Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

This study has been completed.
Sponsor:
Information provided by:
Wyeth is now a wholly owned subsidiary of Pfizer
ClinicalTrials.gov Identifier:
NCT00868530
First received: March 19, 2009
Last updated: April 1, 2011
Last verified: April 2011
  Purpose

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


Condition Intervention Phase
Hemophilia A
Biological: Xyntha
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With Xyntha (B Domain Deleted Recombinant Factor VIII, Albumin Free) In Chinese Subjects With Hemophilia A

Resource links provided by NLM:


Further study details as provided by Wyeth is now a wholly owned subsidiary of Pfizer:

Primary Outcome Measures:
  • Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion [ Time Frame: 8 hours post infusion ] [ Designated as safety issue: No ]
    The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).

  • Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion [ Time Frame: 24 hours post infusion ] [ Designated as safety issue: No ]
    The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).

  • Number of Participants With Factor VIII (FVIII) Inhibitor Development [ Time Frame: Day 1 and Month 6 or Early Termination Visit ] [ Designated as safety issue: Yes ]
    Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by participant exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII 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.


Secondary Outcome Measures:
  • FVIII Recovery : Change From Baseline in FVIII Concentration [ Time Frame: Day 1 and Month 6 or Early Termination Visit ] [ Designated as safety issue: No ]
    FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.

  • Number of Participants With Less Than Expected Therapeutic Effect (LETE) [ Time Frame: 24 hours after each of 2 successive infusion, up to 6 months ] [ Designated as safety issue: No ]
    The incidence of LETE, defined for on-demand treatment as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.

  • Number of Participants With Thrombosis Allergic-Type Reactions [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
  • Number of Participants With Thrombosis [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]

Enrollment: 53
Study Start Date: September 2008
Study Completion Date: December 2009
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Xyntha
This trial was an open-label and included assessments of safety, clinical efficacy, and Factor VIII (FVIII) recovery in Chinese subjects with hemophilia A. Subjects received on-demand treatments with Xyntha over a 6-month (calendar day) period.
Biological: Xyntha
Xyntha for on-demand treatment of bleeding episodes were according to investigator prescription during the 6 months observation period. The recovery assessed by determining the Factor VIII (FVIII) concentration (FVIII:C) levels in individual subjects at the initial and final visits. The dose of Xyntha for recovery assessments is: single 50 IU/kg (±5 IU/kg) IV bolus infusion. All Xyntha administrations occurred in the clinic (hospital).

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • Diagnosed with any bleeding disorder in addition to hemophilia A
  • Current FVIII inhibitor or history of FVIII inhibitor (defined as positive result of the reporting laboratory)
  • Subject has no history of exposure to FVIII products (previously untreated patient [PUP])
  • Subject is currently utilizing primary FVIII 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 (alanine aminotransferase [ALT] and aspartate aminotransferase [AST] >5 x upper limit of normal [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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00868530

Locations
China, Guangzhou
Pfizer Investigational Site
Guangzhou, Guangzhou, China, 510515
China, Jiangsu
Pfizer Investigational Site
Suzhou, Jiangsu, China, 215006
China, Tianjin
Pfizer Investigational Site
Heping District, Tianjin, China, 300020
China, Zhejiang
Pfizer Investigational Site
Hangzhou, Zhejiang, China, 310003
China
Pfizer Investigational Site
Beijing, China, 100730
Pfizer Investigational Site
Shanghai, China, 200025
Sponsors and Collaborators
Wyeth is now a wholly owned subsidiary of Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Director, Clinical Trial Disclosure Group, Pfizer, Inc
ClinicalTrials.gov Identifier: NCT00868530     History of Changes
Other Study ID Numbers: 3082B2-3316, B1831015
Study First Received: March 19, 2009
Results First Received: December 3, 2010
Last Updated: April 1, 2011
Health Authority: China: Food and Drug Administration

Keywords provided by Wyeth is now a wholly owned subsidiary of Pfizer:
On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 24, 2014