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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00950170
First Posted: July 31, 2009
Last Update Posted: October 16, 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
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: Intervention Model: Single Group Assignment
Masking: None (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) ]

Secondary Outcome Measures:
  • Annualized bleeding rates. [ Time Frame: 12 years (study duration) ]
  • Responses to the first on-demand treatment with Refacto AF for all new bleeds. [ Time Frame: 12 years (study duration) ]
  • The number of ReFacto AF infusions to treat each new bleed. [ Time Frame: 12 years (study duration) ]
  • The number of bleeds within 48 hours of a prophylaxis dose of ReFacto AF. [ Time Frame: 12 years (study duration) ]
  • The average infusion dose and total factor consumption [ Time Frame: 12 years (study duration) ]
  • 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) ]
  • The incidence of less-than-expected therapeutic effect (LETE). [ Time Frame: 12 years (study duration) ]

Enrollment: 23
Study Start Date: February 2010
Study Completion Date: November 2016
Primary Completion Date: November 2016 (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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   up to 6 Years   (Child)
Sexes 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
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): NCT00950170


Locations
France
CHU de Nantes
Nantes, France, 44093
Germany
Medizinische Hochschule, Hannover
Hannover, Niedersachsen, Germany, 30625
Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
Bremen, Germany, 28177
Italy
Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
Firenze, Italy, 50134
Netherlands
University Medical Center Groningen
Groningen, Netherlands, 9713 GZ
Spain
Hospital Universitario La Paz
Madrid, Spain, 28046
Hospital Universitario La Fe
Valencia, Spain, 46009
Turkey
Cukurova Universitesi Tip Fakultesi
Balcali/Adana, Turkey, 01330
Ege Universitesi Tip Fakultesi
Bornova/Izmir, Turkey, 35100
Istanbul Universitesi Cerrahpasa Tip Fakultesi
Fatih/Istanbul, Turkey, 34098
Ukraine
Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
Lviv, Ukraine, 79044
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00950170     History of Changes
Other Study ID Numbers: 3082B2-4434
B1831006 ( Other Identifier: Alias Study Number )
2008-008436-93 ( EudraCT Number )
First Submitted: July 29, 2009
First Posted: July 31, 2009
Last Update Posted: October 16, 2017
Last Verified: December 2016

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