The main purpose of this study is to gather information regarding the safety of ReFacto AF, and how well it works (effectiveness) in male patients with severe Hemophilia A =12 years of age.

A Postauthorization Safety Surveillance Study of Patients Switching to ReFacto AF From ReFacto or Other Factor VIII Products in Usual Care Settings

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2008-007997-39-DE

Enrollment

300

Registered

2009-01-22

Start date

2009-04-09

Completion date

Unknown

Last updated

2013-08-19

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

Conditions

Severe haemophilia A (FVIII:C 150 EDs to prior recombinant or plasma-derived FVIII replacement products and transitioning to ReFacto AF from ReFacto or other recombinant or plasma-derived FVIII replacement products MedDRA version: 14.1 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders

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 Concentration unit: IU international unit(s) Conc

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1. Male patients =12 years of age with severe hemophilia A (FVIII:C 150 EDs to prior recombinant or plasma-derived FVIII replacement products. 3. Transitioning to ReFacto AF from ReFacto or other recombinant or plasma-derived FVIII replacement products. 4. Albumin = the lower limit of normal (LLN). 5. Platelet count =100,000/µL. 6. Prothrombin time (PT) =1.25 × ULN, or international normalized ratio (INR) =1.5 7. Documented HIV-positive patients must have CD4 count >200/µL and HIV viral load =65 years) yes F.1.3.1 Number of subjects for this age range 30

Exclusion criteria

Exclusion criteria: 1. Presence of any bleeding disorder in addition to hemophilia A. 2. For laboratory assessment, any measured Bethesda inhibitor titer =0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer >ULN for the testing laboratory at the time of screening. 3. Treated with immunomodulatory therapy (including Immune Tolerance Induction [ITI]) during the screening period. 4. Prior exposure to moroctocog alfa (AF-CC). 5. Treatment with any investigational agent or device within 30 days before the Enrollment visit. 6. Known hypersensitivity to hamster protein. 7. Any condition(s) that compromises the patient’s ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; inability to properly store study drug; expectation of poor compliance in study-related documentation). 8. Unwilling or unable to follow the terms of the protocol.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective is to evaluate the safety of ReFacto AF.;Secondary Objective: The secondary objective is to evaluate the efficacy of ReFacto AF.;Primary end point(s): The primary end point is the development of clinically significant FVIII inhibitors. 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 adverse event reports of decreased drug effect (or other AE indicating a decrease in the efficacy of the test article). ;Timepoint(s) of evaluation of this end point: Primary endpoint will be evaluated at the following visits: ED 1, ED 10-15, ED 50, ED 100 and each M6 visits.	—

Measure

Time frame

Main Objective: The primary objective is to evaluate the safety of ReFacto AF.;Secondary Objective: The secondary objective is to evaluate the efficacy of ReFacto AF.;Primary end point(s): The primary end point is the development of clinically significant FVIII inhibitors. 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 adverse event reports of decreased drug effect (or other AE indicating a decrease in the efficacy of the test article). ;Timepoint(s) of evaluation of this end point: Primary endpoint will be evaluated at the following visits: ED 1, ED 10-15, ED 50, ED 100 and each M6 visits.

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Secondary

Measure	Time frame
Secondary end point(s): The secondary end points include: annualized bleeding rates (ABRs) in patients receiving treatment with ReFacto AF, the responses to the first on-demand treatments with ReFacto AF for all new bleeds (4-point scale of assessment) as assessed by the subject or parent/ legal representative of the subject, 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, and the incidence of less-than-expected therapeutic effect (LETE).;Timepoint(s) of evaluation of this end point: Secondary end points will be evaluated at the following visits: ED 1, ED 10-15, ED 50, ED 100 and each M6 visits.	—

Measure

Time frame

Secondary end point(s): The secondary end points include: annualized bleeding rates (ABRs) in patients receiving treatment with ReFacto AF, the responses to the first on-demand treatments with ReFacto AF for all new bleeds (4-point scale of assessment) as assessed by the subject or parent/ legal representative of the subject, 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, and the incidence of less-than-expected therapeutic effect (LETE).;Timepoint(s) of evaluation of this end point: Secondary end points will be evaluated at the following visits: ED 1, ED 10-15, ED 50, ED 100 and each M6 visits.

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Countries

Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Hungary, Italy, Netherlands, Portugal, Spain, Sweden, United Kingdom

Contacts

Public ContactClinical Trials.gov Call Center

Pfizer Inc

ClinicalTrials.govCallCenter@pfizer.com0018007181021

Outcome results

None listed

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