An observational study in Hemophilia B, sponsored by Pfizer. Completed at 1 site in Korea, Republic of. Per ClinicalTrials.gov, last updated 2013-08-12.
Sponsored by Pfizer · Observational
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including:
The patients who meet the inclusion criteria will be enrolled consecutively.
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 183 is above the median of 80 across 314 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.
Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Hemophilia B (congenital factor IX deficiency or Christmas disease).
Exclusion Criteria:
Drug: BeneFIX (coagulation factor IX (recombinant))
BeneFIX will be administered according to physician's discretion.
Number of Participants With Adverse Events (AEs) According to Baseline Characteristics
AE: any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. AE assessed by baseline characteristics (chr) included age, gender, pediatric/geriatric status, liver disorder, BeneFIX treatment (previously/newly), factor nine (FIX) gene mutation, prior exposure to plasma-derived FIX products, prior FIX regimen(s) utilized, personal history of FIX inhibitor, family history of hemophilia B, severity of bleeding, medical history, concomitant medication and therapy.
Time frame: Baseline up to 6 months
Number of Participants With Adverse Events (AEs) According to Severity
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AE was assessed according to severity; mild (not causing any significant problem, dose adjustment not required), moderate (caused problem that does not interfere significantly with usual activities or the clinical status, dose adjustment needed due to adverse event) and severe (caused problem that interferes significantly with usual activities or the clinical status, study drug stopped due to adverse event).
Time frame: Baseline up to 6 months
Number of Participants With Action Taken in Response to Adverse Events (AEs)
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. After an onset of an AE, relevant actions were undertaken on the study drug or the participant. Actions related to study drug included: dosage reduced, dosage increased, stopped temporarily or permanently, no action taken; actions related to participants included: withdrawal from the study, concomitant medication, no action taken or any other as per physician's discretion.
Time frame: Baseline up to 6 months
Number of Participants With Adverse Events (AEs) According to Seriousness
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Seriousness of an AE was assessed under the criteria of serious adverse event (SAE). An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
Time frame: Baseline up to 6 months
Number of Participants With Outcome in Response to Adverse Events (AEs)
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Outcome of an AE was assessed based on response to a question 'Is the adverse event still present?' as 'yes', 'unknown' or 'no-resolved'.
Time frame: Baseline up to 6 months
Number of Participants With Adverse Events (AEs) by Relationship
AE: untoward medical occurrence in participant who received study drug without regard to causal relationship. All causalities and drug-related AEs reported. Drug-related AEs based on physician's discretion: certain (AE after drug intake, not explained by other drugs, reaction on drug cessation \[DC\], relapse on re-intake of drug), probable/likely (AE after drug intake, not explained by other drugs, reaction on DC, no information on re-intake), possible (AE after drug intake, explained by other drugs, no information on DC), unlikely (not related to drug intake time, explained by other drugs).
Time frame: Baseline up to 6 months
Number of Participants With Unexpected Adverse Events (AEs)
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Unexpected AEs were those that were not included in precaution of local product document.
Time frame: Baseline up to 6 months
Mean Annualized Bleeding Rate (ABR)
An annualized bleeding rate (ABR) was calculated as the number of bleeds requiring administration of BeneFIX (for on-demand therapy and surgery), divided by total period of bleeding multiplied by 365.25. Total period of bleeding is the number of days on treatment for prophylaxis purpose and on-demand therapy and surgery.
Time frame: Baseline up to 6 months
Number of Responses to On-demand Treatment With Study Medication
Responses to on-demand treatment were rated by participant/caregiver or physician each time the drug was administered, on 4-point scale. Score 1=excellent (definite pain relief \[PR\] and improvement \[imp\] within 8 hours \[h\] of infusion \[inf\], no additional inf); score 2=good (definite PR and imp within 8h of inf, at least 1 additional inf for complete resolution \[CR\] of bleeding or starting after 8h of inf, no additional inf); score 3=moderate (probable or slight imp starting after 8h of inf, at least 1 additional inf for CR of bleeding); score 4=no imp at all, or condition worsens).
Time frame: Baseline up to 6 months
Mean Number of Infusion of Study Medication
Mean frequency of BeneFIX administration of each participant was calculated from number of BeneFIX infusions which each participant received for treatment of each new bleed. Mean frequency of BeneFIX administration for total participants was summarized.
Time frame: Baseline up to 6 months
Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication
Mean frequency of breakthrough (spontaneous/non-traumatic) bleeds of each participant within 48 hours of a preventive/prophylaxis dose of BeneFIX was calculated from number of irregular bleeding which occurred in each participant. Mean frequency breakthrough bleeds for total participants within 48 hours of a preventive/prophylaxis dose of BeneFIX was summarized.
Time frame: Baseline up to 6 months
Average Infusion Dose of Study Medication
Average of dose per infusion per kilogram (kg) body weight was reported for prophylaxis purpose or on-demand therapy and surgery.
Time frame: Baseline up to 6 months
Total Infusion of Study Medication
Total dose of study drug infused was calculated over the study duration.
Time frame: Baseline up to 6 months
Percentage of Participants With Efficacy Evaluation
The efficacy of study drug was rated as 'very effective', 'effective', 'slightly ineffective' and 'ineffective'.
Time frame: Baseline up to 6 months
Duration of Adverse Events (AEs)
Total time from onset of adverse event till the event is resolved. Duration of AE per event = AE stop date minus AE start date plus 1.
Time frame: Baseline up to 6 months
Number of Participants Who Discontinued the Study Due to Adverse Events (AEs)
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Number of participants who discontinued the study due to AEs was reported.
Time frame: Baseline up to 6 months
| Milestone | BeneFIX |
|---|---|
| Started | 183 |
| Treated | 178 |
| Completed | 112 |
| Not completed | 71 |
| Withdrew: Lost to follow-up | 44 |
| Withdrew: Other | 20 |
| Withdrew: Randomized but not treated | 5 |
| Withdrew: Death | 2 |
AE: any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. AE assessed by baseline characteristics (chr) included age, gender, pediatric/geriatric status, liver disorder, BeneFIX treatment (previously/newly), factor nine (FIX) gene mutation, prior exposure to plasma-derived FIX products, prior FIX regimen(s) utilized, personal history of FIX inhibitor, family history of hemophilia B, severity of bleeding, medical history, concomitant medication and therapy.
| participants | BeneFIX |
|---|---|
| Age: Less than (<) 10 years | 0 |
| Age: 10 to 19 years | 1 |
| Age: 20 to 29 years | 2 |
| Age: 30 to 39 years | 3 |
| Age: 40 to 49 years | 1 |
| Age: Greater than or equal (>=) to 50 years | 2 |
| Gender: Male | 9 |
| Gender: Female | 0 |
| Pediatric status: < 12 years | 0 |
| Pediatric status: >= 12 years | 9 |
| Geriatric status: < 65 years | 8 |
| Geriatric status: >= 65 years | 1 |
| Liver disorder: Yes | 2 |
| Liver disorder: No | 7 |
| BeneFIX treatment: New | 0 |
| BeneFIX treatment: Previous | 9 |
| Factor IX gene mutation: Yes | 0 |
| Factor IX gene mutation: No | 1 |
| Factor IX gene mutation: Unknown | 8 |
| Exposed to plasma-derived FIX products: Yes | 5 |
| Exposed to plasma-derived FIX products: No | 1 |
| Exposed to plasma-derived FIX products: Unknown | 3 |
| Prior FIX regimen: Yes | 8 |
| Prior FIX regimen: No | 1 |
| Personal history of FIX inhibitor: Yes | 1 |
| Personal history of FIX inhibitor: No | 8 |
| Family history of hemophilia B: Brother | 1 |
| Family history of hemophilia B: Other | 1 |
| Family history of hemophilia B: None | 6 |
| Family history of hemophilia B: Unknown | 1 |
| Severity: Mild | 0 |
| Severity: Moderate | 4 |
| Severity: Severe | 4 |
| Severity: Unknown | 1 |
| Medical history: Yes | 6 |
| Medical history: No | 3 |
| Concomitant medication: Yes | 5 |
| Concomitant medication: No | 4 |
| Concomitant therapy: Yes | 2 |
| Concomitant therapy: No | 7 |
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AE was assessed according to severity; mild (not causing any significant problem, dose adjustment not required), moderate (caused problem that does not interfere significantly with usual activities or the clinical status, dose adjustment needed due to adverse event) and severe (caused problem that interferes significantly with usual activities or the clinical status, study drug stopped due to adverse event).
| participants | BeneFIX |
|---|---|
| Mild | 6 |
| Moderate | 2 |
| Severe | 4 |
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. After an onset of an AE, relevant actions were undertaken on the study drug or the participant. Actions related to study drug included: dosage reduced, dosage increased, stopped temporarily or permanently, no action taken; actions related to participants included: withdrawal from the study, concomitant medication, no action taken or any other as per physician's discretion.
No measurements were reported for this outcome.
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Seriousness of an AE was assessed under the criteria of serious adverse event (SAE). An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
| participants | BeneFIX |
|---|---|
| SAEs | 4 |
| Non-SAEs | 5 |
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Outcome of an AE was assessed based on response to a question 'Is the adverse event still present?' as 'yes', 'unknown' or 'no-resolved'.
No measurements were reported for this outcome.
