Clinical trial NCT00950170: Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
Official title: AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A
Keywords: ReFacto AF
factor VIII
inhibitors
antibodies
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2009-07-31
Enrollment: 23
Sex: MALE
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

Detailed description: Regulatory Commitment

Eligibility 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).

Primary outcomes:
measure: Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
timeFrame: 2 years
Secondary outcomes:
measure: Annualized Bleeding Rate (ABR)
timeFrame: 2 years
measure: Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF
timeFrame: 2 years
measure: Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion
timeFrame: Within 48 hours after infusion, up to 2 years treatment duration
measure: Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF
timeFrame: 2 years
measure: Consumption of Total International Units of Factor VIII
timeFrame: 2 years
measure: Consumption of Total International Units of Factor VIII Per Year
timeFrame: 2 years
measure: Mean Dose (IU) of Study Drug Consumed Per Infusion
timeFrame: 2 years
measure: Consumption of Total International Units of Factor VIII by Weight
timeFrame: 2 years
measure: Consumption of Total International Units of Factor VIII Per Year by Weight
timeFrame: 2 years
measure: Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight
timeFrame: 2 years
measure: Mean of Total Number of Infusions of Study Drug Received
timeFrame: 2 years
measure: Mean of Total Number of Days Participants Exposed to Study Drug
timeFrame: 2 years
measure: Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study
timeFrame: 2 years
measure: Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting
timeFrame: 2 years
measure: Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting
timeFrame: 2 years
measure: Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting
timeFrame: 2 years
Other outcomes:
Locations:
city: Nantes
country: France
facility: CHU de Nantes
city: Hannover
country: Germany
facility: Medizinische Hochschule, Hannover
state: Niedersachsen
city: Bremen
country: Germany
facility: Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
city: Firenze
country: Italy
facility: Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Valencia
country: Spain
facility: Hospital Universitario La Fe
city: Bornova
country: Turkey
facility: Ege Universitesi Tip Fakultesi
state: Izmir
city: Adana
country: Turkey
facility: Cukurova Universitesi Tip Fakultesi
city: Istanbul
country: Turkey
facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi
city: Lviv
country: Ukraine
facility: Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: