders   
Upper respiratory tract inflammation * 1  2/312 (0.64%) 
Cough * 1  1/312 (0.32%) 
Dyspnoea * 1  1/312 (0.32%) 
Epistaxis * 1  1/312 (0.32%) 
Skin and subcutaneous tissue disorders   
Rash * 1  3/312 (0.96%) 
Urticaria * 1  1/312 (0.32%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 17.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01154231     History of Changes
Other Study ID Numbers: 3090X1-4415
B1821004
B1821009 ( Other Identifier: Alias Study Number )
First Submitted: June 28, 2010
First Posted: June 30, 2010
Results First Submitted: February 28, 2017
Results First Posted: April 11, 2017
Last Update Posted: October 17, 2018