AE: untoward medical occurrence in participant who received study drug without regard to causal relationship. All causalities and drug-related AEs reported. Drug-related AEs based on physician's discretion: certain (AE after drug intake, not explained by other drugs, reaction on drug cessation \[DC\], relapse on re-intake of drug), probable/likely (AE after drug intake, not explained by other drugs, reaction on DC, no information on re-intake), possible (AE after drug intake, explained by other drugs, no information on DC), unlikely (not related to drug intake time, explained by other drugs).
| participants | BeneFIX |
|---|---|
| AEs (all-causalities) | 9 |
| AEs (drug-related) | 1 |
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Unexpected AEs were those that were not included in precaution of local product document.
| participants | BeneFIX |
|---|---|
| Number of Participants With Unexpected Adverse Events (AEs) | 0 |
An annualized bleeding rate (ABR) was calculated as the number of bleeds requiring administration of BeneFIX (for on-demand therapy and surgery), divided by total period of bleeding multiplied by 365.25. Total period of bleeding is the number of days on treatment for prophylaxis purpose and on-demand therapy and surgery.
| bleeds per year | BeneFIX |
|---|---|
| Mean Annualized Bleeding Rate (ABR) | 84.25 ± 125.35 |
Responses to on-demand treatment were rated by participant/caregiver or physician each time the drug was administered, on 4-point scale. Score 1=excellent (definite pain relief \[PR\] and improvement \[imp\] within 8 hours \[h\] of infusion \[inf\], no additional inf); score 2=good (definite PR and imp within 8h of inf, at least 1 additional inf for complete resolution \[CR\] of bleeding or starting after 8h of inf, no additional inf); score 3=moderate (probable or slight imp starting after 8h of inf, at least 1 additional inf for CR of bleeding); score 4=no imp at all, or condition worsens).
| responses | BeneFIX |
|---|---|
| Excellent | 1145 |
| Good | 788 |
| Moderate | 65 |
| No response | 0 |
Mean frequency of BeneFIX administration of each participant was calculated from number of BeneFIX infusions which each participant received for treatment of each new bleed. Mean frequency of BeneFIX administration for total participants was summarized.
| infusions | BeneFIX |
|---|---|
| Mean Number of Infusion of Study Medication | 2.25 ± 4.33 |
Mean frequency of breakthrough (spontaneous/non-traumatic) bleeds of each participant within 48 hours of a preventive/prophylaxis dose of BeneFIX was calculated from number of irregular bleeding which occurred in each participant. Mean frequency breakthrough bleeds for total participants within 48 hours of a preventive/prophylaxis dose of BeneFIX was summarized.
| breakthrough bleeds | BeneFIX |
|---|---|
| Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication | 0.27 ± 0.47 |
Average of dose per infusion per kilogram (kg) body weight was reported for prophylaxis purpose or on-demand therapy and surgery.
| international unit/kilogram (IU/kg) | BeneFIX |
|---|---|
| On-demand therapy and surgery: n= 135 | 38.79 ± 5.41 |
| Prophylaxis purpose: n= 123 | 32.92 ± 10.43 |
Total dose of study drug infused was calculated over the study duration.
| IU | BeneFIX |
|---|---|
| Total Infusion of Study Medication | 50356.11 ± 42154.05 |
The efficacy of study drug was rated as 'very effective', 'effective', 'slightly ineffective' and 'ineffective'.
| percentage of participants | BeneFIX |
|---|---|
| Very effective | 81.82 |
| Effective | 18.18 |
| Slightly ineffective | 0.00 |
| Ineffective | 0.00 |
Total time from onset of adverse event till the event is resolved. Duration of AE per event = AE stop date minus AE start date plus 1.
No measurements were reported for this outcome.
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Number of participants who discontinued the study due to AEs was reported.
| participants | BeneFIX |
|---|---|
| Number of Participants Who Discontinued the Study Due to Adverse Events (AEs) | 0 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BeneFIX | — | 4/178 (2.2%) | 5/178 (2.8%) |
| Event | BeneFIX |
|---|---|
| Cerebral haemorrhageNervous system disorders | 2/178 |
| Drug ineffectiveGeneral disorders | 1/178 |
| Haemophilic arthropathyMusculoskeletal and connective tissue disorders | 1/178 |
| Gastrointestinal neoplasmNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/178 |
| Event | BeneFIX |
|---|---|
| StomatitisGastrointestinal disorders | 1/178 |
| CellulitisInfections and infestations | 1/178 |
| NasopharyngitisInfections and infestations | 1/178 |
| Alanine aminotransferase increasedInvestigations | 1/178 |
| Aspartate aminotransferase increasedInvestigations | 1/178 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/178 |
| HeadacheNervous system disorders | 1/178 |
| Age Continuous(years) | BeneFIX |
|---|---|
| Mean | 25.1 ± 16.1 |
| Sex: Female, Male(Participants) | BeneFIX |
|---|---|
| Female | 1 |
| Male | 177 |
This study is completed, as verified in May 2013. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Pfizer