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

NCT00950170 | Completed Phase 4 Results

• 3 other identifiers: 3082B2-4434B18310062008-008436-93
• Study Type: interventional
• Target: 23
• Locations: 7 countries
• Sites: 11

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.

Conditions

Hemophilia A

Keywords

ReFacto AF; factor VIII; inhibitors; antibodies

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study

  Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.

  2 years

Secondary Outcomes (16)

• Annualized Bleeding Rate (ABR)

  2 years

• Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF

  2 years

• Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion

  Within 48 hours after infusion, up to 2 years treatment duration

• Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF

  2 years

• Consumption of Total International Units of Factor VIII

  2 years

Study Arms (1)

1

EXPERIMENTAL

The investigator treats subjects with ReFacto AF in the usual care setting.

Procedure: Laboratory Tests

Interventions

Laboratory Tests PROCEDURE

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).

Also known as: ReFacto AF

1

Eligibility Criteria

• Age: Up to 6 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Pfizer: lead

Study Sites (11)

CHU de Nantes

Nantes, 44093, France

Location

Medizinische Hochschule, Hannover

Hanover, Lower Saxony, 30625, Germany

Location

Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik

Bremen, 28177, Germany

Location

Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite

Florence, 50134, Italy

Location

University Medical Center Groningen

Groningen, 9713 GZ, Netherlands

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Universitario La Fe

Valencia, 46009, Spain

Location

Ege Universitesi Tip Fakultesi

Bornova, İzmir, 35100, Turkey (Türkiye)

Location

Cukurova Universitesi Tip Fakultesi

Adana, 01330, Turkey (Türkiye)

Location

Istanbul Universitesi Cerrahpasa Tip Fakultesi

Istanbul, 34098, Turkey (Türkiye)

Location

Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii

Lviv, 79044, Ukraine

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia A

Interventions

Clinical Laboratory Techniques; Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 29, 2009
• First Posted: July 31, 2009
• Study Start: February 10, 2010
• Primary Completion: November 24, 2016
• Study Completion: November 24, 2016
• Last Updated: July 19, 2019
• Results First Posted: July 19, 2019

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will share

Locations

ES (2); TU (3); NL (1); UA (1); FR (1); DE (2); IT (1)