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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00543439
First Posted: October 15, 2007
Last Update Posted: August 1, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Pfizer
  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
Intervention Model: Crossover Assignment
Masking: None (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 ]

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 ]
  • Number of moroctocog alfa (AF-CC) infusions per bleed [ Time Frame: End of Study ]
  • Response of bleed to moroctocog alfa (AF-CC) treatment (4-point scale of assessment) [ Time Frame: End of Study ]
  • Time interval between bleed onset and prior moroctocog alfa (AF-CC) prophylaxis dose [ Time Frame: End of Study ]
  • Incidence of prophylaxis regimen escalation [ Time Frame: End of Study ]
  • Incidence of Less than Expected Therapeutic Effect [ Time Frame: End of Study ]
  • Consumption of moroctocog alfa (AF-CC) [ Time Frame: End of Study ]
  • Compliance with assigned prophylaxis regimen [ Time Frame: End of Study ]
  • Incidence of Adverse Events [ Time Frame: End of Study ]
  • Incidence of confirmed FVIII inhibitor development [ Time Frame: End of Study ]

Enrollment: 66
Actual Study Start Date: December 11, 2007
Estimated Study Completion Date: April 23, 2018
Estimated Primary Completion Date: April 23, 2018 (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

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 15 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00543439


  Show 28 Study Locations
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00543439     History of Changes
Other Study ID Numbers: 3082B2-313
B1831001 ( Other Identifier: Alias Study Number )
2006-005575-17 ( EudraCT Number )
First Submitted: October 11, 2007
First Posted: October 15, 2007
Last Update Posted: August 1, 2017
Last Verified: July 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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