Hematologic DiseasePhase 4NCT00586521

BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)

A Phase 4 study testing Kogenate (BAY14-2222) in people with Hematologic Disease and Hemophilia A, run by Bayer at 11 sites in 5 countries.

Completed

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

Official record

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Trial summary

The purpose of the study is to evaluate the effect of prophylactic treatment on the number of joint bleeds and quality of life in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule design. Read more about this study

Who is it for?: Ages 30 to 45 years, men only
Conditions the study is for: Hematologic Disease, Hemophilia A
What is being tested?: Kogenate (BAY14-2222)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 20 participants
Where: 11 sitesin 5 countries
Study dates: Feb 2006to Mar 2008
Phase: Phase 4
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 30 to 45 years
Men only
No healthy volunteers
Adult

Who may be able to join

Severe hemophilia A (< 1% FVIII)
30-45 years of age
Previously treated subject (> 100 Exposure days to any FVIII)
On-demand therapy with any FVIII

Who cannot join

No history of inhibitor
No planned elective orthopedic surgery during the study duration (13 months)
No severe concomitant disease
No history of anaphylactic or other severe reaction to previous FVIII treatment

Trial locations and contacts

11 sites in 5 countries.

Country

City, postcode or hospital

11 sites in 5 countries

United States3 sites

Study site
Aurora, Colorado, 80045
Study site
Chapel Hill, North Carolina, 27599
Study site
Houston, Texas, 77030

Other trials in United States

Italy3 sites

Study site
Florence, 50134
Study site
Pavia, 27100
Study site
Roma, 00161

Other trials in Italy

Spain2 sites

Study site
Madrid, Madrid, 28046
Study site
Santa Cruz de Tenerife, Santa Cruz de Tenerife, 38009

Other trials in Spain

United Kingdom2 sites

Study site
Cardiff, South Glamorgan, CF14 4XW
Study site
Sheffield, South Yorkshire, S10 2JF

Other trials in United Kingdom

France1 site

Study site
Strasbourg, 67098

Other trials in France

About this study

Official title: A Prospective Controlled Study on the Effect on Bleeding Events and Joint Function in Young Adults With Severe Hemophilia A After a 6 Month Prophylaxis Treatment Compared to on Demand Treatment

In addition, the effect of prophylactic treatment on the joint function, the number of all bleeds, and on the quality of life compared to on-demand treatment and health-economic data will be assessed.

Conditions: Hematologic Disease, Hemophilia A
Being tested: Kogenate (BAY14-2222)
Keywords: Coagulation Disorders
Medical subject headings: Hematologic Diseases, Hemophilia A, Hemostatic Disorders

What participants receive

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

Kogenate (BAY14-2222)Drug
One group two treatment schedules, first on-demand then switch to prophylaxis

Study groups in NCT00586521
Study group	Role	What they receive
rFVIII-FS (octocog-alfa), (Kogenate FS)	Experimental	On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose. Receives: Drug: Kogenate (BAY14-2222)

What is being measured

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

Number of Joint Bleeds
Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6
Measured: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)

Secondary outcomes (3)

Number of All Bleeds
Mean number of all bleeds during Months 8-13 (prophylactic) compared to mean number of all bleeds during Months 1-6 (on-demand)
Measured: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)
Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score
Total score with a range of 0-100, evaluating ankle, knee and elbow, 0 indicates normal function, higher values indicate joint damage
Measured: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)
Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire
Total transformed score with a range of 0-100, higher values indicate better outcome. 41 items in 6 domains: physical functioning; role functioning; worry; consequences; positive affect; treatment concern.
Measured: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)

Timeline

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

Feb 2006Mar 2008

First posted4 Jan 2008 (estimated)
Study startFeb 2006
Primary completionMar 2008
Study completionMar 2008

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 Hematologic DiseaseStudies of the same condition, open and under way.
More trials run by BayerThe 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: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 20 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00586521
Sponsor's protocol ID: 11859
Other IDs: 2005-002757-45, 2005
Status last verified: Oct 2014
First submitted: 21 Dec 2007
Last updated: 3 Nov 2014
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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Study at a glance

From the registry record, last updated by the sponsor on 3 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective Controlled Study on the Effect on Bleeding Events and Joint Function in Young Adults With Severe Hemophilia A After a 6 Month Prophylaxis Treatment Compared to on Demand Treatment
ClinicalTrials.gov ID: NCT00586521
Acronym / sponsor ID: 11859
Conditions: Hematologic Disease, Hemophilia A
Keywords: Coagulation Disorders
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 20 participants (actual)
Lead sponsor: BayerIndustry (pharma, biotech, devices)
Study start: Feb 2006
Primary completion: Mar 2008 (actual)
Study completion: Mar 2008 (actual)
First posted: 4 Jan 2008 (estimated)
Results: Posted 19 Nov 2009

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
rFVIII-FS (octocog-alfa), (Kogenate FS)	Experimental	Drug: Kogenate (BAY14-2222)On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.

Who may qualify

Ages: 30 to 45 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (4 listed)

Severe hemophilia A (< 1% FVIII)
30-45 years of age
Previously treated subject (> 100 Exposure days to any FVIII)
On-demand therapy with any FVIII

Key exclusion criteria (4 listed)

No history of inhibitor
No planned elective orthopedic surgery during the study duration (13 months)
No severe concomitant disease
No history of anaphylactic or other severe reaction to previous FVIII treatment

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
Kogenate (BAY14-2222)One group two treatment schedules, first on-demand then switch to prophylaxis	Drug	rFVIII-FS (octocog-alfa), (Kogenate FS)

Primary outcome

Number of Joint Bleeds
Time frame: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)
Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6

3 secondary outcomes

Number of All Bleeds
Time frame: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)
Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score
Time frame: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)
Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire
Time frame: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)

Results and safety

Results posted to the registry on 19 Nov 2009, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
rFVIII-FS (Octocog-alfa), (Kogenate FS)	20	19

Primary outcome: Number of Joint Bleeds

Time frame: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment). Mean (Standard Deviation). Unit: Number of joint bleeds

Measure	rFVIII-FS (Octocog-alfa), (Kogenate FS) n = 20
Months 1-6 (on-demand)	18.5 (11.6)
Months 8-13 (prophylactic)	1.5 (2.1)

rFVIII-FS (Octocog-alfa), (Kogenate FS): p <0.001, Wilcoxon (Mann-Whitney)
rFVIII-FS (Octocog-alfa), (Kogenate FS): p <0.001, paired t-test

Population analysed: intent-to-treat population

3 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
rFVIII-FS (Octocog-alfa), (Kogenate FS)	2 of 20 (10%)	12 of 20 (60%)	Not reported

Limitations and caveats (from the sponsor): The short observation period of 6 months may be responsible for the lack of statistical significance between treatments in the subject's overall assessment of disease-related quality of life; however, a positive trend was noted.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2014.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Click here to find information about studies related to Bayer Healthcare products conducted in EuropeLink 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.

Bayer Study Director study director

Bayer

11 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Kogenate (BAY14-2222) in people with Hematologic Disease and Hemophilia A, run by Bayer at 11 sites in 5 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 4 inclusion and 4 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 3 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States, Italy, Spain, United Kingdom, France. 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.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Nov 2009). 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 Joint Bleeds (time frame: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)). 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 NCT00586521.

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 3 Nov 2014.