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

This study has been terminated.
(See termination reason in detailed description.)
Sponsor:
Information provided by:
Wyeth is now a wholly owned subsidiary of Pfizer
ClinicalTrials.gov Identifier:
NCT00759655
First received: September 23, 2008
Last updated: February 3, 2011
Last verified: February 2011
  Purpose

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.


Condition Intervention Phase
Hemophilia A
Biological: Moroctocog alfa
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Wyeth is now a wholly owned subsidiary of Pfizer:

Primary Outcome Measures:
  • Percentage of Participants With Factor VIII (FVIII) Inhibitor Development [ Time Frame: Baseline to 24 months or early withdrawal. ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: Baseline to 24 months or early withdrawal. ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline to 24 months or early withdrawal. ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline to 24 months or early withdrawal. ] [ Designated as safety issue: No ]
    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) [ Time Frame: Baseline to 24 months or early withdrawal. ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline to 24 months or early withdrawal. ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline to 24 months or early withdrawal ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline to 24 months or early withdrawal. ] [ Designated as safety issue: No ]
    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).


Enrollment: 1
Study Start Date: June 2009
Study Completion Date: December 2009
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
open label Biological: Moroctocog alfa
Patients will receive Moroctocog alfa according to their investigator's prescription.

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.

  Eligibility

Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00759655

Sponsors and Collaborators
Wyeth is now a wholly owned subsidiary of Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Director, Clinical Trial Disclosure Group, Pfizer, Inc
ClinicalTrials.gov Identifier: NCT00759655     History of Changes
Other Study ID Numbers: 3082B2-3315, B1831002, 3082B2-3315-WW
Study First Received: September 23, 2008
Results First Received: December 14, 2010
Last Updated: February 3, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Wyeth is now a wholly owned subsidiary of Pfizer:
hemophilia A
Xyntha
ReFacto
moroctocog alfa
bleeding disorder

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 19, 2014