Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
Information source: Pfizer
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hemophilia A
Intervention: Laboratory Tests (Procedure)
Phase: Phase 4
Status: Active, not recruiting
Sponsored by: Pfizer
Official(s) and/or principal investigator(s):
Pfizer CT.gov Call Center, Study Director, Affiliation: Pfizer
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.
Official title: An Open-label Study Of The Safety And Efficacy Of Refacto Af In Previously Untreated Patients In Usual Care Settings.
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label
Primary outcome: Proportion of subjects who develop clinically significant FVIII inhibitors during the course of the study.
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.
The number of bleeds within 48 hours of a prophylaxis dose of ReFacto AF.
The average infusion dose and total factor consumption
The number of subjects who require dose escalation of their prescribed prophylaxis regimen during their participation in this study.
The incidence of less-than-expected therapeutic effect (LETE).
Minimum age: N/A.
Maximum age: 6 Years.
- 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.
- 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).
Locations and Contacts
CHU de Nantes, Nantes 44093, France
LTD Medinvesti- Institute of Hematology and Transfuziology, Tbilisi, Georgia
Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik, Bremen 28177, Germany
Medizinische Hochschule, Hannover, Hannover 30625, Germany
KlinikumStuttgart, Stuttgart 70176, Germany
Haemophilia Center/Haemostasis Unit, Athens 11527, Greece
"Hippokration" University Hospital of Thessaloniki, Thessaloniki 546 42, Greece
Azienda Ospedaliera Careggi, Firenze 50134, Italy
Universitair Medisch Centrum Groningen, Groningen 9713 GZ, Netherlands
Complexo Hospitalario Juan Canalejo, A Coruna 15006, Spain
Hospital La Paz, Madrid 28046, Spain
Hospital Universitario Central de Asturias, Oviedo 33006, Spain
Hospital Universitario Virgen del Rocio, Sevilla 41013, Spain
Hospital Politécnico Universitario La Fe, Valencia 46026, Spain
Hospital Universitario y Politécnico La Fe, Valencia 46026, Spain
Cukurova Universitesi Tip Fakultesi, Balcali/Adana 01330, Turkey
Ege Universitesi Tip Fakultesi, Bornova/Izmir 35100, Turkey
Istanbul Universitesi Cerrahpasa Tip Fakultesi, Fatih/Istanbul 34098, Turkey
State institution "The Institute of blood pathology and transfusion medicine of AMS of Ukraine", Lviv 79044, Ukraine
Oblasna Klinichna Dytiacha Likarnia, Hematolohichne Viddilennia, Zaporizhzhia 69063, Ukraine
Klinikum Stuttgart-Olgahospital, Stuttgart, Baden-Wuerttemberg 70174, Germany
To obtain contact information for a study center near you, click here.
Starting date: February 2010
Last updated: June 15, 2015