Trial Outcomes & Findings for Post Marketing Observational Study of Reformulated BeneFIX (NCT NCT00835068)
NCT ID: NCT00835068
Last Updated: 2014-09-17
Results Overview
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.
Recruitment status
COMPLETED
Target enrollment
58 participants
Primary outcome timeframe
Baseline up to Year 3.5
Results posted on
2014-09-17
Participant Flow
Participant milestones
| Measure |
BeneFIX (Previously Treated Participants)
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Overall Study
STARTED
|
57
|
1
|
|
Overall Study
COMPLETED
|
47
|
0
|
|
Overall Study
NOT COMPLETED
|
10
|
1
|
Reasons for withdrawal
| Measure |
BeneFIX (Previously Treated Participants)
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Adverse Event
|
0
|
1
|
|
Overall Study
Other
|
8
|
0
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Post Marketing Observational Study of Reformulated BeneFIX
Baseline characteristics by cohort
| Measure |
BeneFIX (Previously Treated Participants)
n=57 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
n=1 Participants
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
Total
n=58 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
23.36 years
STANDARD_DEVIATION 16.237 • n=5 Participants
|
1.30 years
n=7 Participants
|
22.98 years
STANDARD_DEVIATION 16.353 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
57 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
58 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Year 3.5Population: Safety population included all participants who received at least one dose of BeneFIX.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=57 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
n=1 Participants
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) Prior to Safety Amendment
Treatment-related AE
|
1 participants
|
1 participants
|
|
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) Prior to Safety Amendment
Treatment-related SAE
|
0 participants
|
1 participants
|
PRIMARY outcome
Timeframe: Year 3.5 up to 4.75Population: Safety population included all participants who received at least one dose of BeneFIX. Previously untreated participants were not evaluable for this outcome measure due to discontinuation prior to safety amendment.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=57 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment
AE
|
26 participants
|
—
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment
SAE
|
10 participants
|
—
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment
Treatment-related AE
|
1 participants
|
—
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment
Treatment-related SAE
|
0 participants
|
—
|
PRIMARY outcome
Timeframe: Baseline up to Year 4.75Population: Safety population included all participants who received at least one dose of BeneFIX.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=57 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
n=1 Participants
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Number of Participants With Events of Special Interest
FIX inhibitor
|
0 participants
|
1 participants
|
|
Number of Participants With Events of Special Interest
Allergic reactions
|
0 participants
|
0 participants
|
|
Number of Participants With Events of Special Interest
RBC agglutination
|
0 participants
|
0 participants
|
|
Number of Participants With Events of Special Interest
Lack of efficacy/ low recovery
|
0 participants
|
0 participants
|
|
Number of Participants With Events of Special Interest
Thrombotic event
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Baseline up to Year 4.75Population: Efficacy population: participants with basal FIX activity less than or equal to (\<=) 1 percent (%) with real exposure days in diary at least 70% of planned exposure days for prophylaxis period, and without FIX inhibitor before or during study (no FIX inhibitor history at baseline; FIX inhibitor titer \<0.6 Bethesda Unit \[BU\] during follow up).
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=42 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Number of Bleeding Episodes
Prophylaxis (n=28)
|
261 bleeding episodes
|
—
|
|
Number of Bleeding Episodes
On demand (n=21)
|
695 bleeding episodes
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Year 4.75Population: Efficacy population. Efficacy population included only those participants who were previously treated with BeneFIX. Here, 'number of bleeding episodes analyzed' signifies episodes evaluable for this measure. 'n' signifies those bleeding episodes with injection data available during specified period.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=955 bleeding episodes
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Number of Bleeding Episodes Requiring Treatment by Injection
Prophylaxis: 1 Injection (n = 261)
|
144 bleeding episodes
|
—
|
|
Number of Bleeding Episodes Requiring Treatment by Injection
On demand: 1 Injection (n = 694)
|
515 bleeding episodes
|
—
|
|
Number of Bleeding Episodes Requiring Treatment by Injection
Prophylaxis: 2 Injection (n = 261)
|
63 bleeding episodes
|
—
|
|
Number of Bleeding Episodes Requiring Treatment by Injection
On demand: 2 Injection (n = 694)
|
89 bleeding episodes
|
—
|
|
Number of Bleeding Episodes Requiring Treatment by Injection
Prophylaxis: 3 Injection (n = 261)
|
21 bleeding episodes
|
—
|
|
Number of Bleeding Episodes Requiring Treatment by Injection
On demand: 3 Injection (n = 694)
|
35 bleeding episodes
|
—
|
|
Number of Bleeding Episodes Requiring Treatment by Injection
Prophylaxis: >=4 Injection (n = 261)
|
33 bleeding episodes
|
—
|
|
Number of Bleeding Episodes Requiring Treatment by Injection
On demand: >=4 Injection (n = 694)
|
55 bleeding episodes
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Year 4.75Population: Safety population included all participants who received at least one dose of BeneFIX. Participants with at least one follow-up visit were evaluable for this outcome measure.
