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 September 2014 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00950170
First received: July 29, 2009
Last updated: September 4, 2014
Last verified: September 2014
  Purpose

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.


Condition Intervention Phase
Hemophilia A
Procedure: Laboratory Tests
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: An Open-Label Study Of The Safety And Efficacy Of ReFacto AF In Previously Untreated Patients In Usual Care Settings

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 50
Study Start Date: February 2010
Estimated Study Completion Date: February 2022
Estimated Primary Completion Date: February 2022 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
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

Detailed Description:

Regulatory Commitment

  Eligibility

Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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).
  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: NCT00950170

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

Locations
France
Pfizer Investigational Site Recruiting
Lille Cedex, France, 59037
Pfizer Investigational Site Completed
Nantes, France, 44093
Georgia
Pfizer Investigational Site Not yet recruiting
Tbilisi, Georgia
Germany
Pfizer Investigational Site Completed
Bremen, Germany, 28177
Pfizer Investigational Site Recruiting
Hannover, Germany, 30625
Pfizer Investigational Site Recruiting
Stuttgart, Germany, 70176
Greece
Pfizer Investigational Site Recruiting
Athens, Greece
Pfizer Investigational Site Recruiting
Athens, Greece, 11527
Pfizer Investigational Site Recruiting
Thessaloniki, Greece, 546 42
Italy
Pfizer Investigational Site Completed
Firenze, Italy, 50134
Netherlands
Pfizer Investigational Site Recruiting
Groningen, Netherlands, 9713 GZ
Spain
Pfizer Investigational Site Recruiting
A Coruna, Spain, 15006
Pfizer Investigational Site Recruiting
Almeria, Spain, 04009
Pfizer Investigational Site Recruiting
Madrid, Spain, 28046
Pfizer Investigational Site Recruiting
Oviedo, Spain, 33006
Pfizer Investigational Site Recruiting
Sevilla, Spain, 41013
Pfizer Investigational Site Recruiting
Valencia, Spain, 46026
Turkey
Pfizer Investigational Site Recruiting
Antalya, Turkey, 07059
Pfizer Investigational Site Recruiting
Balcali/Adana, Turkey, 01330
Pfizer Investigational Site Recruiting
Bornova/Izmir, Turkey, 35100
Pfizer Investigational Site Recruiting
Fatih/Istanbul, Turkey, 34098
Ukraine
Pfizer Investigational Site Not yet recruiting
Lviv, Ukraine, 79044
Pfizer Investigational Site Not yet recruiting
Zaporizhzhia, Ukraine, 69063
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: NCT00950170     History of Changes
Other Study ID Numbers: 3082B2-4434, B1831006
Study First Received: July 29, 2009
Last Updated: September 4, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
ReFacto AF
factor VIII
inhibitors
antibodies

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 September 16, 2014