Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

Hemophilia B Clinical Trial

Official title:

An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With BeneFIX (Nonacog Alfa, Recombinant Factor IX) In Chinese Subjects With Hemophilia B

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00866606
• Other study ID #: 3090A1-3305
• Secondary ID: B1821004
• Status: Completed
• Phase: Phase 3
• First received: March 19, 2009
• Last updated: April 1, 2011
• Start date: February 2009
• Est. completion date: December 2009

Study information

• Verified date: April 2011
• Source: Wyeth is now a wholly owned subsidiary of Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 35
• Est. completion date: December 2009
• Est. primary completion date: December 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Biological:
• Benefix
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.

Locations

Country	Name	City	State
China	Pfizer Investigational Site	Beijing
China	Pfizer Investigational Site	Guangzhou	Guangzhou
China	Pfizer Investigational Site	Hangzhou	Zhejiang
China	Pfizer Investigational Site	Shanghai
China	Pfizer Investigational Site	Suzhou	Jiangsu
China	Pfizer Investigational Site	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Wyeth is now a wholly owned subsidiary of Pfizer

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Primary	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	No
Primary	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	Yes
Secondary	Number of Infusions Required to Treat Each Bleed	The number of BeneFIX infusions required to treat each bleeding episode were analyzed. The average frequency of BeneFIX infusions per hemorrhage incidence to treat every hemorrhage was equal to the total number of injections throughout the study divided by total number of hemorrhagic events.	Baseline up to 6 months	No
Secondary	FIX Incremental Recovery	FIX recovery was assessed by evaluating FIX:C after initial exposure and following 6 months of repeated exposures to BeneFIX. A modified FIX recovery study was performed at Day 1 (Visit 2) and Month 6/Final/Early Termination visits (Visit 4) and when clinically indicated at the applicable on-demand visits. Blood samples for determination of FIX:C were collected immediately before BeneFIX infusion and at 30 minutes (±5 minutes) after the start of infusion. Post-infusion blood samples were collected via venipuncture in arm contralateral to arm used for infusion.	Baseline (Visit 2) up to 6 months (Visit 4)	No
Secondary	Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)	The incidence of LETE for on-demand treatment was defined as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.	Baseline up to 6 months	No
Secondary	Percentage of Participants With Allergic-Type Allergic Reactions	Hypersensitivity to undesirable (damaging, discomfort-producing and sometimes fatal) reactions produced by the normal immune system. Hypersensitivity reactions require a pre-sensitized (immune) state of the host.	Baseline up to 6 months	Yes
Secondary	Percentage of Participants With Thrombosis	Thrombosis is the formation of a blood clot (thrombus) inside a blood vessel, obstructing the flow of blood through the circulatory system. When a blood vessel is injured, the body uses platelets and fibrin to form a blood clot to prevent blood loss.	Baseline up to 6 months	Yes
Secondary	Percentage of Participants With Red Blood Cell (RBC) Agglutination	RBC Agglutination is the clumping of red blood cells in the presence of an antibody. The antibody or other molecule bonded multiple particles and joined them, creating a large complex.	Baseline up to 6 months	Yes

External Links

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