Hemophilia BNCT00484185
Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B
An observational study of Hemophilia B, run by Pfizer at 1 site in South Korea.
Trial summary
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia B
- What is being tested?
- BeneFIX (coagulation factor IX (recombinant))
- How many people
- 183 participants
- Where
- 1 siteSouth Korea
- Study dates
- Aug 2007to Jun 2012
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Hemophilia B (congenital factor IX deficiency or Christmas disease).
Who may be able to join
- Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement).
- Pediatric and adult patients who have been treated with original or reformulated BeneFIX for hemophilia B (congenital factor IX deficiency or Christmas disease) from first approved date by KFDA, or who are planned to be newly prescribed BeneFIX (for example, patients switching from pdFIX to BeneFIX).
Who cannot join
- Patients with a known history of hypersensitivity to original or reformulated BeneFIX or any component of the product.
- Patients with a known history of hypersensitivity to hamster protein.
- Patients participating in an interventional trial of any investigational drug or device.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Post Marketing Surveillance To Observe Safety And Efficacy Of BeneFIX In Patients With Hemophilia B
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:
- Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses.
- Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug
Read the detailed description
The patients who meet the inclusion criteria will be enrolled consecutively.
- Conditions
- Hemophilia B
- Being tested
- BeneFIX (coagulation factor IX (recombinant))
- Keywords
- Hemophilia B, BeneFIX, Safety, Efficacy
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BeneFIX (coagulation factor IX (recombinant))Drug
BeneFIX will be administered according to physician's discretion.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Not described Receives: Drug: BeneFIX (coagulation factor IX (recombinant)) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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'.
Measured: 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).
Measured: 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.
Measured: Baseline up to 6 months
Secondary outcomes (7)
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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline up to 6 months
Total Infusion of Study Medication
Total dose of study drug infused was calculated over the study duration.
Measured: 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'.
Measured: Baseline up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2007 (estimated)
- Study startAug 2007
- Primary completionJun 2012
- Study completionJun 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 183 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00484185
- Sponsor's protocol ID
- 3090X1-4403
- Other IDs
- B1821005
- Status last verified
- May 2013
- First submitted
- 7 Jun 2007
- Last updated
- 12 Aug 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post Marketing Surveillance To Observe Safety And Efficacy Of BeneFIX In Patients With Hemophilia B
- ClinicalTrials.gov ID
- NCT00484185
- Acronym / sponsor ID
- 3090X1-4403
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, BeneFIX, Safety, Efficacy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 183 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- Aug 2007
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 8 Jun 2007 (estimated)
- Results
- Posted 12 Aug 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses.
- Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug
Detailed description
The patients who meet the inclusion criteria will be enrolled consecutively.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | Drug: BeneFIX (coagulation factor IX (recombinant)) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Hemophilia B (congenital factor IX deficiency or Christmas disease).
Key inclusion criteria (2 listed)
- Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement).
- Pediatric and adult patients who have been treated with original or reformulated BeneFIX for hemophilia B (congenital factor IX deficiency or Christmas disease) from first approved date by KFDA, or who are planned to be newly prescribed BeneFIX (for example, patients switching from pdFIX to BeneFIX).
Key exclusion criteria (3 listed)
- Patients with a known history of hypersensitivity to original or reformulated BeneFIX or any component of the product.
- Patients with a known history of hypersensitivity to hamster protein.
- Patients participating in an interventional trial of any investigational drug or device.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BeneFIX (coagulation factor IX (recombinant))BeneFIX will be administered according to physician's discretion. | Drug | 1 |
Primary outcome
Number of Participants With Adverse Events (AEs) According to Baseline Characteristics
Time frame: Baseline up to 6 months
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.
Number of Participants With Adverse Events (AEs) According to Severity
Time frame: 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. 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).
Number of Participants With Action Taken in Response to Adverse Events (AEs)
Time frame: 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. 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.
Number of Participants With Adverse Events (AEs) According to Seriousness
Time frame: 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. 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.
Number of Participants With Outcome in Response to Adverse Events (AEs)
Time frame: 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. Outcome of an AE was assessed based on response to a question 'Is the adverse event still present?' as 'yes', 'unknown' or 'no-resolved'.
Number of Participants With Adverse Events (AEs) by Relationship
Time frame: Baseline up to 6 months
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).
Number of Participants With Unexpected Adverse Events (AEs)
Time frame: 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. Unexpected AEs were those that were not included in precaution of local product document.
7 secondary outcomes
Mean Annualized Bleeding Rate (ABR)
Time frame: Baseline up to 6 months
Number of Responses to On-demand Treatment With Study Medication
Time frame: Baseline up to 6 months
Mean Number of Infusion of Study Medication
Time frame: Baseline up to 6 months
Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication
Time frame: Baseline up to 6 months
Average Infusion Dose of Study Medication
Time frame: Baseline up to 6 months
Total Infusion of Study Medication
Time frame: Baseline up to 6 months
Percentage of Participants With Efficacy Evaluation
Time frame: Baseline up to 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B, run by Pfizer at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Aug 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Aug 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) According to Baseline Characteristics (time frame: Baseline up to 6 months). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2013.