Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00950170
First received: July 29, 2009
Last updated: August 11, 2014
Last verified: August 2014

July 29, 2009
August 11, 2014
February 2010
February 2022   (final data collection date for primary outcome measure)
Proportion of subjects who develop clinically significant FVIII inhibitors during the course of the study. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: Yes ]
The primary safety outcome measure will be the proportion of subjects who develop clinically significant FVIII inhibitors during the course of the study. [ Time Frame: 5 years (study duration) ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00950170 on ClinicalTrials.gov Archive Site
  • Annualized bleeding rates. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
  • Responses to the first on-demand treatment with Refacto AF for all new bleeds. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
  • The number of ReFacto AF infusions to treat each new bleed. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
  • The number of bleeds within 48 hours of a prophylaxis dose of ReFacto AF. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
  • The average infusion dose and total factor consumption [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
  • The number of subjects who require dose escalation of their prescribed prophylaxis regimen during their participation in this study. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
  • The incidence of less-than-expected therapeutic effect (LETE). [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
Efficacy endpoints include annualized bleeding rates, responses to the first on-demand treatment with Refacto AF for all new bleeds, the number of ReFacto AF infusions to treat each new bleed, and additional efficacy measurements. [ Time Frame: 5 years (study duration) ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
An Open-Label Study Of The Safety And Efficacy Of ReFacto AF In Previously Untreated Patients In Usual Care Settings

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.

Regulatory Commitment

Interventional
Phase 4
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Hemophilia A
Procedure: Laboratory Tests
Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
Other Name: ReFacto AF
Experimental: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
Intervention: Procedure: Laboratory Tests
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
50
February 2022
February 2022   (final data collection date for primary outcome measure)

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).
Male
up to 5 Years
No
Contact: Pfizer CT.gov Call Center 1-800-718-1021
France,   Georgia,   Germany,   Greece,   Italy,   Netherlands,   Romania,   Spain,   Sweden,   Turkey,   Ukraine
 
NCT00950170
3082B2-4434, B1831006
No
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP