Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

NCT00866606 | Completed Phase 3 Results

• 2 other identifiers: 3090A1-3305B1821004
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 6

Brief Summary

This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.

Conditions

Hemophilia B

Keywords

On-demand treatment; Hemostatic efficacy; Factor IX activity; Factor IX recovery; Factor IX inhibitor; Chinese

Outcome Measures

Primary Outcomes (3)

• Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion

  Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.

  8 hours post infusion

• Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion

  Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.

  24 hours post infusion

• Percentage of Participants With FIX Inhibitor Development

  Incidence of FIX inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory with Nijmegen assay result \>=0.6 Bethesda Unit (BU). Incidence was stratified by participant exposure history - Minimally Treated Patients (MTPs): those who had received at least one prior FIX infusion, and \<= 100 documented Exposure Days (EDs); while Previously Treated Patients (PTPs): those who had received \>100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.

  Baseline up to 6 months

Secondary Outcomes (6)

• Number of Infusions Required to Treat Each Bleed

  Baseline up to 6 months

• FIX Incremental Recovery

  Baseline (Visit 2) up to 6 months (Visit 4)

• Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)

  Baseline up to 6 months

• Percentage of Participants With Allergic-Type Allergic Reactions

  Baseline up to 6 months

• Percentage of Participants With Thrombosis

  Baseline up to 6 months

Study Arms (1)

Benefix

EXPERIMENTAL

Subjects received on-demand treatments with BeneFIX over a 6-month (calendar day) period.

Biological: Benefix

Interventions

Benefix BIOLOGICAL

BeneFIX for on-demand treatment of bleeding episodes were according to investigator prescription. FIX recovery was assessed by determining the FIX activity (FIX:C) levels in individual subjects. BeneFIX dosage for recovery assessments: single 75 IU/kg (±5 IU/kg) IV bolus infusion.

Benefix

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia B (FIX activity: more than 5%, 1-5%, or less than 1%, respectively)
• Subjects with previous exposure to FIX replacement therapy
• If HIV positive, documented CD4 count more than 200/µL within 6 months of study entry

You may not qualify if:

• Diagnosed with any bleeding disorder in addition to hemophilia B
• Current FIX inhibitor or history of FIX inhibitor (defined as \>ULN of the reporting laboratory)
• Subject has no history of exposure to FIX products (previously untreated patient \[PUP\])
• Subject is currently utilizing primary FIX prophylaxis
• Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry
• Treated with immunomodulatory therapy within 30 days prior to study entry or planned use for the duration of their study participation
• Participated in another investigational drug or device study within 30 days prior to study entry or planned participation for the duration of their study participation
• Subjects with a known hypersensitivity to hamster protein
• Significant hepatic or renal impairment (ALT and AST \>5 x ULN, bilirubin \>2 mg/dL or serum creatinine \>1.25 x ULN)
• Prothrombin Time \>1.5 x ULN
• Platelet count \<80,000/µL
• Pregnant or breastfeeding women
• Unwilling or unable to follow the terms of the protocol
• Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation, in the opinion of the Investigator or Sponsor

Sponsors & Collaborators

• Wyeth is now a wholly owned subsidiary of Pfizer: lead

Study Sites (6)

Pfizer Investigational Site

Guangzhou, Guangzhou, 510515, China

Location

Pfizer Investigational Site

Suzhou, Jiangsu, 215006, China

Location

Pfizer Investigational Site

Tianjin, Tianjin Municipality, 300020, China

Location

Pfizer Investigational Site

Hangzhou, Zhejiang, 310003, China

Location

Pfizer Investigational Site

Beijing, 100730, China

Location

Pfizer Investigational Site

Shanghai, 200025, China

Location

Related Publications (1)

• Yang R, Zhao Y, Wang X, Sun J, Jin J, Wu D, Charnigo R, O'Brien A, Zhong Z, Rendo P. Evaluation of the safety and efficacy of recombinant factor IX (nonacog alfa) in minimally treated and previously treated Chinese patients with haemophilia B. Haemophilia. 2012 Sep;18(5):e374-8. doi: 10.1111/j.1365-2516.2012.02907.x. Epub 2012 Jul 9. No abstract available.

  PMID: 22776196 DERIVED

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: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: March 19, 2009
• First Posted: March 20, 2009
• Study Start: February 1, 2009
• Primary Completion: December 1, 2009
• Study Completion: December 1, 2009
• Last Updated: April 5, 2011
• Results First Posted: January 4, 2011

Record last verified: 2011-04

Locations

CN (6)