Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

Phase 3

Completed

Conditions: Hemophilia A

Interventions: Biological: Xyntha

Registration Number: NCT00868530

Lead Sponsor: Pfizer

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

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 53

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Xyntha	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.

Primary Outcome Measures

Name	Time	Method
Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion	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).
Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion	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).
Number of Participants With Factor VIII (FVIII) Inhibitor Development	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

Name	Time	Method
FVIII Recovery : Change From Baseline in FVIII Concentration	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.
Number of Participants With Less Than Expected Therapeutic Effect (LETE)	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.
Number of Participants With Thrombosis Allergic-Type Reactions	Baseline up to 6 months
Number of Participants With Thrombosis	Baseline up to 6 months