Hemophilia APhase 3NCT00868530

Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

A Phase 3 study testing Xyntha in people with Hemophilia A, run by Pfizer at 6 sites in China.

Completed

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

Trial summary

This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects. Read more about this study

Who is it for?: Ages 6 years and older
Conditions the study is for: Hemophilia A
What is being tested?: Xyntha
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 53 participants
Where: 6 sitesChina
Study dates: Sep 2008to Dec 2009
Phase: Phase 3
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 6 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia A (FVIII activity: more than 5%, 1-5%, or less than 1%, respectively)
Subjects with previous exposure to FVIII replacement therapy
If human immunodeficiency virus (HIV) positive, documented cluster of differentiation (CD4) count more than 200/µL within 6 months of study entry

Who cannot join

Diagnosed with any bleeding disorder in addition to hemophilia A
Current FVIII inhibitor or history of FVIII inhibitor (defined as positive result of the reporting laboratory)
Subject has no history of exposure to FVIII products (previously untreated patient [PUP])
Subject is currently utilizing primary FVIII prophylaxis
Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry
Treated with immunomodulatory therapy within 30 days prior to study entry or planned use for the duration of their study participation

Show all 14Show fewer

Participated in another investigational drug or device study within 30 days prior to study entry or planned participation for the duration of their study participation
Subjects with a known hypersensitivity to hamster protein
Significant hepatic or renal impairment (alanine aminotransferase [ALT] and aspartate aminotransferase [AST] >5 x upper limit of normal [ULN], bilirubin >2 mg/dL or serum creatinine >1.25 x ULN)
Prothrombin Time >1.5 x ULN
Platelet count <80,000 / µL
Pregnant or breastfeeding women
Unwilling or unable to follow the terms of the protocol
Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation, in the opinion of the Investigator or Sponsor

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

China6 sites

Pfizer Investigational Site
Beijing, 100730
Pfizer Investigational Site
Guangzhou, 510515
Pfizer Investigational Site
Hangzhou, Zhejiang, 310003
Pfizer Investigational Site
Heping District, Tianjin Municipality, 300020
Pfizer Investigational Site
Shanghai, 200025
Pfizer Investigational Site
Suzhou, Jiangsu, 215006

Other trials in China

About this study

Official title: An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With Xyntha (B Domain Deleted Recombinant Factor VIII, Albumin Free) In Chinese Subjects With Hemophilia A

This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects.

Conditions: Hemophilia A
Being tested: Xyntha
Keywords: On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese
Medical subject headings: Hemophilia A

What participants receive

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

XynthaBiological
Xyntha for on-demand treatment of bleeding episodes were according to investigator prescription during the 6 months observation period. The recovery assessed by determining the Factor VIII (FVIII) concentration (FVIII:C) levels in individual subjects at the initial and final visits. The dose of Xyntha for recovery assessments is: single 50 IU/kg (±5 IU/kg) IV bolus infusion. All Xyntha administrations occurred in the clinic (hospital).

Study groups in NCT00868530
Study group	Role	What they receive
Xyntha	Experimental	This trial was an open-label and included assessments of safety, clinical efficacy, and Factor VIII (FVIII) recovery in Chinese subjects with hemophilia A. Subjects received on-demand treatments with Xyntha over a 6-month (calendar day) period. Receives: Biological: Xyntha

What is being measured

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

Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Measured: 8 hours post infusion
Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Measured: 24 hours post infusion
Number of Participants With Factor VIII (FVIII) Inhibitor Development
Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by participant exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII infusion, and <= 100 documented Exposure Days (EDs), while Previously Treated Patients (PTPs): those who had received >100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.
Measured: Day 1 and Month 6 or Early Termination Visit

Secondary outcomes (4)

FVIII Recovery : Change From Baseline in FVIII Concentration
FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.
Measured: Day 1 and Month 6 or Early Termination Visit
Number of Participants With Less Than Expected Therapeutic Effect (LETE)
The incidence of LETE, defined for on-demand treatment as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.
Measured: 24 hours after each of 2 successive infusion, up to 6 months
Number of Participants With Thrombosis Allergic-Type Reactions
Measured: Baseline up to 6 months
Number of Participants With Thrombosis
Measured: Baseline up to 6 months

Timeline

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

Sep 2008Dec 2009

First posted25 Mar 2009 (estimated)
Study startSep 2008
Primary completionDec 2009
Study completionDec 2009

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.
Publications (1)Papers the sponsor has linked to this study.
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: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 53 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00868530
Sponsor's protocol ID: 3082B2-3316
Other IDs: B1831015
Status last verified: Jun 2025
First submitted: 19 Mar 2009
Last updated: 12 Jun 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Yang R, Zhao Y, Wang X, Sun J, Wu R, Jin C, Jin J, Wu D, Rendo P, Sun F, Rupon J, Huard F, Korth-Bradley JM, Xu L, Luo B, Liu YC. Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies. J Blood Med. 2020 Nov 25;11:439-448. doi: 10.2147/JBM.S241605. eCollection 2020.
PubMed 33269010

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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The CACHE Study: Coronary Artery Care in HaEmophilia
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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
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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
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Haemophilia A, Haemophilia B

All Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 12 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With Xyntha (B Domain Deleted Recombinant Factor VIII, Albumin Free) In Chinese Subjects With Hemophilia A
ClinicalTrials.gov ID: NCT00868530
Acronym / sponsor ID: 3082B2-3316
Conditions: Hemophilia A
Keywords: On-demand treatment; Hemostatic Efficacy Assessments; Factor VIII recovery; Factor VIII Inhibitor; Chinese
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 53 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Sep 2008
Primary completion: Dec 2009 (actual)
Study completion: Dec 2009 (actual)
First posted: 25 Mar 2009 (estimated)
Results: Posted 8 Feb 2011

Clinical summary

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

This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects.

Study design

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

Arm	Type	Receives
Xyntha	Experimental	Biological: XynthaThis trial was an open-label and included assessments of safety, clinical efficacy, and Factor VIII (FVIII) recovery in Chinese subjects with hemophilia A. Subjects received on-demand treatments with Xyntha over a 6-month (calendar day) period.

Who may qualify

Ages: 6 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia A (FVIII activity: more than 5%, 1-5%, or less than 1%, respectively)
Subjects with previous exposure to FVIII replacement therapy
If human immunodeficiency virus (HIV) positive, documented cluster of differentiation (CD4) count more than 200/µL within 6 months of study entry

Key exclusion criteria (14 listed)

Diagnosed with any bleeding disorder in addition to hemophilia A
Current FVIII inhibitor or history of FVIII inhibitor (defined as positive result of the reporting laboratory)
Subject has no history of exposure to FVIII products (previously untreated patient [PUP])
Subject is currently utilizing primary FVIII prophylaxis
Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry

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
XynthaXyntha for on-demand treatment of bleeding episodes were according to investigator prescription during the 6 months observation period. The recovery assessed by determining the Factor VIII (FVIII) concentration (FVIII:C) levels in individual subjects at the initial and final visits. The dose of Xyntha for recovery assessments is: single 50 IU/kg (±5 IU/kg) IV bolus infusion. All Xyntha administrations occurred in the clinic (hospital).	Biological	Xyntha

Primary outcome

Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
Time frame: 8 hours post infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion
Time frame: 24 hours post infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Number of Participants With Factor VIII (FVIII) Inhibitor Development
Time frame: Day 1 and Month 6 or Early Termination Visit
Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by participant exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII infusion, and <= 100 documented Exposure Days (EDs), while Previously Treated Patients (PTPs): those who had received >100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.

4 secondary outcomes

FVIII Recovery : Change From Baseline in FVIII Concentration
Time frame: Day 1 and Month 6 or Early Termination Visit
Number of Participants With Less Than Expected Therapeutic Effect (LETE)
Time frame: 24 hours after each of 2 successive infusion, up to 6 months
Number of Participants With Thrombosis Allergic-Type Reactions
Time frame: Baseline up to 6 months
Number of Participants With Thrombosis
Time frame: Baseline up to 6 months

Results and safety

Results posted to the registry on 8 Feb 2011, 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	53	49

Primary outcome: Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion

Time frame: 8 hours post infusion. Mean (Standard Deviation). Unit: Units on a scale

Measure	Xyntha n = 51
Value	1.86 (0.65)

Population analysed: The Full Analysis Set (FAS) consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Primary outcome: Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion

Time frame: 24 hours post infusion. Mean (Standard Deviation). Unit: Units on a scale

Measure	Xyntha n = 51
Value	1.74 (0.61)

Population analysed: The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Primary outcome: Number of Participants With Factor VIII (FVIII) Inhibitor Development

Time frame: Day 1 and Month 6 or Early Termination Visit. Number. Unit: Participants

Measure	Xyntha n = 53
MTP (n=36)	6
PTP (n=17)	1

Population analysed: The Safety Set (SS) consisted of all participants who had taken at least 1 dose of investigational drug.

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Xyntha	9 of 53 (17%)	13 of 53 (24.5%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 33269010Yang R, Zhao Y, Wang X, Sun J, Wu R, Jin C, Jin J, Wu D, Rendo P, Sun F, Rupon J, Huard F, Korth-Bradley JM, Xu L, Luo B, Liu YC. Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies. J Blood Med. 2020 Nov 25;11:439-448. doi: 10.2147/JBM.S241605. eCollection 2020.
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
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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

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

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Xyntha in people with Hemophilia A, run by Pfizer at 6 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 3 inclusion and 14 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 12 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 8 Feb 2011). 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?

Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion (time frame: 8 hours post infusion). The study lists 3 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 NCT00868530, which also lists 1 linked publication.

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 12 Jun 2025.