Study Evaluating Refacto For Pharmacovigilance

Completed

• Conditions: Hemophilia A
• Interventions: Drug: Moroctocog alfa

• Registration Number: NCT00195442
• Lead Sponsor: Wyeth is now a wholly owned subsidiary of Pfizer

Brief Summary

The purpose of this study is to investigate the effectiveness and safety of treatment with ReFacto under conditions of routine therapy. Furthermore a continuous benefit/risk assessment will be done.

Detailed Description

Non-interventional study: subjects to be selected according to the usual clinical practice of their physician

Study Timeline

• Study Start: 1999-07
• Study First Submitted: 2005-09-13
• Study First Submitted QC: 2005-09-13
• Study First Posted: 2005-09-19
• Primary Completion: 2010-01
• Study Completion: 2010-01
• Results First Submitted: 2011-01-12
• Results First Submitted QC: 2011-01-12
• Status Verified: 2011-02
• Results First Posted: 2011-02-07
• Last Update Submitted: 2011-02-07
• Last Update Posted: 2011-02-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 288

Inclusion Criteria

• Proven diagnosis of Hemophilia A

Exclusion Criteria

• Contraindications according to Summary of Product Characteristics

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
A	Moroctocog alfa	Patients with Hemophilia A

Primary Outcome Measures

Name	Time	Method
Mean Number of Bleeding Episodes Per Patient Year	Baseline up to a mean duration of 54 months	Participants with hemophilia A suffer from a hereditary lack of blood clotting factor VIII. As a consequence, the ability of the blood to coagulate is reduced and bleedings at any site or organ of the body may occur after minor injury or even spontaneously. Predominantly, joints, muscles, and internal organs are affected by bleeding complications. Participants reported the occurrence of each bleeding episode while on study. The bleeding rate for each participant was calculated by number of reported episodes per years on study.
Mean Number of Bleeding-related Exposure Days Per Patient Year	Baseline up to a mean duration of 54 months	Exposure days are the number of days of treatment with ReFacto.
Mean Number of Exposure Days Per Patient Year	Baseline up to a mean duration of 54 months	Exposure days are the number of days of treatment with ReFacto.

Secondary Outcome Measures

Name	Time	Method
Number of Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs)	Baseline up to a mean duration of 54 months	AEs are any undesired side effect which occurred in a participant undergoing study treatment independent of whether a correlation with study treatment was suspected or not. SAEs are undesired events which were lethal or life-threatening, made hospitalization or extension of hospital stay necessary, lead to permanent damage with handicap (inability to work), as well as congenital anomalies, malignant disease, or overdosing. Also presence of inhibitors, thrombotic events, erythrocyte agglutination, allergic reactions, less than therapeutic effect, and inhibitor development were considered SAEs.
Number of Participants With de Novo Inhibitor Formation	Baseline up to a mean duration of 54 months	The applied criteria of clinical relevance for de novo inhibitor formation was defined as normal Factor VIII dosage was ineffective to control a bleeding, control of bleeding episodes required increasing Factor VIII dosage, change of concentrate type (administration of activated Prothrombin-Complex Concentrate \[aPCC\] or recombinant Factor VII \[rFVII \]) was needed to stop a bleeding, or change of therapy strategy (intensive prophylaxis or Immune Tolerance Induction \[ITI\]) was required.
Mean Annual ReFacto Consumption Per Patient Year	Baseline up to a mean duration of 54 months	ReFacto administered as International Units (IU) according to the physician's decision following the drug's summary of product characteristics (SPC) and according to usual care principles.
Number of Participants for Physicians' Assessment of Satisfaction With Treatment Success	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate treatment success (i.e., control of bleeding, Factor VIII consumption, treatment efficacy and tolerance, handling of preparation, and days missing from work or school). Physician rated assessment could be categorized as Very satisfied, Satisfied, Unsatisfied, or Very unsatisfied; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Physicians' Assessment of Efficacy	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate control of bleeding. Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Patients' Assessment of Efficacy	Baseline up to a mean duration of 54 months	Subjective assessment by the participant to evaluate control of bleeding. Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Physicians' Assessment of Tolerance	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate the participants' tolerance of treatment with ReFacto (i.e., dose, administration method, or adverse effects). Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Patients' Assessment of Tolerance	Baseline up to a mean duration of 54 months	Subjective assessment by the participant to evaluate tolerance of treatment with ReFacto (i.e., dose, administration method, or adverse effects). Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Physicians' Assessment of Handling of ReFacto	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate the participants' handling (preparation and administration) of ReFacto. Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Patients' Assessment of Handling of ReFacto	Baseline up to a mean duration of 54 months	Subjective assessment by the participant on handling (preparation and administration) of ReFacto. Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Days of Sick Leave Per Month	Baseline up to a mean duration of 54 months	Days of sick leave (missing work or school) per month categorized as No days of absence, Number of days of absence, Long-term inability to work or study, Not employed or at school, or No specification.

Trial Locations

Locations (1)

Pfizer Investigational Site; 🇩🇪 Wiesbaden, Germany