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CompletedNCT00895037Updated Jul 17, 2018Results posted

Study Evaluating Pharmacovigilance Of Refacto AF

An observational study in Hemophilia A, sponsored by Pfizer. Completed at 24 sites in 2 countries. Per ClinicalTrials.gov, last updated 2018-07-17.

Sponsored by Pfizer · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
101
Sex
All
01

Study summary

The purpose of this observational study is to describe the incidence of adverse events among patients treated with Refacto AF in usual health care settings in Germany and Austria.

Read the detailed description

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

02

Conditions studied

  • Hemophilia A

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03

In context

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 101 is above the median of 80 across 314 observational studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.

Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Patients with hemophilia A

Inclusion criteria

  • Patients with hemophilia A of any severity already receiving or starting treatment with ReFacto AF.

Exclusion criteria

Exclusion Criteria:

  • Patients with Hemophilia A treated with a product other than Refacto AF.
05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
101 participants (actual)
Patient registry
No

Groups and cohorts

  • 1

    Patients treated with Refacto AF

    Drug: ReFacto AF (Moroctocog alfa)

Interventions

  • DrugReFacto AF (Moroctocog alfa)

    Patients will be treated with intravenous infusion of ReFacto AF per dosing and frequency as prescribed by the subjects' treating physician. ReFacto AF® will be prescribed in the context of routine clinical practice in Germany and Austria, respectively

06

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability or incapacity; cancer; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to last visit (up to 87 months) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.

    Time frame: Baseline until last visit (up to 87 months)

  2. Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; cancer; congenital anomaly. AEs included both serious and non-serious adverse events. Relatedness of AEs with Refacto AF was assessed by the investigator.

    Time frame: Baseline until last visit (up to 87 months)

  3. Number of Participants With Factor VIII (FVIII) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay

    FVIII inhibitor development was defined as measured inhibitor titer of greater than (\>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay.

    Time frame: Baseline until last visit (up to 87 months)

  4. Mean Total Number of Bleeding Episodes in Participants

    Participants documented all bleeding episodes in a diary during the study.

    Time frame: Baseline until last visit (up to 87 months)

Secondary outcomes

  1. Mean Total Number of Bleeding Episodes Per Year in Participants

    Participants documented all bleeding episodes in a diary during the study. Mean total number of bleeding episodes per year was calculated as: mean total number of bleeding episodes divided by duration of observation period (in years) for bleeding documentation.

    Time frame: Baseline until last visit (up to 87 months)

  2. Number of Participants With Change From Baseline Status in Days Missed From School or Work

    Change from baseline status in days missed from school or work was categorized in 3 categories: Improvement, unchanged and worsening. Improvement defined as a decrease in number of days missed by participants from school/work as compared to baseline; worsening was defined as an increase in number of days missed by participants from school/work as compared to baseline; unchanged was defined as no change in number of days missed by participants from school/work as compared to baseline. In this outcome measure, number of participants with change from baseline status (as improved, worsen, unchanged) in days missed from school/work were reported.

    Time frame: Baseline until last visit (up to 87 months)

  3. Participant Assessment of Satisfaction With Treatment Handling

    Participants evaluated their satisfaction with handing (administration) of Refacto AF and rated it in 4 categories as: very satisfied, satisfied, unsatisfied and very unsatisfied.

    Time frame: End of study visit (any time up to 87 months)

  4. Investigator Assessment of Treatment Satisfaction of Participants

    Investigator assessed the treatment satisfaction of participants and categorized as very satisfied, satisfied, unsatisfied, or very unsatisfied.

    Time frame: End of study visit (any time up to 87 months)

07

Results

Posted Jul 17, 2018
Limitations and caveats
Prioritization of outcome measures as primary and secondary was based on the study team's discretion, as it was not specified in source documents.

Participant flow

Participant flow — Overall Study
MilestoneReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Started22772
Completed22751
Not completed021
Withdrew: Death021

Outcome measures

PrimaryNumber of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability or incapacity; cancer; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to last visit (up to 87 months) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.

Time frame:
Baseline until last visit (up to 87 months)
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
participantsReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
AEs15592
SAEs7252
PrimaryNumber of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)

Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; cancer; congenital anomaly. AEs included both serious and non-serious adverse events. Relatedness of AEs with Refacto AF was assessed by the investigator.

