The main purpose of this study is to gather information regarding the safety of ReFacto AF, and how well it works (effectiveness) in very young children with severe Hemophilia A under the age of 6.

An Open-Label Study of the Safety and Efficacy of ReFacto AF in Previously Untreated Patients in Usual Care Settings

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2008-008436-93-DE

Enrollment

Registered

2009-05-14

Start date

2009-12-16

Completion date

Unknown

Last updated

2017-03-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Severe hemophilia A (FVIII:C <1%) in male subjects <6 years of age. MedDRA version: 17.0 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 100000004850

Interventions

Trade Name: ReFacto AF Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: MOROCTOCOG-ALFA CAS Number: 284036-24-4 Current Sponsor code: Not Applicable Other descri

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1) Male subjects =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1) Presence of any bleeding disorder in addition to hemophilia A. 2) Treatment with any investigational agent or device within the past 30 days. 3) 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).

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients less than 6 years of age.;Secondary Objective: The secondary objective is to evaluate the efficacy of ReFacto AF.;Primary end point(s): The primary safety outcome is the proportion of patients who develop clinically significant FVIII inhibitors during the course of the study. Clinically significant inhibitors are defined as a central laboratory confirmed positive inhibitor (= 0.6 BU using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6 week interval) and one of the following within 4 weeks before the initial or within 4 weeks following the second positive FVIII inhibitor sample collection: the need for the subject to administer alternative hemostatic products in order to achieve sufficient efficacy, or =2 events indicating a decrease in the efficacy of the test article.;Timepoint(s) of evaluation of this end point: The primary safety outcome measure will be the proportion of patients (previously untreated and less than 6 years of age) who develop clinically significant FVIII inhibitors during the course of the study (approx 24 months).	—

Measure

Time frame

Main Objective: The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients less than 6 years of age.;Secondary Objective: The secondary objective is to evaluate the efficacy of ReFacto AF.;Primary end point(s): The primary safety outcome is the proportion of patients who develop clinically significant FVIII inhibitors during the course of the study. Clinically significant inhibitors are defined as a central laboratory confirmed positive inhibitor (= 0.6 BU using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6 week interval) and one of the following within 4 weeks before the initial or within 4 weeks following the second positive FVIII inhibitor sample collection: the need for the subject to administer alternative hemostatic products in order to achieve sufficient efficacy, or =2 events indicating a decrease in the efficacy of the test article.;Timepoint(s) of evaluation of this end point: The primary safety outcome measure will be the proportion of patients (previously untreated and less than 6 years of age) who develop clinically significant FVIII inhibitors during the course of the study (approx 24 months).

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Secondary

Measure	Time frame
Secondary end point(s): The efficacy endpoints include: Annualized bleeding rates (ABRs) in subjects receiving treatment with ReFacto AF. The responses to the first on-demand treatment with ReFacto AF for all new bleeds (4-point scale of assessment) as assessed by the parent/legal representative. This includes treatment of a bleed while on a preventive/prophylaxis regimen. 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);Timepoint(s) of evaluation of this end point: The secondary endpoints will be monitored throughout the study and will be evaluated and summarized at the end of the study.	—

Measure

Time frame

Secondary end point(s): The efficacy endpoints include: Annualized bleeding rates (ABRs) in subjects receiving treatment with ReFacto AF. The responses to the first on-demand treatment with ReFacto AF for all new bleeds (4-point scale of assessment) as assessed by the parent/legal representative. This includes treatment of a bleed while on a preventive/prophylaxis regimen. 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);Timepoint(s) of evaluation of this end point: The secondary endpoints will be monitored throughout the study and will be evaluated and summarized at the end of the study.

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Countries

Austria, France, Germany, Greece, Italy, Netherlands, Poland, Romania, Spain, Sweden, Turkey

Contacts

Public ContactClinical Trials.gov Call Center

Pfizer Inc

ClinicalTrials.govCallCenter@pfizer.com+18007181021

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026