Hemophilia ANCT00895037

Study Evaluating Pharmacovigilance Of Refacto AF

An observational study of Hemophilia A, run by Pfizer at 24 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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 more about this study

Who is it for?: Any age
Conditions the study is for: Hemophilia A
What is being tested?: ReFacto AF (Moroctocog alfa)
Type of treatment: Injection or infusion
How many people: 101 participants
Where: 24 sitesin 2 countries
Study dates: 17 Jul 2009to 19 Oct 2016
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
All sexes
No healthy volunteers
Child, Adult, Older adult

Study population: Patients with hemophilia A

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

24 sites in 2 countries.

Country

City, postcode or hospital

24 sites in 2 countries

Germany21 sites

Vivantes Klinikum im Friedrichshain
Berlin, 10249
Klinikum Bremen Mitte
Bremen, 28205
CRC Coagulation Research Centre GmbH
Duisburg, 47051
Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie
Düsseldorf, 40225
Universitaetsklinikum Duesseldorf
Düsseldorf, 40225
Praxis zur Diagnostik und Therapie Von Blutgerinnungstoerungen
Frankfurt A. M., 60596
Praxis fur Kinder- und Jugendmedizin
Grünwald, 82031
Werlhof-Institut für Haemostaseologie GmbH
Hanover, Lower Saxony, 30159
Medizinische Hochschule Hannover
Hanover, 30625
SRH Kurpfalzkrankenhaus Heidelberg
Heidelberg, 69123
Donaustrasse 78
Memmingen, 87700
Praxis Dr. Autenrieth
Metzingen, 72555
Hämophilie-Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546
Sonnengesundheitszentrum
München, Bavaria, 80336
Institut for Thrombophilia and Hemastaseologie
Münster, 48143
Stauferklinikum Schwaebisch Gmuend
Mutlangen, 73557
Universität Regensburg
Regensburg, 93042
Asklepios Fachklinikum Stadtroda GmbH
Stadtroda, 07646
Klinikum Stuttgart
Stuttgart, 70176
Universitaetsklinik fuer Kinder- und Jugendmedizin
Tübingen, 72076
Stiftung Deutsche Klinik fur Diagnostik GmbH
Wiesbaden, 65191

Other trials in Germany

Austria3 sites

LKH - Univ. Klinikum Graz,Abt. fur Hamatologie
Graz, 8036
Allgemeines Krankenhaus Linz, Kinderklinik
Linz, 4020
Universitaetsklinik fuer Innere Medizin 1
Vienna, 1090

Other trials in Austria

About this study

Official title: Pharmacovigilance Evaluation Of Refacto Af (Registered) In Germany And Austria

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

Conditions: Hemophilia A
Being tested: ReFacto AF (Moroctocog alfa)
Medical subject headings: Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ReFacto AF (Moroctocog alfa)Drug
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

Study groups in NCT00895037
Study group	Role	What they receive
1		Patients treated with Refacto AF Receives: Drug: ReFacto AF (Moroctocog alfa)

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline until last visit (up to 87 months)
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.
Measured: Baseline until last visit (up to 87 months)
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.
Measured: Baseline until last visit (up to 87 months)
Mean Total Number of Bleeding Episodes in Participants
Participants documented all bleeding episodes in a diary during the study.
Measured: Baseline until last visit (up to 87 months)

Secondary outcomes (4)

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.
Measured: Baseline until last visit (up to 87 months)
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.
Measured: Baseline until last visit (up to 87 months)
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.
Measured: End of study visit (any time up to 87 months)
Investigator Assessment of Treatment Satisfaction of Participants
Investigator assessed the treatment satisfaction of participants and categorized as very satisfied, satisfied, unsatisfied, or very unsatisfied.
Measured: End of study visit (any time up to 87 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

17 Jul 200919 Oct 2016

First posted7 May 2009 (estimated)
Study start17 Jul 2009
Primary completion19 Oct 2016
Study completion19 Oct 2016

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.
More trials run by PfizerThe sponsor's other registered studies.
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: 101 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00895037
Sponsor's protocol ID: 3082B2-4420
Other IDs: B1831016
Status last verified: Sep 2017
First submitted: 1 May 2009
Last updated: 17 Jul 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
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All Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Pharmacovigilance Evaluation Of Refacto Af (Registered) In Germany And Austria
ClinicalTrials.gov ID: NCT00895037
Acronym / sponsor ID: 3082B2-4420
Conditions: Hemophilia A
Study type: Observational
Recruitment status: Completed
Enrolment: 101 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: 17 Jul 2009 (actual)
Primary completion: 19 Oct 2016 (actual)
Study completion: 19 Oct 2016 (actual)
First posted: 7 May 2009 (estimated)
Results: Posted 17 Jul 2018

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 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.

