Back to resultsStudy Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
Primary Purpose
Hemophilia A
Status
Completed
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Xyntha
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia A focused on measuring On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese
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
Sites / Locations
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Xyntha
Arm Description
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.
Outcomes
Primary Outcome Measures
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).
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).
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.
Secondary Outcome Measures
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.
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.
Number of Participants With Thrombosis Allergic-Type Reactions
Number of Participants With Thrombosis
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00868530
Brief Title
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
2. Study Status
Record Verification Date
March 2017
Overall Recruitment Status
Completed
Study Start Date
September 2008 (undefined)
Primary Completion Date
December 2009 (Actual)
Study Completion Date
December 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
53 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Xyntha
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
Xyntha
Intervention Description
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 Outcome Measure Information:
Title
Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
Description
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).
Time Frame
8 hours post infusion
Title
Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion
Description
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).
Time Frame
24 hours post infusion
Title
Number of Participants With Factor VIII (FVIII) Inhibitor Development
Description
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.
Time Frame
Day 1 and Month 6 or Early Termination Visit
Secondary Outcome Measure Information:
Title
FVIII Recovery : Change From Baseline in FVIII Concentration
Description
FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.
Time Frame
Day 1 and Month 6 or Early Termination Visit
Title
Number of Participants With Less Than Expected Therapeutic Effect (LETE)
Description
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.
Time Frame
24 hours after each of 2 successive infusion, up to 6 months
Title
Number of Participants With Thrombosis Allergic-Type Reactions
Time Frame
Baseline up to 6 months
Title
Number of Participants With Thrombosis
Time Frame
Baseline up to 6 months
Other Pre-specified Outcome Measures:
Title
Frequency of Xyntha Infusions Required Per Hemorrhage
Description
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.
Time Frame
Day 1 to Month 6 or Early Termination Visit
Title
Average Dose of Xyntha Infusions Required Per Hemorrhage
Description
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.
Time Frame
Day 1 to Month 6 or Early Termination Visit
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Pfizer Investigational Site
City
Suzhou
State/Province
Jiangsu
ZIP/Postal Code
215006
Country
China
Facility Name
Pfizer Investigational Site
City
Heping District
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Facility Name
Pfizer Investigational Site
City
Hangzhou
State/Province
Zhejiang
ZIP/Postal Code
310003
Country
China
Facility Name
Pfizer Investigational Site
City
Beijing
ZIP/Postal Code
100730
Country
China
Facility Name
Pfizer Investigational Site
City
Guangzhou
ZIP/Postal Code
510515
Country
China
Facility Name
Pfizer Investigational Site
City
Shanghai
ZIP/Postal Code
200025
Country
China
12. IPD Sharing Statement
Citations:
PubMed Identifier
33269010
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.
Results Reference
derived
Links:
URL
https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=3082B2-3316&StudyName=Study%20Evaluating%20On-Demand%20Treatment%20Of%20Xyntha%20In%20Chinese%20Subjects
Description
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Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
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