Trial Outcomes & Findings for BeneFIX Drug Use Results Survey [All-Case Surveillance] (NCT NCT01154231)

Last Updated: 2025-07-16

Results Overview

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.

Recruitment status

COMPLETED

Target enrollment

314 participants

Primary outcome timeframe

2 years for PTPs, 1 year for PUPs

Results posted on

2025-07-16

Participant Flow

Participant milestones

Participant milestones
Measure	BeneFIX (Nonacog Alfa) 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 Study STARTED	314
Overall Study COMPLETED	312
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	BeneFIX (Nonacog Alfa) 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 Study Safety Unassessable, No Drug Admin.	1
Overall Study No Info. After 1st Day, No Drug Admin.	1

Baseline Characteristics

BeneFIX Drug Use Results Survey [All-Case Surveillance]

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BeneFIX (Nonacog Alfa) n=312 Participants 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.
Age, Customized <15 years	124 Participants n=5 Participants
Age, Customized ≥15 years <65 years	179 Participants n=5 Participants
Age, Customized ≥65 years	8 Participants n=5 Participants
Age, Customized Unknown	1 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	308 Participants n=5 Participants
Previously Treated History PTPs	281 Participants n=5 Participants
Previously Treated History PUPs	28 Participants n=5 Participants
Previously Treated History Others	3 Participants n=5 Participants
Severity Severe	156 Participans n=5 Participants
Severity Moderate	98 Participans n=5 Participants
Severity Mild	43 Participans n=5 Participants
Severity Unknown	15 Participans n=5 Participants
Number of Bleeding in the Past 1 Year ˂10 Times	194 Participants n=5 Participants
Number of Bleeding in the Past 1 Year ≥10 to ˂50 Times	73 Participants n=5 Participants
Number of Bleeding in the Past 1 Year ≥50 Times	8 Participants n=5 Participants
Number of Bleeding in the Past 1 Year Unknown	37 Participants n=5 Participants
History of Inhibitor Yes	6 Participants n=5 Participants
History of Inhibitor No	306 Participants n=5 Participants
Hemophilic Arthropathy Yes	133 Participants n=5 Participants
Hemophilic Arthropathy No	179 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years for PTPs, 1 year for PUPs

Population: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.

Outcome measures

Outcome measures
Measure	BeneFIX (Nonacog Alfa) n=178 Participants 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.
Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy	2.00 Bleeding events/year Inter-Quartile Range 11.29 • Interval 0.0 to 5.99

PRIMARY outcome

Timeframe: 2 years for PTPs, 1 year for PUPs

Population: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.

Mean number of administrations for hemostasis in replacement therapy for bleeding events.

Outcome measures

Outcome measures
Measure	BeneFIX (Nonacog Alfa) n=1161 dosing for hemostasis 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.
Number of Administrations Required for Hemostasis for Bleeding Events	1.8 administrations Full Range 4.18 • Interval 0.31 to 33.0

SECONDARY outcome

Timeframe: 2 years for PTPs, 1 year for PUPs

Population: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.

Propotion of subjects whose physicians evaluated the effect of BeneFIX for bleeding episodes as excellent or good was calculated.

Outcome measures

Outcome measures
Measure	BeneFIX (Nonacog Alfa) n=671 evaluations by physicians 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.
Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes	93.7 percentage Interval 91.6 to 95.5

Adverse Events

BeneFIX (Nonacog Alfa)

Serious events: 5 serious events

Other events: 35 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	BeneFIX (Nonacog Alfa) n=312 participants at risk 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.
Infections and infestations Herpes zoster	0.32% 1/312 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.
Gastrointestinal disorders Duodenal ulcer	0.32% 1/312 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.
Congenital, familial and genetic disorders Pulmonary artery atresia	0.32% 1/312 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.
General disorders Gait disturbance	0.32% 1/312 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.
Investigations Anti factor IX antibody positive	0.32% 1/312 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.

Other adverse events

Other adverse events
Measure	BeneFIX (Nonacog Alfa) n=312 participants at risk 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.
Infections and infestations Nasopharyngitis	0.64% 2/312 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.
Infections and infestations Urinary tract infection	0.64% 2/312 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.
Blood and lymphatic system disorders Iron deficiency anaemia	0.64% 2/312 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.
Nervous system disorders Dizziness	0.64% 2/312 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.
Nervous system disorders Headache	0.64% 2/312 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.
Respiratory, thoracic and mediastinal disorders Upper respiratory tract inflammation	0.64% 2/312 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.
Gastrointestinal disorders Diarrhoea	0.64% 2/312 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.
Skin and subcutaneous tissue disorders Rash	0.96% 3/312 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.
Infections and infestations Adenovirus infection	0.32% 1/312 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.
Infections and infestations Bronchitis	0.32% 1/312 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.
Infections and infestations Bronchopneumonia	0.32% 1/312 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.
Infections and infestations Gastroenteritis	0.32% 1/312 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.
Infections and infestations Pneumonia	0.32% 1/312 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.
Infections and infestations Rhinitis	0.32% 1/312 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.
Nervous system disorders Epilepsy	0.32% 1/312 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.
Respiratory, thoracic and mediastinal disorders Cough	0.32% 1/312 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.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.32% 1/312 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.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.32% 1/312 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.
Gastrointestinal disorders Abdominal discomfort	0.32% 1/312 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.
Gastrointestinal disorders Nausea	0.32% 1/312 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.
Gastrointestinal disorders Stomatitis	0.32% 1/312 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.
Skin and subcutaneous tissue disorders Urticaria	0.32% 1/312 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.
Musculoskeletal and connective tissue disorders Muscle haemorrhage	0.32% 1/312 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.
General disorders Injection site pruritus	0.32% 1/312 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.
General disorders Pyrexia	0.32% 1/312 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.
Investigations CD4 lymphocytes decreased	0.32% 1/312 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.

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer, Inc.

Phone: 1-800-718-1021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER