Hemophilia ANCT01790828

Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A

An observational study of Hemophilia A, run by Pfizer at 4 sites in South Korea.

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

This study is to describe the safety and efficacy of Xyntha® during the usual care setting. Read more about this study

Who is it for?: Any age
Conditions the study is for: Hemophilia A
What is being tested?: Xyntha : coagulation factor IIIV (recombinant)
How many people: 42 participants
Where: 4 sitesSouth Korea
Study dates: Feb 2014to Aug 2014
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: Hemophilia A

Who may be able to join

Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement).
Pediatric and adult patients who have been treated with Xyntha for Hemophilia A from first approved date by KFDA, or who are planned to be newly prescribed Xyntha.

Who cannot join

Patients with a known history of hypersensitivity to original or reformulated Xyntha or any component of the product.
Patients with a known history of hypersensitivity to hamster protein.
Patients participating in an interventional trial of any investigational drug or device.

Trial locations and contacts

4 sites in 1 country.

South Korea4 sites

Pusan National University Hospital
Busan, 632-739
Yonsei Rehabilitation Clinic
Guro-gu, Seoul, 152-834
Kyung Hee University Hospital at Gangdong
Seoul, 134-727
Kim Hugh Chul Internal Medicine Clinic
Songpa-gu, Seoul, 138-879

Other trials in South Korea

About this study

Official title: Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha 'Registered' In Subjects With Hemophilia A

This study is to describe the safety and efficacy of Xyntha® during the usual care setting.

Read the detailed description

non probability sample

Conditions: Hemophilia A
Being tested: Xyntha : coagulation factor IIIV (recombinant)
Medical subject headings: Hemophilia A

What participants receive

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

Xyntha : coagulation factor IIIV (recombinant)Drug
Also called Xyntha, MOROCTOCOG ALFA
Xyntha will be administered according to physician's discretion.

Study groups in NCT01790828
Study group	Role	What they receive
Xyntha group		Xyntha will be administered according to physician's discretion. Receives: Drug: Xyntha : coagulation factor IIIV (recombinant)

What is being measured

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

Percentage of Participants by Family History of Factor VIII Inhibitor
Measured: 4 years

Secondary outcomes (19)

Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy (All Participants)
Measured: 4 years
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Less Than (<)18 Years of Age
Measured: 4 years
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Greater Than or Equal to (≥) 18 Years of Age
Measured: 4 years
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants)
Response categories were excellent, good, moderate, or no response.
Measured: 4 years
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age
Response categories were excellent, good, moderate, or no response.
Measured: 4 years
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age
Response categories were excellent, good, moderate, and no response.
Measured: 4 years
Number of Participants With Less-Than-Expected Therapeutic Effect (LETE) for On-Demand Therapy
Less than expected therapeutic effect was defined as a 'no response' rating after each of two successive infusions less than or equl to (≤) 24 hours of on-demand therapy.
Measured: 4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy (All Participants)
Measured: 4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants <18 Years of Age
Measured: 4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants ≥18 Years of Age
Measured: 4 years
Percentage of Participants Experiencing Hemorrhages During Prophylaxis
Measured: 4 years
Annualized Bleeding Rates During Prophylaxis
Annualized bleeding rate defined as total number of breakthrough bleeds within 48 hours (for prophylaxis purpose) divided by (/) [(total period of date of bleeding)/365.25)]
Measured: 4 years
Number of Participants With LETE Bleeds Within 48 Hours of a Preventive/Prophylaxis Dose of Xyntha
Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleed wtihin 48 hours of prophylaxis infusion.
Measured: 4 years
Average Infusion Dose During Prophylaxis (All Participants)
Measured: 4 years
Average Infusion Dose During Prophylaxis in Participants <18 Years of Age
Measured: 4 years
Average Infusion Dose During Prophylaxis in Participants ≥18 Years of Age
Measured: 4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis (All Participants)
Measured: 4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants <18 Years of Age
Measured: 4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants ≥18 Years of Age
Measured: 4 years

Timeline

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

Feb 2014Aug 2014

First posted13 Feb 2013 (estimated)
Study startFeb 2014
Primary completionAug 2014
Study completionAug 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 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
Time perspective: Prospective
Enrolment: 42 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01790828
Sponsor's protocol ID: B1831078
Status last verified: Jul 2015
First submitted: 11 Feb 2013
Last updated: 17 Aug 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 17 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha 'Registered' In Subjects With Hemophilia A
ClinicalTrials.gov ID: NCT01790828
Acronym / sponsor ID: B1831078
Conditions: Hemophilia A
Study type: Observational
Recruitment status: Completed
Enrolment: 42 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Feb 2014
Primary completion: Aug 2014 (actual)
Study completion: Aug 2014 (actual)
First posted: 13 Feb 2013 (estimated)
Results: Posted 17 Aug 2015

Clinical summary

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

This study is to describe the safety and efficacy of Xyntha® during the usual care setting.

Detailed description

non probability sample

Study design

Time perspective: Prospective
Number of arms: 1

Arm	Type	Receives
Xyntha group	–	Drug: Xyntha : coagulation factor IIIV (recombinant)Xyntha will be administered according to physician's discretion.

Who may qualify

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

Key inclusion criteria (2 listed)

Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement).
Pediatric and adult patients who have been treated with Xyntha for Hemophilia A from first approved date by KFDA, or who are planned to be newly prescribed Xyntha.

Key exclusion criteria (3 listed)

Patients with a known history of hypersensitivity to original or reformulated Xyntha or any component of the product.
Patients with a known history of hypersensitivity to hamster protein.
Patients participating in an interventional trial of any investigational drug or device.

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
Xyntha : coagulation factor IIIV (recombinant)Also: Xyntha, MOROCTOCOG ALFAXyntha will be administered according to physician's discretion.	Drug	Xyntha group

Primary outcome

Percentage of Participants by Family History of Factor VIII Inhibitor
Time frame: 4 years

19 secondary outcomes

Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy (All Participants)
Time frame: 4 years
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Less Than (<)18 Years of Age
Time frame: 4 years
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Greater Than or Equal to (≥) 18 Years of Age
Time frame: 4 years
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants)
Time frame: 4 years
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age
Time frame: 4 years
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age
Time frame: 4 years
Number of Participants With Less-Than-Expected Therapeutic Effect (LETE) for On-Demand Therapy
Time frame: 4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy (All Participants)
Time frame: 4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants <18 Years of Age
Time frame: 4 years
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants ≥18 Years of Age
Time frame: 4 years
Percentage of Participants Experiencing Hemorrhages During Prophylaxis
Time frame: 4 years
Annualized Bleeding Rates During Prophylaxis
Time frame: 4 years
Number of Participants With LETE Bleeds Within 48 Hours of a Preventive/Prophylaxis Dose of Xyntha
Time frame: 4 years
Average Infusion Dose During Prophylaxis (All Participants)
Time frame: 4 years
Average Infusion Dose During Prophylaxis in Participants <18 Years of Age
Time frame: 4 years
Average Infusion Dose During Prophylaxis in Participants ≥18 Years of Age
Time frame: 4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis (All Participants)
Time frame: 4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants <18 Years of Age
Time frame: 4 years
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants ≥18 Years of Age
Time frame: 4 years

Results and safety

Results posted to the registry on 17 Aug 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
Xyntha	42	8

Primary outcome: Percentage of Participants by Family History of Factor VIII Inhibitor

Time frame: 4 years. Number. Unit: percentage of participants

Measure	Xyntha n = 42
Yes	0
No	100.0

Population analysed: All enrolled participants.

19 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were collected up to 6 months after the first administration of Xyntha.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Xyntha	0 of 42 (0%)	10 of 42 (23.8%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2015.
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

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.

Pfizer CT.gov Call Center study director

Pfizer

4 sites in 1 country: 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 4 sites in South Korea.

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 2 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 17 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in South Korea. 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 Aug 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?

Percentage of Participants by Family History of Factor VIII Inhibitor (time frame: 4 years). 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 NCT01790828.

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 Aug 2015.