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Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age

Primary Purpose

Hemophilia A

Status
Terminated
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Moroctocog alfa
Sponsored by
Wyeth is now a wholly owned subsidiary of Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring hemophilia A, Xyntha, ReFacto, moroctocog alfa, bleeding disorder

Eligibility Criteria

0 Years - 5 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Male patients less than 6 years of age with moderately severe to severe hemophilia A (FVIII less than or equal to 2%).
  • Treatment history of less than 50 exposure days to prior recombinant or plasma-derived FVIII replacement products.
  • Not receiving treatment for HIV or hepatitis infection, or the patient is on a stable antiviral regimen at the time of enrollment in the study.

Exclusion Criteria:

  • Presence of any bleeding disorder in addition to hemophilia A.
  • Inhibitor titer of greater than or equal to 5 Bethesda Units (BU) at screening.
  • Treated with immunomodulatory therapy during the screening period
  • Treatment history of more than 5 exposure days (ED) to Xyntha.
  • Known hypersensitivity to hamster protein.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Other

    Arm Label

    open label

    Arm Description

    Outcomes

    Primary Outcome Measures

    Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
    Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of >=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
    Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
    LETE in the on-demand setting was based on the response to the treatment of a bleeding episode. LETE in the on-demand setting occurred if the participant recorded 2 successive "No Response" ratings (indicated there was no improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsened) after 2 successive Xyntha infusions, respectively. The infusions was to be administered within 24 hours (=<24 hours) of each other for the treatment of the same bleeding event in the absence of confounding factor.
    Percentage of Participants With LETE in the Prophylaxis Setting
    The LETE in the prophylaxis setting was the occurrence of a bleed. LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (=<48 hours) after a regularly scheduled prophylactic dose of Xyntha (which was not used to treat a bleed) in the absence of confounding factors.
    Percentage of Participants With Low Recovery LETE
    The LETE could be considered lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors.

    Secondary Outcome Measures

    Mean Annualized Bleed Rate (ABR)
    An annualized bleeding rate (ABR) for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the electronic Infusion Log Diary), divided by his total therapy duration (in days), and then multiplied by 365.25.
    Number of Xyntha Infusions Needed to Treat Each New Bleed
    The data from the electronic Infusion Log Diary plus the Test Article case report form (CRF) was used to determine the number of infusions administered to treat a bleed. This was calculated by adding the initial 'for a new bleed' (on demand) infusion to any subsequent (on demand) infusions for the (same) 'previously treated bleed'. An on-demand infusion for a 'previously treated bleed' was counted toward the bleed with the most recent start time prior to that infusion.
    Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver
    A 4-point response scale to be completed is as defined as follows: (Excellent: definite pain relief/improvement in signs of bleeding starting within 8 hrs after an infusion, with no additional infusion; Good: definite pain relief/improvement in signs of bleeding starting within 8 hrs or following the infusion; Moderate: probable/slight improvement starting after 8 hours following the infusion; No Response: no improvement at all between infusions).
    Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds
    The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours following a prophylaxis dose of Xyntha was summarized. The data from the electronic Infusion Log Diary plus the Test Article CRF was used to determine the number of infusions administered to treat a new bleed, counting only those infusions administered =<48 hours after an infusion marked as 'prophylaxis' (which had no associated bleed).

    Full Information

    First Posted
    September 23, 2008
    Last Updated
    May 20, 2022
    Sponsor
    Wyeth is now a wholly owned subsidiary of Pfizer
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00759655
    Brief Title
    Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
    Official Title
    An Open-Label Study To Evaluate The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age In Usual Care Settings
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    May 2022
    Overall Recruitment Status
    Terminated
    Why Stopped
    See termination reason in detailed description.
    Study Start Date
    June 2009 (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
    Wyeth is now a wholly owned subsidiary of Pfizer

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old. Annualized bleeding rates and physician / caregiver assessments of responses to treatment will be characterized. FVIII inhibitor levels will be assessed throughout the study.
    Detailed Description
    The study was terminated on 22 Sept 2009 due to competition with another Wyeth study for a similar patient population. The decision to terminate the trial was not based on any safety issues.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Hemophilia A
    Keywords
    hemophilia A, Xyntha, ReFacto, moroctocog alfa, bleeding disorder