Time frame:
Baseline until last visit (up to 87 months)
Reported as:
Number · participants
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
participantsReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
AEs270
SAEs000
PrimaryNumber of Participants With Factor VIII (FVIII) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay

FVIII inhibitor development was defined as measured inhibitor titer of greater than (\>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay.

Time frame:
Baseline until last visit (up to 87 months)
Reported as:
Number · participants
Number of Participants With Factor VIII (FVIII) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
participantsReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Number of Participants With Factor VIII (FVIII) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay120
PrimaryMean Total Number of Bleeding Episodes in Participants

Participants documented all bleeding episodes in a diary during the study.

Time frame:
Baseline until last visit (up to 87 months)
Reported as:
Mean · bleeding episodes
Mean Total Number of Bleeding Episodes in Participants
bleeding episodesReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Mean Total Number of Bleeding Episodes in Participants71.2 ± 73.315.4 ± 14.768.5 ± 31.8
SecondaryMean Total Number of Bleeding Episodes Per Year in Participants

Participants documented all bleeding episodes in a diary during the study. Mean total number of bleeding episodes per year was calculated as: mean total number of bleeding episodes divided by duration of observation period (in years) for bleeding documentation.

Time frame:
Baseline until last visit (up to 87 months)
Reported as:
Mean · bleeding episodes per year
Mean Total Number of Bleeding Episodes Per Year in Participants
bleeding episodes per yearReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Mean Total Number of Bleeding Episodes Per Year in Participants18.6 ± 19.65.1 ± 5.813.4 ± 0.5
SecondaryNumber of Participants With Change From Baseline Status in Days Missed From School or Work

Change from baseline status in days missed from school or work was categorized in 3 categories: Improvement, unchanged and worsening. Improvement defined as a decrease in number of days missed by participants from school/work as compared to baseline; worsening was defined as an increase in number of days missed by participants from school/work as compared to baseline; unchanged was defined as no change in number of days missed by participants from school/work as compared to baseline. In this outcome measure, number of participants with change from baseline status (as improved, worsen, unchanged) in days missed from school/work were reported.

Time frame:
Baseline until last visit (up to 87 months)
Reported as:
Number · participants
Number of Participants With Change From Baseline Status in Days Missed From School or Work
participantsReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis Treatment
Improvement425
Unchanged429
Worsening36
SecondaryParticipant Assessment of Satisfaction With Treatment Handling

Participants evaluated their satisfaction with handing (administration) of Refacto AF and rated it in 4 categories as: very satisfied, satisfied, unsatisfied and very unsatisfied.

Time frame:
End of study visit (any time up to 87 months)
Reported as:
Number · participants
Participant Assessment of Satisfaction With Treatment Handling
participantsReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Very satisfied13420
Satisfied5282
Unsatisfied040
Very unsatisfied000
SecondaryInvestigator Assessment of Treatment Satisfaction of Participants

Investigator assessed the treatment satisfaction of participants and categorized as very satisfied, satisfied, unsatisfied, or very unsatisfied.

Time frame:
End of study visit (any time up to 87 months)
Reported as:
Number · participants
Investigator Assessment of Treatment Satisfaction of Participants
participantsReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Very satisfied13440
Satisfied4292
Unsatisfied010
Very unsatisfied000

