Hemophilia APhase 4NCT01810666

Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

A Phase 4 study testing Recombinant Factor VIII (Kogenate FS, BAY14-2222) in people with Hemophilia A, run by Bayer at 5 sites in China.

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

Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A. Read more about this study

Who is it for?: Ages 2 to 16 years, men only
Conditions the study is for: Hemophilia A
What is being tested?: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 30 participants
Where: 5 sitesChina
Study dates: Mar 2013to Jan 2014
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 2 to 16 years
Men only
No healthy volunteers
Child

Who may be able to join

Male, aged 2-16yrs
Severe hemophilia A (<1% FVIII:C [Blood Clotting Factor VIII:C] )
Minimum of at least 50 documented ED (exposure day) prior to enrolment
No measurable inhibitor activity at baseline and history of FVIII inhibitor antibody formation
Parents or legal guardians document, sign, and date informed consent

Who cannot join

Another bleeding disease that is different from hemophilia A
Known hypersensitivity to the active substance, mouse or hamster protein
Thrombocytopenia (platelet count <100 000/mm3) based on previous medical records

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

China5 sites

Study site
Beijing, 100730
Study site
Beijing
Study site
Guangzhou, Guangdong, 510515
Study site
Tianjin
Study site
Wuhan, Hubei, 430022

Other trials in China

About this study

Official title: Routine Prophylaxis Treatment Versus On-demand Treatment for Children With Severe Hemophilia A: Comparison of All Bleeding Events in Chinese Hemophilia Patients

Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.

Conditions: Hemophilia A
Being tested: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Medical subject headings: Hemophilia A

What participants receive

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

Recombinant Factor VIII (Kogenate FS, BAY14-2222)Biological
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.

Study groups in NCT01810666
Study group	Role	What they receive
Recombinant Factor VIII	Experimental	Not described Receives: Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

What is being measured

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

Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period
Annualized bleedings period 1 minus period 2 ITT analysis set.
Measured: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)

Secondary outcomes (2)

Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period
Annualized joint bleedings period 1 minus period 2 ITT analysis set.
Measured: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period
Hemophilia Joint Health Score(HJHS) ranges from 0 to 124. Higher values in the HJHS represent worse situation for the subject. 2-sided Hodges Lehmann estimates for median 95% CI HJHS values difference of changes ITT analysis set.
Measured: From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)

Timeline

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

Mar 2013Jan 2014

First posted13 Mar 2013 (estimated)
Study startMar 2013
Primary completionJan 2014
Study completionJan 2014

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 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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 30 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01810666
Sponsor's protocol ID: 16287
Other IDs: 2014-001362-10, 2014
Status last verified: Apr 2015
First submitted: 12 Mar 2013
Last updated: 19 May 2015
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

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

All Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 19 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Routine Prophylaxis Treatment Versus On-demand Treatment for Children With Severe Hemophilia A: Comparison of All Bleeding Events in Chinese Hemophilia Patients
ClinicalTrials.gov ID: NCT01810666
Acronym / sponsor ID: 16287
Conditions: Hemophilia A
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 30 participants (actual)
Lead sponsor: BayerIndustry (pharma, biotech, devices)
Study start: Mar 2013
Primary completion: Jan 2014 (actual)
Study completion: Jan 2014 (actual)
First posted: 13 Mar 2013 (estimated)
Results: Posted 5 Jan 2015

Clinical summary

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

Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Recombinant Factor VIII	Experimental	Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Who may qualify

Ages: 2 to 16 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

Male, aged 2-16yrs
Severe hemophilia A (<1% FVIII:C [Blood Clotting Factor VIII:C] )
Minimum of at least 50 documented ED (exposure day) prior to enrolment
No measurable inhibitor activity at baseline and history of FVIII inhibitor antibody formation
Parents or legal guardians document, sign, and date informed consent

Key exclusion criteria (3 listed)

Another bleeding disease that is different from hemophilia A
Known hypersensitivity to the active substance, mouse or hamster protein
Thrombocytopenia (platelet count <100 000/mm3) based on previous medical records

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
Recombinant Factor VIII (Kogenate FS, BAY14-2222)Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.	Biological	Recombinant Factor VIII

Primary outcome

Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period
Time frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
Annualized bleedings period 1 minus period 2 ITT analysis set.

2 secondary outcomes

Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period
Time frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period
Time frame: From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)

Results and safety

Results posted to the registry on 5 Jan 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Rec. Factor VIII On-demand Followed by Prophylaxis	30	30

Primary outcome: Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period

Time frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment). Median (95% Confidence Interval). Unit: Bleeds

Measure	Rec. Factor VIII On-demand Followed by Prophylaxis n = 30
Value	56.00 (48.80 to 62.54)

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first treatment through 3 days after the last dose of study drug, up to 6 months. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Rec. Factor VIII On-demand Followed by Prophylaxis	0 of 30 (0%)	2 of 30 (6.7%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2015.
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.

Related trials for Hemophilia A

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

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

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Recombinant Factor VIII (Kogenate FS, BAY14-2222) in people with Hemophilia A, run by Bayer at 5 sites in China.

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 5 inclusion and 3 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 19 May 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in China. 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 5 Jan 2015). 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?

Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period (time frame: Week 1-12 (on-demand treatment) and 13-24 (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 NCT01810666.

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 19 May 2015.