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Completed

NCT Number: NCT00868530

Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

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

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Pfizer Investigational Site, Suzhou, Jiangsu, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Xyntha

Biological

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).

Primary outcomes

  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 outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study 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

Important dates

Study start
2008
Primary completion
2009
Study completion
2009
First posted
Mar 25, 2009
Registry last updated
Jun 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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