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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Pfizer Identifier:
First received: July 29, 2009
Last updated: September 14, 2015
Last verified: September 2015

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 ]

Enrollment: 24
Study Start Date: February 2010
Estimated Study Completion Date: November 2016
Estimated Primary Completion Date: October 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


Ages Eligible for Study:   up to 6 Years
Genders 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).
  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 identifier: NCT00950170

CHU de Nantes
Nantes, France, 44093
Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
Bremen, Germany, 28177
Medizinische Hochschule, Hannover
Hannover, Germany, 30625
Stuttgart, Germany, 70176
Klinikum Stuttgart-Olgahospital
Stuttgart, Germany, 70174
Azienda Ospedaliera Careggi
Firenze, Italy, 50134
Universitair Medisch Centrum Groningen
Groningen, Netherlands, 9713 GZ
Hospital La Paz
Madrid, Spain, 28046
Hospital Politécnico Universitario La Fe
Valencia, Spain, 46026
Hospital Universitario y Politécnico La Fe
Valencia, Spain, 46026
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
State institution "The Institute of blood pathology and transfusion medicine of AMS of Ukraine"
Lviv, Ukraine, 79044
Sponsors and Collaborators
Study Director: Pfizer Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer Identifier: NCT00950170     History of Changes
Other Study ID Numbers: 3082B2-4434, B1831006, 2008-008436-93
Study First Received: July 29, 2009
Last Updated: September 14, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
ReFacto AF
factor VIII

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders
Factor VIII
Hematologic Agents
Pharmacologic Actions
Therapeutic Uses processed this record on October 09, 2015