Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

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

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

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Biological: Xyntha

• Study Type: Interventional
• Enrollment (Actual): 53
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100730
    • Pfizer Investigational Site
  • Guangzhou, China, 510515
    • Pfizer Investigational Site
  • Shanghai, China, 200025
    • Pfizer Investigational Site
  • Jiangsu
    • Suzhou, Jiangsu, China, 215006
      • Pfizer Investigational Site
  • Tianjin
    • Heping District, Tianjin, China, 300020
      • Pfizer Investigational Site
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310003
      • Pfizer Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Investigator Hemostatic Efficacy Assessment 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).	8 hours post infusion
Investigator Hemostatic Efficacy Assessment 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).	24 hours post infusion
Number of Participants With Factor VIII (FVIII) Inhibitor Development	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.	Day 1 and Month 6 or Early Termination Visit

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
FVIII Recovery : Change From Baseline in FVIII Concentration	FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.	Day 1 and Month 6 or Early Termination Visit
Number of Participants With Less Than Expected Therapeutic Effect (LETE)	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.	24 hours after each of 2 successive infusion, up to 6 months
Number of Participants With Thrombosis Allergic-Type Reactions		Baseline up to 6 months
Number of Participants With Thrombosis		Baseline up to 6 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of Xyntha Infusions Required Per Hemorrhage	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.	Day 1 to Month 6 or Early Termination Visit
Average Dose of Xyntha Infusions Required Per Hemorrhage	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.	Day 1 to Month 6 or Early Termination Visit

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

General Publications

• 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.

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start: September 1, 2008
• Primary Completion (Actual): December 1, 2009
• Study Completion (Actual): December 1, 2009

Study Registration Dates

• First Submitted: March 19, 2009
• First Submitted That Met QC Criteria: March 23, 2009
• First Posted (Estimated): March 25, 2009

Study Record Updates

• Last Update Posted (Actual): June 12, 2025
• Last Update Submitted That Met QC Criteria: June 10, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A
• Other Study ID Numbers: 3082B2-3316; B1831015