Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B

NCT00484185 | Completed Results

• 2 other identifiers: 3090X1-4403B1821005
• Study Type: observational
• Target: 183
• Locations: 1 country
• Sites: 1

Brief 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 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

Conditions

Hemophilia B

Keywords

Hemophilia B; BeneFIX; Safety; Efficacy

Outcome Measures

Primary Outcomes (7)

• 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.

  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

Secondary Outcomes (7)

• Mean Annualized Bleeding Rate (ABR)

  Baseline up to 6 months

• Number of Responses to On-demand Treatment With Study Medication

  Baseline up to 6 months

• Mean Number of Infusion of Study Medication

  Baseline up to 6 months

• Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication

  Baseline up to 6 months

• Average Infusion Dose of Study Medication

  Baseline up to 6 months

Other Outcomes (2)

• Duration of Adverse Events (AEs)

  Baseline up to 6 months

• Number of Participants Who Discontinued the Study Due to Adverse Events (AEs)

  Baseline up to 6 months

Study Arms (1)

1

Drug: BeneFIX (coagulation factor IX (recombinant))

Interventions

BeneFIX (coagulation factor IX (recombinant)) DRUG

BeneFIX will be administered according to physician's discretion.

1

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Hemophilia B (congenital factor IX deficiency or Christmas disease).

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (1)

Pfizer Investigational Site

Seoul, 137-882, South Korea

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2007
• First Posted: June 8, 2007
• Study Start: August 1, 2007
• Primary Completion: June 1, 2012
• Study Completion: June 1, 2012
• Last Updated: August 12, 2013
• Results First Posted: August 12, 2013

Record last verified: 2013-05

Locations

KR (1)