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Clinical Trials/NCT00950170
NCT00950170CompletedPhase 4

AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS

Pfizer22 sites in 7 countries23 target enrollmentStarted: February 10, 2010Last updated:
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Conditions
Interventions

Trial Snapshot

Phase
Phase 4
Status
Completed
Sponsor
Pfizer
Enrollment
23
Locations
22
Primary Endpoint
Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia. Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study. The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.

Detailed Description

Regulatory Commitment

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Other
Masking
None

Eligibility Criteria

Ages
— to 6 Years (Child)
Sex
Male
Accepts Healthy Volunteers
No

Inclusion Criteria

  • •Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
  • •No prior exposure to factor products or any blood products.

Exclusion Criteria

  • •Presence of any bleeding disorder in addition to hemophilia A.
  • •Treatment with any investigational agent or device within the past 30 days.
  • •Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).

Arms & Interventions

1

Experimental

The investigator treats subjects with ReFacto AF in the usual care setting.

Intervention: Laboratory Tests (Procedure)

Outcomes

Primary Outcomes

Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study

Time Frame: 2 years

Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.

Secondary Outcomes

  • Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF(2 years)
  • Annualized Bleeding Rate (ABR)(2 years)
  • Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion(Within 48 hours after infusion, up to 2 years treatment duration)
  • Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF(2 years)
  • Consumption of Total International Units of Factor VIII(2 years)
  • Consumption of Total International Units of Factor VIII Per Year(2 years)
  • Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight(2 years)
  • Mean of Total Number of Infusions of Study Drug Received(2 years)
  • Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting(2 years)
  • Mean Dose (IU) of Study Drug Consumed Per Infusion(2 years)
  • Consumption of Total International Units of Factor VIII by Weight(2 years)
  • Consumption of Total International Units of Factor VIII Per Year by Weight(2 years)
  • Mean of Total Number of Days Participants Exposed to Study Drug(2 years)
  • Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study(2 years)
  • Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting(2 years)
  • Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting(2 years)

Investigators

Sponsor
Pfizer
Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (22)

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