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

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. Identifier: NCT00950170
Recruitment Status : Completed
First Posted : July 31, 2009
Last Update Posted : December 29, 2016
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Procedure: Laboratory Tests Phase 4

Detailed Description:
Regulatory Commitment

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
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
Study Start Date : February 2010
Actual Primary Completion Date : November 2016
Actual Study Completion Date : November 2016

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. Proportion of subjects who develop clinically significant FVIII inhibitors during the course of the study. [ Time Frame: 12 years (study duration) ]

Secondary Outcome Measures :
  1. Annualized bleeding rates. [ Time Frame: 12 years (study duration) ]
  2. Responses to the first on-demand treatment with Refacto AF for all new bleeds. [ Time Frame: 12 years (study duration) ]
  3. The number of ReFacto AF infusions to treat each new bleed. [ Time Frame: 12 years (study duration) ]
  4. The number of bleeds within 48 hours of a prophylaxis dose of ReFacto AF. [ Time Frame: 12 years (study duration) ]
  5. The average infusion dose and total factor consumption [ Time Frame: 12 years (study duration) ]
  6. 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) ]
  7. The incidence of less-than-expected therapeutic effect (LETE). [ Time Frame: 12 years (study duration) ]

Information from the National Library of Medicine

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

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 identifier (NCT number): NCT00950170

CHU de Nantes
Nantes, France, 44093
Medizinische Hochschule, Hannover
Hannover, Niedersachsen, Germany, 30625
Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
Bremen, Germany, 28177
Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
Firenze, Italy, 50134
University Medical Center Groningen
Groningen, Netherlands, 9713 GZ
Hospital Universitario La Paz
Madrid, Spain, 28046
Hospital Universitario La Fe
Valencia, Spain, 46009
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
Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
Lviv, Ukraine, 79044
Sponsors and Collaborators
Study Director: Pfizer Call Center Pfizer

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

Keywords provided by Pfizer:
ReFacto AF
factor VIII

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