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    1 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    open label
    Arm Type
    Other
    Intervention Type
    Biological
    Intervention Name(s)
    Moroctocog alfa
    Intervention Description
    Patients will receive Moroctocog alfa according to their investigator's prescription.
    Primary Outcome Measure Information:
    Title
    Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
    Description
    Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of >=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
    Time Frame
    Baseline to 24 months or early withdrawal.
    Title
    Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
    Description
    LETE in the on-demand setting was based on the response to the treatment of a bleeding episode. LETE in the on-demand setting occurred if the participant recorded 2 successive "No Response" ratings (indicated there was no improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsened) after 2 successive Xyntha infusions, respectively. The infusions was to be administered within 24 hours (=<24 hours) of each other for the treatment of the same bleeding event in the absence of confounding factor.
    Time Frame
    Baseline to 24 months or early withdrawal.
    Title
    Percentage of Participants With LETE in the Prophylaxis Setting
    Description
    The LETE in the prophylaxis setting was the occurrence of a bleed. LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (=<48 hours) after a regularly scheduled prophylactic dose of Xyntha (which was not used to treat a bleed) in the absence of confounding factors.
    Time Frame
    Baseline to 24 months or early withdrawal.
    Title
    Percentage of Participants With Low Recovery LETE
    Description
    The LETE could be considered lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors.
    Time Frame
    Baseline to 24 months or early withdrawal.
    Secondary Outcome Measure Information:
    Title
    Mean Annualized Bleed Rate (ABR)
    Description
    An annualized bleeding rate (ABR) for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the electronic Infusion Log Diary), divided by his total therapy duration (in days), and then multiplied by 365.25.
    Time Frame
    Baseline to 24 months or early withdrawal.
    Title
    Number of Xyntha Infusions Needed to Treat Each New Bleed
    Description
    The data from the electronic Infusion Log Diary plus the Test Article case report form (CRF) was used to determine the number of infusions administered to treat a bleed. This was calculated by adding the initial 'for a new bleed' (on demand) infusion to any subsequent (on demand) infusions for the (same) 'previously treated bleed'. An on-demand infusion for a 'previously treated bleed' was counted toward the bleed with the most recent start time prior to that infusion.
    Time Frame
    Baseline to 24 months or early withdrawal.
    Title
    Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver
    Description
    A 4-point response scale to be completed is as defined as follows: (Excellent: definite pain relief/improvement in signs of bleeding starting within 8 hrs after an infusion, with no additional infusion; Good: definite pain relief/improvement in signs of bleeding starting within 8 hrs or following the infusion; Moderate: probable/slight improvement starting after 8 hours following the infusion; No Response: no improvement at all between infusions).
    Time Frame
    Baseline to 24 months or early withdrawal
    Title
    Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds
    Description
    The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours following a prophylaxis dose of Xyntha was summarized. The data from the electronic Infusion Log Diary plus the Test Article CRF was used to determine the number of infusions administered to treat a new bleed, counting only those infusions administered =<48 hours after an infusion marked as 'prophylaxis' (which had no associated bleed).
    Time Frame
    Baseline to 24 months or early withdrawal.

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    0 Years
    Maximum Age & Unit of Time
    5 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Male patients less than 6 years of age with moderately severe to severe hemophilia A (FVIII less than or equal to 2%). Treatment history of less than 50 exposure days to prior recombinant or plasma-derived FVIII replacement products. Not receiving treatment for HIV or hepatitis infection, or the patient is on a stable antiviral regimen at the time of enrollment in the study. Exclusion Criteria: Presence of any bleeding disorder in addition to hemophilia A. Inhibitor titer of greater than or equal to 5 Bethesda Units (BU) at screening. Treated with immunomodulatory therapy during the screening period Treatment history of more than 5 exposure days (ED) to Xyntha. Known hypersensitivity to hamster protein.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Pfizer CT.gov Call Center
    Organizational Affiliation
    Pfizer
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Links:
    URL
    https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=3082B2-3315&StudyName=Study%20Evaluating%20The%20Efficacy%20And%20Safety%20Of%20Xyntha%20In%20Children%20Less%20Than%206%20Years%20Of%20Age
    Description
    To obtain contact information for a study center near you, click here.

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    Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age

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