Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

NCT00868530 | Completed Phase 3 Results

• 2 other identifiers: 3082B2-3316B1831015
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 6

Brief Summary

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

Conditions

Hemophilia A

Keywords

On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (4)

• FVIII Recovery : Change From Baseline in FVIII Concentration

  Day 1 and Month 6 or Early Termination Visit

• Number of Participants With Less Than Expected Therapeutic Effect (LETE)

  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 Outcomes (2)

• Frequency of Xyntha Infusions Required Per Hemorrhage

  Day 1 to Month 6 or Early Termination Visit

• Average Dose of Xyntha Infusions Required Per Hemorrhage

  Day 1 to Month 6 or Early Termination Visit

Study Arms (1)

Xyntha

EXPERIMENTAL

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

Interventions

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).

Xyntha

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

Study Sites (6)

Pfizer Investigational Site

Suzhou, Jiangsu, 215006, China

Location

Pfizer Investigational Site

Heping District, Tianjin Municipality, 300020, China

Location

Pfizer Investigational Site

Hangzhou, Zhejiang, 310003, China

Location

Pfizer Investigational Site

Beijing, 100730, China

Location

Pfizer Investigational Site

Guangzhou, 510515, China

Location

Pfizer Investigational Site

Shanghai, 200025, China

Location

Related Publications (1)

• 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.

  PMID: 33269010 DERIVED

Related Links

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

MeSH Terms

Conditions

Hemophilia A

Interventions

F8 protein, human

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2009
• First Posted: March 25, 2009
• Study Start: September 1, 2008
• Primary Completion: December 1, 2009
• Study Completion: December 1, 2009
• Last Updated: June 12, 2025
• Results First Posted: February 8, 2011

Record last verified: 2025-06

Locations

CN (6)