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

Condition(s) targeted: Hemophilia A

Intervention: Laboratory Tests (Procedure)

Phase: Phase 4

Status: Recruiting

Sponsored by: Pfizer

Official(s) and/or principal investigator(s):
Pfizer CT.gov Call Center, Study Director, Affiliation: Pfizer

Overall contact:
Pfizer CT.gov Call Center, Phone: 1-800-718-1021

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.

Secondary outcome:

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).

Detailed description: Regulatory Commitment

Minimum age: N/A. Maximum age: 5 Years. Gender(s): Male.

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).

Pfizer CT.gov Call Center, Phone: 1-800-718-1021

Pfizer Investigational Site, Lille Cedex 59037, France; Not yet recruiting

Pfizer Investigational Site, Nantes 44093, France; Recruiting

Pfizer Investigational Site, Bremen 28177, Germany; Active, not recruiting

Pfizer Investigational Site, Hannover 30625, Germany; Recruiting

Pfizer Investigational Site, Firenze 50134, Italy; Recruiting

Pfizer Investigational Site, Milano 20122, Italy; Recruiting

Pfizer Investigational Site, Parma 43100, Italy; Not yet recruiting

Pfizer Investigational Site, Warszawa 00-576, Poland; Not yet recruiting

Pfizer Investigational Site, Bucharest 011155, Romania; Not yet recruiting

Pfizer Investigational Site, A Coruna 15006, Spain; Not yet recruiting

Pfizer Investigational Site, Barcelona 08035, Spain; Not yet recruiting

Pfizer Investigational Site, Madrid 28046, Spain; Recruiting

Pfizer Investigational Site, Sevilla 41013, Spain; Not yet recruiting

Pfizer Investigational Site, Valencia 46026, Spain; Recruiting

Pfizer Investigational Site, Stockholm 171 76, Sweden; Not yet recruiting

Pfizer Investigational Site, Antalya 07059, Turkey; Recruiting

Pfizer Investigational Site, Balcali/Adana 01330, Turkey; Recruiting

Pfizer Investigational Site, Bornova/Izmir 35100, Turkey; Recruiting

Pfizer Investigational Site, Fatih/Istanbul 34098, Turkey; Recruiting

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

Starting date: February 2010
Last updated: February 5, 2013