Hemophilia ANCT00195442
Study Evaluating Refacto For Pharmacovigilance
An observational study of Hemophilia A, run by Wyeth is now a wholly owned subsidiary of Pfizer at 26 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Moroctocog alfa
- How many people
- 288 participants
- Where
- 26 sitesin 2 countries
- Study dates
- Jul 1999to Jan 2010
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with hemophilia A
Who may be able to join
- Proven diagnosis of Hemophilia A
Who cannot join
- Contraindications according to Summary of Product Characteristics
Trial locations and contacts
26 sites in 2 countries.
26 sites in 2 countries
Germany25 sites
Pfizer Investigational Site
Berlin, Germany, 10249
Pfizer Investigational Site
Berlin, 13353
Pfizer Investigational Site
Bermen, 28205
Pfizer Investigational Site
Bonn, North Rhine-Westphalia, 53105
Pfizer Investigational Site
Erlangen, 91054
Pfizer Investigational Site
Frankfurt A. M., 60594
Pfizer Investigational Site
Frankfurt am Main, Hesse, 60596
Pfizer Investigational Site
Giessen, Hesse, 35385
Pfizer Investigational Site
Halle, Saxony-Anhalt, 06120
Pfizer Investigational Site
Hamburg, 20246
Pfizer Investigational Site
Hanover, Lower Saxony, 30625
Pfizer Investigational Site
Heidelberg, 69123
Pfizer Investigational Site
Homburg, 66424
Pfizer Investigational Site
Klipphausen, 01665
Pfizer Investigational Site
Leipzig, 04103
Pfizer Investigational Site
Lübeck, 23538
Pfizer Investigational Site
München, 80336
Pfizer Investigational Site
München, 80337
Pfizer Investigational Site
Münster, D-48143
Pfizer Investigational Site
Potsdam, 14467
Pfizer Investigational Site
Rostock, Mecklenburg-Vorpommern, 18055
Pfizer Investigational Site
Schwerin, 19049
Pfizer Investigational Site
Stadtroda, Thuringia, 07646
Pfizer Investigational Site
Ulm, 89081
Pfizer Investigational Site
Wiesbaden, Germany, 65191
About this study
Official title: Pharmacovigilance Evaluation Of Refacto In Usual Care Settings
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.
Read the detailed description
Non-interventional study: subjects to be selected according to the usual clinical practice of their physician
- Conditions
- Hemophilia A
- Being tested
- Moroctocog alfa
- Keywords
- Hemophilia A
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Moroctocog alfaDrug
Also called ReFacto
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.
| Study group | Role | What they receive |
|---|---|---|
| A | Patients with Hemophilia A Receives: Drug: Moroctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Number of Bleeding Episodes Per Patient Year
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.
Measured: Baseline up to a mean duration of 54 months
Mean Number of Bleeding-related Exposure Days Per Patient Year
Exposure days are the number of days of treatment with ReFacto.
Measured: Baseline up to a mean duration of 54 months
Mean Number of Exposure Days Per Patient Year
Exposure days are the number of days of treatment with ReFacto.
Measured: Baseline up to a mean duration of 54 months
Secondary outcomes (11)
Number of Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants With de Novo Inhibitor Formation
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.
Measured: Baseline up to a mean duration of 54 months
Mean Annual ReFacto Consumption Per Patient Year
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Satisfaction With Treatment Success
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Efficacy
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Patients' Assessment of Efficacy
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Tolerance
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Patients' Assessment of Tolerance
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Handling of ReFacto
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Patients' Assessment of Handling of ReFacto
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.
Measured: Baseline up to a mean duration of 54 months
Number of Participants for Days of Sick Leave Per Month
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.
Measured: Baseline up to a mean duration of 54 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2005 (estimated)
- Study startJul 1999
- Primary completionJan 2010
- Study completionJan 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 288 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00195442
- Sponsor's protocol ID
- 3082A-100690
- Status last verified
- Feb 2011
- First submitted
- 13 Sep 2005
- Last updated
- 11 Feb 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacovigilance Evaluation Of Refacto In Usual Care Settings
- ClinicalTrials.gov ID
- NCT00195442
- Acronym / sponsor ID
- 3082A-100690
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 288 participants (actual)
- Lead sponsor
- Wyeth is now a wholly owned subsidiary of PfizerIndustry (pharma, biotech, devices)
- Study start
- Jul 1999
- Primary completion
- Jan 2010 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 19 Sep 2005 (estimated)
- Results
- Posted 7 Feb 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | – | Drug: Moroctocog alfaPatients with Hemophilia A |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with hemophilia A
Key inclusion criteria (1 listed)
- Proven diagnosis of Hemophilia A
Key exclusion criteria (1 listed)
- Contraindications according to Summary of Product Characteristics
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Moroctocog alfaAlso: ReFactoPatients 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. | Drug | A |
Primary outcome
Mean Number of Bleeding Episodes Per Patient Year
Time frame: 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
Time frame: 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
Time frame: Baseline up to a mean duration of 54 months
Exposure days are the number of days of treatment with ReFacto.
11 secondary outcomes
Number of Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to a mean duration of 54 months
Number of Participants With de Novo Inhibitor Formation
Time frame: Baseline up to a mean duration of 54 months
Mean Annual ReFacto Consumption Per Patient Year
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Satisfaction With Treatment Success
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Efficacy
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Patients' Assessment of Efficacy
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Tolerance
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Patients' Assessment of Tolerance
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Physicians' Assessment of Handling of ReFacto
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Patients' Assessment of Handling of ReFacto
Time frame: Baseline up to a mean duration of 54 months
Number of Participants for Days of Sick Leave Per Month
Time frame: Baseline up to a mean duration of 54 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
26 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Wyeth is now a wholly owned subsidiary of Pfizer at 26 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Feb 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in Germany, Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Feb 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Number of Bleeding Episodes Per Patient Year (time frame: Baseline up to a mean duration of 54 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2011.