Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2014 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00543439
First received: October 11, 2007
Last updated: October 9, 2014
Last verified: October 2014
  Purpose

The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately severe hemophilia A.


Condition Intervention Phase
Hemophilia A
Biological: Moroctocog alfa (AF-CC)
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label Study To Evaluate Prophylaxis Treatment, And To Characterize The Efficacy, Safety, And Pharmacokinetics Of B-domain Deleted Recombinant Factor Viii Albumin Free (Moroctocog Alfa [Af-cc]) In Children With Hemophilia A

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • The annualized bleed rate is the primary endpoint for testing the primary objective of the study (comparing prophylaxis to on-demand therapy) [ Time Frame: End of Study ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The annualized bleed rate is the endpoint for testing one of the secondary objectives (comparing high vs low frequency prophylaxis regimens). [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Number of moroctocog alfa (AF-CC) infusions per bleed [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Response of bleed to moroctocog alfa (AF-CC) treatment (4-point scale of assessment) [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Time interval between bleed onset and prior moroctocog alfa (AF-CC) prophylaxis dose [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Incidence of prophylaxis regimen escalation [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Incidence of Less than Expected Therapeutic Effect [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Consumption of moroctocog alfa (AF-CC) [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Compliance with assigned prophylaxis regimen [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • Incidence of Adverse Events [ Time Frame: End of Study ] [ Designated as safety issue: Yes ]
  • Incidence of confirmed FVIII inhibitor development [ Time Frame: End of Study ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 72
Study Start Date: December 2007
Estimated Study Completion Date: January 2016
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
On-Demand therapy for 6 months, followed by Routine Prophylaxis treatment for 1 year.
Biological: Moroctocog alfa (AF-CC)
On-demand therapy for 6 months, followed by routine prophylaxis 25 IU/kg, administered every other day for 1 year.
Other Name: Xyntha
Experimental: 2
Routine Prophylaxis Crossover
Biological: Moroctocog alfa (AF-CC)

Routine prophylaxis crossover:

45 IU/kg, administered 2 times a week for 1 year followed by 25 IU/kg administered every other day for 1 year, or, 25 IU/kg, administered every other day for 1 year, followed by 45 IU/kg, administered 2 times a week for 1 year.

Other Name: Xyntha

  Eligibility

Ages Eligible for Study:   up to 6 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A.
  • A negative FVIII inhibitor titer at screening, and a medical history negative for a past FVIII inhibitor.
  • At least 20 exposure days to any FVIII replacement product.
  • Adequate hepatic and renal function
  • CD4 count > 400 cells/uL, and if receiving antiviral therapy must be on a stable regimen

Additional criteria for subjects participating in the PK assessment:

  • Male subjects as described immediately above except they must have a FVIII Activity of less than or equal to 1% confirmed by the central laboratory screening test
  • Age < 6 years at time of PK assessment.
  • The subject's size is sufficient to permit PK-related phlebotomy.
  • The subject is able to comply with the procedures conducted during the PK assessment, including a mandatory 72-hour washout period preceding the PK assessment.

Exclusion Criteria:

  • A history of FVIII inhibitor.
  • Presence of a bleeding disorder in addition to hemophilia A.
  • Treatment with any investigational drug or device within 30 days before the time of signing the informed consent form.
  • Major or orthopedic surgery planned to occur during the course of the study.
  • Regular (e.g., daily, every other day) use of antifibrinolytic agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDs), or regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids).
  • 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: NCT00543439

Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021

  Show 26 Study Locations
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00543439     History of Changes
Other Study ID Numbers: 3082B2-313, B1831001, 2006-005575-17
Study First Received: October 11, 2007
Last Updated: October 9, 2014
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on October 20, 2014