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Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

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ClinicalTrials.gov Identifier: NCT00868530
Recruitment Status : Completed
First Posted : March 25, 2009
Results First Posted : February 8, 2011
Last Update Posted : April 5, 2017
Sponsor:
Information provided by (Responsible Party):
Pfizer

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

Condition or disease Intervention/treatment Phase
Hemophilia A Biological: Xyntha Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 53 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : September 2008
Actual Primary Completion Date : December 2009
Actual Study Completion Date : December 2009

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arms and Interventions
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Arm Intervention/treatment
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).


Outcome Measures
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Primary Outcome Measures :
  1. Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion [ Time Frame: 8 hours post infusion ]
    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).

  2. Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion [ Time Frame: 24 hours post infusion ]
    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).

  3. Number of Participants With Factor VIII (FVIII) Inhibitor Development [ Time Frame: Day 1 and Month 6 or Early Termination Visit ]
    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 :
  1. FVIII Recovery : Change From Baseline in FVIII Concentration [ Time Frame: Day 1 and Month 6 or Early Termination Visit ]
    FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.

  2. Number of Participants With Less Than Expected Therapeutic Effect (LETE) [ Time Frame: 24 hours after each of 2 successive infusion, up to 6 months ]
    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.

  3. Number of Participants With Thrombosis Allergic-Type Reactions [ Time Frame: Baseline up to 6 months ]
  4. Number of Participants With Thrombosis [ Time Frame: Baseline up to 6 months ]

Other Outcome Measures:
  1. Frequency of Xyntha Infusions Required Per Hemorrhage [ Time Frame: Day 1 to Month 6 or Early Termination Visit ]
    The mean frequency of Xyntha infusions per hemorrhage was calculated as total number of injections throughout the study divided by total number of hemorrhagic events.

  2. Average Dose of Xyntha Infusions Required Per Hemorrhage [ Time Frame: Day 1 to Month 6 or Early Termination Visit ]
    The average dose of Xyntha per hemorrhagic event was calculated as total dose of Xyntha throughout the study (in IU) divided by total number of hemorrhage incidence.


Eligibility Criteria
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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00868530


Locations
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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
Guangzhou, China, 510515
Pfizer Investigational Site
Shanghai, China, 200025
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00868530    
Other Study ID Numbers: 3082B2-3316
B1831015
First Posted: March 25, 2009    Key Record Dates
Results First Posted: February 8, 2011
Last Update Posted: April 5, 2017
Last Verified: March 2017
Keywords provided by Pfizer:
On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn