Clinical trial NCT00868530: Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia A
Keywords: On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2009-03-25
Enrollment: 53
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility 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

Primary outcomes:
measure: Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
timeFrame: 8 hours post infusion
measure: Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion
timeFrame: 24 hours post infusion
measure: Number of Participants With Factor VIII (FVIII) Inhibitor Development
timeFrame: Day 1 and Month 6 or Early Termination Visit
Secondary outcomes:
measure: FVIII Recovery : Change From Baseline in FVIII Concentration
timeFrame: Day 1 and Month 6 or Early Termination Visit
measure: Number of Participants With Less Than Expected Therapeutic Effect (LETE)
timeFrame: 24 hours after each of 2 successive infusion, up to 6 months
measure: Number of Participants With Thrombosis Allergic-Type Reactions
timeFrame: Baseline up to 6 months
measure: Number of Participants With Thrombosis
timeFrame: Baseline up to 6 months
Other outcomes:
measure: Frequency of Xyntha Infusions Required Per Hemorrhage
timeFrame: Day 1 to Month 6 or Early Termination Visit
measure: Average Dose of Xyntha Infusions Required Per Hemorrhage
timeFrame: Day 1 to Month 6 or Early Termination Visit
Locations:
city: Suzhou
country: China
facility: Pfizer Investigational Site
state: Jiangsu
city: Heping District
country: China
facility: Pfizer Investigational Site
state: Tianjin
city: Hangzhou
country: China
facility: Pfizer Investigational Site
state: Zhejiang
city: Beijing
country: China
facility: Pfizer Investigational Site
city: Guangzhou
country: China
facility: Pfizer Investigational Site
city: Shanghai
country: China
facility: Pfizer Investigational Site
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Yang R, Zhao Y, Wang X, Sun J, Wu R, Jin C, Jin J, Wu D, Rendo P, Sun F, Rupon J, Huard F, Korth-Bradley JM, Xu L, Luo B, Liu YC. Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies. J Blood Med. 2020 Nov 25;11:439-448. doi: 10.2147/JBM.S241605. eCollection 2020.