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| ID | Type | Description | Link |
|---|---|---|---|
| B1821005 |
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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:
1) Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses.
3) Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug
The patients who meet the inclusion criteria will be enrolled consecutively.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BeneFIX (coagulation factor IX (recombinant)) | Drug | BeneFIX will be administered according to physician's discretion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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). | 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. | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline up to 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline up to 6 months |
| Number of Participants Who Discontinued the Study Due to Adverse Events (AEs) |
Inclusion Criteria:
Exclusion Criteria:
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Hemophilia B (congenital factor IX deficiency or Christmas disease).
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pfizer Investigational Site | Seoul | 137-882 | South Korea |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | BeneFIX | Participants who received original or reformulated BeneFIX (recombinant coagulation factor IX) infusion intravenously as indicated according to the approved local product document, dosage solely adjusted as per physician's discretion, were observed for a period of 6 months. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | BeneFIX | Participants who received original or reformulated BeneFIX (recombinant coagulation factor IX) infusion intravenously as indicated according to the approved local product document, dosage solely adjusted as per physician's discretion, were observed for a period of 6 months. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Safety analysis set included all participants who received at least 1 dose of study medication including dropouts due to AEs. | Posted | Number | participants | Baseline up to 6 months |
|
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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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | BeneFIX | Participants who received original or reformulated BeneFIX (recombinant coagulation factor IX) infusion intravenously as indicated according to the approved local product document, dosage solely adjusted as per physician's discretion, were observed for a period of 6 months. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Drug ineffective | General disorders | MedDRA v15.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Stomatitis | Gastrointestinal disorders | MedDRA v15.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D005164 | Factor IX |
| ID | Term |
|---|---|
| D004792 | Enzyme Precursors |
| D045762 | Enzymes and Coenzymes |
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
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| 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'. | 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). | 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. | 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). | 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. | 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. | 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. | Baseline up to 6 months |
| Total Infusion of Study Medication | Total dose of study drug infused was calculated over the study duration. | 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'. | Baseline up to 6 months |
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. |
| Baseline up to 6 months |
| Death |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
|
|
| Primary | 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). | Safety analysis set included all participants who received at least 1 dose of study medication including dropouts due to AEs. Same participant may be represented in more than 1 category. | Posted | Number | participants | Baseline up to 6 months |
|
|
|
| Primary | 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. | Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis. | Posted | Baseline up to 6 months |
|
|
| Primary | 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. | Safety analysis set included all participants who received at least 1 dose of study medication including dropouts due to AEs. | Posted | Number | participants | Baseline up to 6 months |
|
|
|
| Primary | 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'. | Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis. | Posted | Baseline up to 6 months |
|
|
| Primary | 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). | Safety analysis set included all participants who received at least 1 dose of study medication including dropouts due to AEs. | Posted | Number | participants | Baseline up to 6 months |
|
|
|
| Primary | 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. | Safety analysis set included all participants who received at least 1 dose of study medication including dropouts due to AEs. | Posted | Number | participants | Baseline up to 6 months |
|
|
|
| Secondary | 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. | Efficacy analysis set included all participants who received at least 1 dose of study medication for the approved indications and were evaluated upon its related parameters at least once. Here 'N' (number of participants analyzed) signifies participants who were evaluable for this measure. | Posted | Mean | Standard Deviation | bleeds per year | Baseline up to 6 months |
|
|
|
| Secondary | 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). | Efficacy analysis set included all participants who received at least 1 dose of study medication for the approved indications and were evaluated upon its related parameters at least once. Here 'N' (number of participants analyzed) signifies participants who were evaluable for this measure. | Posted | Number | responses | Baseline up to 6 months |
|
|
|
| Secondary | 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. | Efficacy analysis set included all participants who received at least 1 dose of study medication for the approved indications and were evaluated upon its related parameters at least once. Here 'N' (number of participants analyzed) signifies participants who were evaluable for this measure. | Posted | Mean | Standard Deviation | infusions | Baseline up to 6 months |
|
|
|
| Secondary | 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. | Efficacy analysis set included all participants who received at least 1 dose of study medication for the approved indications and were evaluated upon its related parameters at least once. Here 'N' (number of participants analyzed) signifies participants who were evaluable for this measure. | Posted | Mean | Standard Deviation | breakthrough bleeds | Baseline up to 6 months |
|
|
|
| Secondary | 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. | Efficacy analysis set included all participants who received at least 1 dose of study medication for the approved indications and were evaluated upon its related parameters at least once. Here 'N' (number of participants analyzed)= participants evaluable for this measure and 'n' = participants evaluable for the specified category. | Posted | Mean | Standard Deviation | international unit/kilogram (IU/kg) | Baseline up to 6 months |
|
|
|
| Secondary | Total Infusion of Study Medication | Total dose of study drug infused was calculated over the study duration. | Efficacy analysis set included all participants who received at least 1 dose of study medication for the approved indications and were evaluated upon its related parameters at least once. | Posted | Mean | Standard Deviation | IU | Baseline up to 6 months |
|
|
|
| Secondary | Percentage of Participants With Efficacy Evaluation | The efficacy of study drug was rated as 'very effective', 'effective', 'slightly ineffective' and 'ineffective'. | Efficacy analysis set included all participants who received at least 1 dose of study medication for the approved indications and were evaluated upon its related parameters at least once. Here 'N' (number of participants analyzed) signifies participants who were evaluable for this measure. | Posted | Number | percentage of participants | Baseline up to 6 months |
|
|
|
| Other Pre-specified | 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. | Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis. | Posted | Baseline up to 6 months |
|
|
| Other Pre-specified | 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. | Safety analysis set included all participants who received at least 1 dose of study medication including dropouts due to AEs. | Posted | Number | participants | Baseline up to 6 months |
|
|
|
| 4 |
| 178 |
| 5 |
| 178 |
| Haemophilic arthropathy | Musculoskeletal and connective tissue disorders | MedDRA v15.0 | Non-systematic Assessment |
|
| Gastrointestinal neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v15.0 | Non-systematic Assessment |
|
| Cerebral haemorrhage | Nervous system disorders | MedDRA v15.0 | Non-systematic Assessment |
|
| Cellulitis | Infections and infestations | MedDRA v15.0 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA v15.0 | Non-systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA v15.0 | Non-systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA v15.0 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v15.0 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA v15.0 | Non-systematic Assessment |
|
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.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D011506 |
| Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
| Title | Measurements |
|---|
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| Title | Measurements |
|---|
|
| No response |
|
| Title | Measurements |
|---|---|
|
| Ineffective |
|