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
1	–	Drug: ReFacto AF (Moroctocog alfa)Patients treated with Refacto AF

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Patients with hemophilia A

Key inclusion criteria (1 listed)

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

Key exclusion criteria (1 listed)

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

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
ReFacto 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	Drug	1

Primary outcome

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline until last visit (up to 87 months)
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.
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline until last visit (up to 87 months)
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.
Number of Participants With Factor VIII (FVIII) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
Time frame: Baseline until last visit (up to 87 months)
FVIII inhibitor development was defined as measured inhibitor titer of greater than (>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay.
Mean Total Number of Bleeding Episodes in Participants
Time frame: Baseline until last visit (up to 87 months)
Participants documented all bleeding episodes in a diary during the study.

4 secondary outcomes

Mean Total Number of Bleeding Episodes Per Year in Participants
Time frame: Baseline until last visit (up to 87 months)
Number of Participants With Change From Baseline Status in Days Missed From School or Work
Time frame: Baseline until last visit (up to 87 months)
Participant Assessment of Satisfaction With Treatment Handling
Time frame: End of study visit (any time up to 87 months)
Investigator Assessment of Treatment Satisfaction of Participants
Time frame: End of study visit (any time up to 87 months)

Results and safety

Results posted to the registry on 17 Jul 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
ReFacto AF: On Demand Treatment	22	22
ReFacto AF: Prophylaxis Treatment	77	75
ReFacto AF: Intermediate Prophylaxis Treatment	2	1

Primary outcome: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Baseline until last visit (up to 87 months). Number. Unit: participants

Measure	ReFacto AF: On Demand Treatment n = 22	ReFacto AF: Prophylaxis Treatment n = 77	ReFacto AF: Intermediate Prophylaxis Treatment n = 2
AEs	15	59	2
SAEs	7	25	2

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

Primary outcome: Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Baseline until last visit (up to 87 months). Number. Unit: participants

Measure	ReFacto AF: On Demand Treatment n = 22	ReFacto AF: Prophylaxis Treatment n = 77	ReFacto AF: Intermediate Prophylaxis Treatment n = 2
AEs	2	7	0
SAEs	0	0	0

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

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

Time frame: Baseline until last visit (up to 87 months). Number. Unit: participants

Measure	ReFacto AF: On Demand Treatment n = 22	ReFacto AF: Prophylaxis Treatment n = 77	ReFacto AF: Intermediate Prophylaxis Treatment n = 2
Value	1	2	0

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

Primary outcome: Mean Total Number of Bleeding Episodes in Participants

Time frame: Baseline until last visit (up to 87 months). Mean (Standard Deviation). Unit: bleeding episodes

Measure	ReFacto AF: On Demand Treatment n = 17	ReFacto AF: Prophylaxis Treatment n = 74	ReFacto AF: Intermediate Prophylaxis Treatment n = 2
Value	71.2 (73.3)	15.4 (14.7)	68.5 (31.8)

Population analysed: Effectiveness analysis set =participants registered in study and had at least 1 dose of ReFacto AF documented by participants diary entries. Participant's registration included confirmation of obtained participant's informed consent from the physician. Here, 'N' (number of participants analyzed) = participants evaluable for this outcome measure.

4 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
ReFacto AF: On Demand Treatment	7 of 22 (31.8%)	14 of 22 (63.6%)	Not reported
ReFacto AF: Prophylaxis Treatment	25 of 77 (32.5%)	56 of 77 (72.7%)	Not reported
ReFacto AF: Intermediate Prophylaxis Treatment	2 of 2 (100%)	2 of 2 (100%)	Not reported

Limitations and caveats (from the sponsor): Prioritization of outcome measures as primary and secondary was based on the study team's discretion, as it was not specified in source documents.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2018.
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.

Related trials for Hemophilia A

Not yet recruitingPhase 1
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A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
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RecruitingPhase 3
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A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
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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

24 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 Pfizer at 24 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 17 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 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 17 Jul 2018). 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?

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Baseline until last visit (up to 87 months)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00895037.

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 17 Jul 2018.