Adverse events

Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
ReFacto AF: On Demand Treatment—7/22 (31.8%)14/22 (63.6%)
ReFacto AF: Prophylaxis Treatment—25/77 (32.5%)56/77 (72.7%)
ReFacto AF: Intermediate Prophylaxis Treatment—2/2 (100%)2/2 (100%)
Most frequent serious events
Showing 10 of 55
Most frequent serious events
EventReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Gastric polypsGastrointestinal disorders0/220/771/2
Oesophageal polypGastrointestinal disorders0/220/771/2
Upper gastrointestinal haemorrhageGastrointestinal disorders0/220/771/2
Varices oesophagealGastrointestinal disorders0/220/771/2
VomitingGastrointestinal disorders0/221/771/2
Disease progressionGeneral disorders0/220/771/2
Hepatic cirrhosisHepatobiliary disorders0/221/771/2
Haemophilic arthropathyMusculoskeletal and connective tissue disorders1/220/771/2
OsteonecrosisMusculoskeletal and connective tissue disorders0/220/771/2
Basal cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/220/771/2
Most frequent other events
Showing 10 of 171
Most frequent other events
EventReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis Treatment
Abdominal distensionGastrointestinal disorders0/220/771/2
Abdominal tendernessGastrointestinal disorders0/220/771/2
Varices oesophagealGastrointestinal disorders0/220/771/2
VomitingGastrointestinal disorders0/220/771/2
Peripheral swellingGeneral disorders0/220/771/2
Hepatic cirrhosisHepatobiliary disorders0/220/771/2
Localised infectionInfections and infestations0/220/771/2
Viral upper respiratory tract infectionInfections and infestations1/224/771/2
Post procedural haemorrhageInjury, poisoning and procedural complications0/220/771/2
Decreased appetiteMetabolism and nutrition disorders0/220/771/2

Baseline characteristics

Safety analysis set included all participants with informed consent and treated with at least 1 dose of ReFacto AF.

Age, Continuous
Age, Continuous(Years)ReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis TreatmentTotal
Mean34.0 ± 20.218.5 ± 12.462.0 ± 7.122.7 ± 16.6
Sex: Female, Male
Sex: Female, Male(Participants)ReFacto AF: On Demand TreatmentReFacto AF: Prophylaxis TreatmentReFacto AF: Intermediate Prophylaxis TreatmentTotal
Female0000
Male22772101
08

Study locations

24 sites
  • LKH - Univ. Klinikum Graz,Abt. fur Hamatologie
    Graz, 8036, Austria
  • Allgemeines Krankenhaus Linz, Kinderklinik
    Linz, 4020, Austria
  • Universitaetsklinik fuer Innere Medizin 1
    Wien, 1090, Austria
  • Sonnengesundheitszentrum
    München, Bayern 80336, Germany
  • Werlhof-Institut für Haemostaseologie GmbH
    Hannover, Niedersachsen 30159, Germany
  • Vivantes Klinikum im Friedrichshain
    Berlin, 10249, Germany
  • Klinikum Bremen Mitte
    Bremen, 28205, Germany
  • Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie
    Duesseldorf, 40225, Germany
  • Universitaetsklinikum Duesseldorf
    Duesseldorf, 40225, Germany
  • CRC Coagulation Research Centre GmbH
    Duisburg, 47051, Germany
  • Praxis zur Diagnostik und Therapie Von Blutgerinnungstoerungen
    Frankfurt a. M., 60596, Germany
  • Praxis fur Kinder- und Jugendmedizin
    Grunwald, 82031, Germany
  • Medizinische Hochschule Hannover
    Hannover, 30625, Germany
  • SRH Kurpfalzkrankenhaus Heidelberg
    Heidelberg, 69123, Germany
  • Donaustrasse 78
    Memmingen, 87700, Germany
  • Praxis Dr. Autenrieth
    Metzingen, 72555, Germany
  • Institut for Thrombophilia and Hemastaseologie
    Muenster, 48143, Germany
  • Stauferklinikum Schwaebisch Gmuend
    Mutlangen, 73557, Germany
  • Hämophilie-Zentrum Rhein Main GmbH
    Mörfelden-Walldorf, 64546, Germany
  • Universität Regensburg
    Regensburg, 93042, Germany
  • Asklepios Fachklinikum Stadtroda GmbH
    Stadtroda, 07646, Germany
  • Klinikum Stuttgart
    Stuttgart, 70176, Germany
  • Universitaetsklinik fuer Kinder- und Jugendmedizin
    Tuebingen, 72076, Germany
  • Stiftung Deutsche Klinik fur Diagnostik GmbH
    Wiesbaden, 65191, Germany
09

References and documents

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 17, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00895037
Lead sponsor
Pfizer
Responsible party
Sponsor
First posted
May 7, 2009
Start date
Jul 17, 2009
Primary completion
Oct 19, 2016
Completion
Oct 19, 2016
Results posted
Jul 17, 2018
Last update
Jul 17, 2018

Study contacts

Pfizer CT.gov Call Center
study director · Pfizer

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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