Study Evaluating Refacto For Pharmacovigilance

This study has been completed.
Sponsor:
Information provided by:
Wyeth is now a wholly owned subsidiary of Pfizer
ClinicalTrials.gov Identifier:
NCT00195442
First received: September 13, 2005
Last updated: February 7, 2011
Last verified: February 2011
  Purpose

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.


Condition Intervention
Hemophilia A
Drug: Moroctocog alfa

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Pharmacovigilance Evaluation Of Refacto In Usual Care Settings

Resource links provided by NLM:


Further study details as provided by Wyeth is now a wholly owned subsidiary of Pfizer:

Primary Outcome Measures:
  • Mean Number of Bleeding Episodes Per Patient Year [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    Exposure days are the number of days of treatment with ReFacto.

  • Mean Number of Exposure Days Per Patient Year [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    Exposure days are the number of days of treatment with ReFacto.


Secondary Outcome Measures:
  • Number of Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline up to a mean duration of 54 months ] [ Designated as safety issue: No ]
    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.


Enrollment: 288
Study Start Date: July 1999
Study Completion Date: January 2010
Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
A
Patients with Hemophilia A
Drug: Moroctocog alfa
Patients will be treated in accordance with the requirements of the labeling of ReFacto (Moroctocog alfa) in Germany. The dosage and duration of therapy is to be determined by the physician to meet the patients' individual needs for treatment.
Other Name: ReFacto

Detailed Description:

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

  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patients with hemophilia A

Criteria

Inclusion Criteria:

  • Proven diagnosis of Hemophilia A

Exclusion Criteria:

  • Contraindications according to Summary of Product Characteristics
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00195442

Locations
Austria
Pfizer Investigational Site
Vienna, Austria, A-1090
Germany
Pfizer Investigational Site
Frankfurt, Hessen, Germany, 60596
Pfizer Investigational Site
Giessen, Hessen, Germany, 35385
Pfizer Investigational Site
Rostock, Mecklenburg-Vorpommern, Germany, 18055
Pfizer Investigational Site
Hannover, Niedersachsen, Germany, 30625
Pfizer Investigational Site
Bonn, Nordrhein-Westfalen, Germany, 53105
Pfizer Investigational Site
Halle, Sachsen-Anhalt, Germany, 06120
Pfizer Investigational Site
Stadtroda, Thuringen, Germany, 07646
Pfizer Investigational Site
Berlin, Germany, 10249
Pfizer Investigational Site
Berlin, Germany, 13353
Pfizer Investigational Site
Bermen, Germany, 28205
Pfizer Investigational Site
Erlangen, Germany, 91054
Pfizer Investigational Site
Frankfurt a. M., Germany, 60594
Pfizer Investigational Site
Hamburg, Germany, 20246
Pfizer Investigational Site
Heidelberg, Germany, 69123
Pfizer Investigational Site
Homburg, Germany, 66424
Pfizer Investigational Site
Klipphausen, Germany, 01665
Pfizer Investigational Site
Leipzig, Germany, 04103
Pfizer Investigational Site
Lubeck, Germany, 23538
Pfizer Investigational Site
Muenchen, Germany, 80336
Pfizer Investigational Site
Muenchen, Germany, 80337
Pfizer Investigational Site
Muenster, Germany, D-48143
Pfizer Investigational Site
Potsdam, Germany, 14467
Pfizer Investigational Site
Schwerin, Germany, 19049
Pfizer Investigational Site
Ulm, Germany, 89081
Pfizer Investigational Site
Wiesbaden, Germany, 65191
Sponsors and Collaborators
Wyeth is now a wholly owned subsidiary of Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Director, Clinical Trial Disclosure Group, Pfizer, Inc
ClinicalTrials.gov Identifier: NCT00195442     History of Changes
Other Study ID Numbers: 3082A-100690
Study First Received: September 13, 2005
Results First Received: January 12, 2011
Last Updated: February 7, 2011
Health Authority: Germany: Paul-Ehrlich-Institut

Keywords provided by Wyeth is now a wholly owned subsidiary of Pfizer:
Hemophilia A

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 02, 2014