Total consumption of BeneFIX included consumption during prophylaxis, on demand, during bleeding episodes, preventive injections, surgeries or immune tolerance.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=51 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
n=1 Participants
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Total Consumption of BeneFIX
|
434751.0 International Unit (IU)
Standard Deviation 446077.9
|
15000.0 International Unit (IU)
|
SECONDARY outcome
Timeframe: Baseline up to Year 4.75Population: Efficacy population. Efficacy population included only those participants who were previously treated with BeneFIX.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=42 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Subjective Assessment of Efficacy by Participant
Latest assessment: Missing Values
|
4 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Latest assessment: Very Good
|
19 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Latest assessment: Good
|
19 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Latest assessment: Moderate
|
0 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Latest assessment: Bad
|
0 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Worst assessment: Missing Values
|
4 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Worst assessment: Very Good
|
17 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Worst assessment: Good
|
20 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Worst assessment: Moderate
|
1 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Worst assessment: Bad
|
0 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Best assessment: Missing Values
|
4 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Best assessment: Very Good
|
25 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Best assessment: Good
|
13 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Best assessment: Moderate
|
0 participants
|
—
|
|
Subjective Assessment of Efficacy by Participant
Best assessment: Bad
|
0 participants
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Year 4.75Population: Efficacy population. 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.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=42 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Dose Per Injection of BeneFIX
Prophylaxis (n=28)
|
45.3 IU/kg
Standard Deviation 11.41
|
—
|
|
Dose Per Injection of BeneFIX
On demand (n=21)
|
49.0 IU/kg
Standard Deviation 14.25
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Year 4.75Population: Efficacy population. Efficacy population included only those participants who were previously treated with BeneFIX.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=42 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Subjective Assessment of Efficacy by Physician
Latest assessment: Very Good
|
17 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Latest assessment: Good
|
23 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Latest assessment: Moderate
|
2 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Latest assessment: Bad
|
0 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Worst assessment: Very Good
|
10 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Worst assessment: Good
|
29 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Worst assessment: Moderate
|
3 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Worst assessment: Bad
|
0 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Best assessment: Very Good
|
31 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Best assessment: Good
|
11 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Best assessment: Moderate
|
0 participants
|
—
|
|
Subjective Assessment of Efficacy by Physician
Best assessment: Bad
|
0 participants
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Year 4.75Population: Efficacy population. Efficacy population included only those participants who were previously treated with BeneFIX.
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.
Outcome measures
| Measure |
BeneFIX (Previously Treated Participants)
n=42 Participants
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Subjective Assessment of Ease of Use by Participant
Latest assessment: Missing
|
5 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Latest assessment: Very Good
|
17 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Latest assessment: Good
|
19 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Latest assessment: Moderate
|
1 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Latest assessment: Bad
|
0 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Worst assessment: Missing Value
|
5 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Worst assessment: Very Good
|
13 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Worst assessment: Good
|
22 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Worst assessment: Moderate
|
2 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Worst assessment: Bad
|
0 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Best assessment: Missing Values
|
5 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Best assessment: Very Good
|
21 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Best assessment: Good
|
16 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Best assessment: Moderate
|
0 participants
|
—
|
|
Subjective Assessment of Ease of Use by Participant
Best assessment: Bad
|
0 participants
|
—
|
Adverse Events
BeneFIX (Previously Treated Participants)
Serious events: 10 serious events
Other events: 21 other events
Deaths: 0 deaths
BeneFIX (Previously Untreated Participants)
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BeneFIX (Previously Treated Participants)
n=57 participants at risk
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
n=1 participants at risk
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Blood and lymphatic system disorders
Factor IX inhibition
|
0.00%
0/57
The same event may appear as both an AE and a SAE. 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.
|
100.0%
1/1
The same event may appear as both an AE and a SAE. 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
Back pain
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Haemarthrosis
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Haemophilic arthropathy
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Device failure
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Hyperthermia
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Ear and labyrinth disorders
Otitis media
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Ear and labyrinth disorders
Tympanic membrane perforation
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Congenital genitourinary abnormality
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Appendicitis
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
| Measure |
BeneFIX (Previously Treated Participants)
n=57 participants at risk
Participants with hemophilia B who were previously treated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection per routine clinical practice and were observed for up to 5 years.
|
BeneFIX (Previously Untreated Participants)
n=1 participants at risk
Participants with hemophilia B who were previously untreated with reformulated BeneFIX, received reformulated BeneFIX intravenous injection as per routine clinical practice and were observed for up to 5 years.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Branchial cyst
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Ear and labyrinth disorders
Ear infection
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Eye disorders
Conjunctivitis
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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 pain upper
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Vomiting
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Asthenia
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Malaise
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Mucosal dryness
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Chronic sinusitis
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Influenza
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Varicella
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Injury, poisoning and procedural complications
Wound
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Vitamin D increased
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Arthralgia
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Carpal tunnel syndrome
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Dysgeusia
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
|
5.3%
3/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Loss of consciousness
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Speech disorder
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Psychiatric disorders
Sleep disorder
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Renal and urinary disorders
Haematuria
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Reproductive system and breast disorders
Haematospermia
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Asthma exercise induced
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Bronchitis
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Lung disorder
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
Throat irritation
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Vascular disorders
Epistaxis
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Vascular disorders
Haemorrhoids
|
3.5%
2/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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.
|
|
Vascular disorders
Rectal haemorrhage
|
1.8%
1/57
The same event may appear as both an AE and a SAE. 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.
|
0.00%
0/1
The same event may appear as both an AE and a SAE. 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
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