Overview

This trial is active, not recruiting.

Condition hemophilia a
Treatment laboratory tests
Phase phase 4
Sponsor Pfizer
Start date February 2010
End date November 2016
Trial size 50 participants
Trial identifier NCT00950170, 2008-008436-93, 3082B2-4434, B1831006

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Masking open label
Arm
(Experimental)
The investigator treats subjects with ReFacto AF in the usual care setting.
laboratory tests ReFacto AF
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).

Primary Outcomes

Measure
Proportion of subjects who develop clinically significant FVIII inhibitors during the course of the study.
time frame: 12 years (study duration)

Secondary Outcomes

Measure
Annualized bleeding rates.
time frame: 12 years (study duration)
Responses to the first on-demand treatment with Refacto AF for all new bleeds.
time frame: 12 years (study duration)
The number of ReFacto AF infusions to treat each new bleed.
time frame: 12 years (study duration)
The number of bleeds within 48 hours of a prophylaxis dose of ReFacto AF.
time frame: 12 years (study duration)
The average infusion dose and total factor consumption
time frame: 12 years (study duration)
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)
The incidence of less-than-expected therapeutic effect (LETE).
time frame: 12 years (study duration)

Eligibility Criteria

Male participants up to 6 years old.

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).

Additional Information

Official title An Open-label Study Of The Safety And Efficacy Of Refacto Af In Previously Untreated Patients In Usual Care Settings
Description Regulatory Commitment
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Pfizer.