Hemophilia BPhase 3NCT00866606

Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

A Phase 3 study testing Benefix in people with Hemophilia B, run by Wyeth is now a wholly owned subsidiary of Pfizer at 6 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

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

Who is it for?: Ages 6 years and older
Conditions the study is for: Hemophilia B
What is being tested?: Benefix
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 35 participants
Where: 6 sitesChina
Study dates: Feb 2009to 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 B (FIX activity: more than 5%, 1-5%, or less than 1%, respectively)
Subjects with previous exposure to FIX replacement therapy
If HIV positive, documented CD4 count more than 200/µL within 6 months of study entry

Who cannot join

Diagnosed with any bleeding disorder in addition to hemophilia B
Current FIX inhibitor or history of FIX inhibitor (defined as >ULN of the reporting laboratory)
Subject has no history of exposure to FIX products (previously untreated patient [PUP])
Subject is currently utilizing primary FIX 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 (ALT and AST >5 x 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, Guangzhou, 510515
Pfizer Investigational Site
Hangzhou, Zhejiang, 310003
Pfizer Investigational Site
Shanghai, 200025
Pfizer Investigational Site
Suzhou, Jiangsu, 215006
Pfizer Investigational Site
Tianjin, Tianjin Municipality, 300020

Other trials in China

About this study

Official title: An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With BeneFIX (Nonacog Alfa, Recombinant Factor IX) In Chinese Subjects With Hemophilia B

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

Conditions: Hemophilia B
Being tested: Benefix
Keywords: On-demand treatment, Hemostatic efficacy, Factor IX activity, Factor IX recovery, Factor IX inhibitor, Chinese
Medical subject headings: Hemophilia B

What participants receive

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

BenefixBiological
BeneFIX for on-demand treatment of bleeding episodes were according to investigator prescription. FIX recovery was assessed by determining the FIX activity (FIX:C) levels in individual subjects. BeneFIX dosage for recovery assessments: single 75 IU/kg (±5 IU/kg) IV bolus infusion.

Study groups in NCT00866606
Study group	Role	What they receive
Benefix	Experimental	Subjects received on-demand treatments with BeneFIX over a 6-month (calendar day) period. Receives: Biological: Benefix

What is being measured

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

Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion
Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
Measured: 8 hours post infusion
Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion
Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
Measured: 24 hours post infusion
Percentage of Participants With FIX Inhibitor Development
Incidence of FIX inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory with Nijmegen assay result >=0.6 Bethesda Unit (BU). Incidence was stratified by participant exposure history - Minimally Treated Patients (MTPs): those who had received at least one prior FIX 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: Baseline up to 6 months

Secondary outcomes (6)

Number of Infusions Required to Treat Each Bleed
The number of BeneFIX infusions required to treat each bleeding episode were analyzed. The average frequency of BeneFIX infusions per hemorrhage incidence to treat every hemorrhage was equal to the total number of injections throughout the study divided by total number of hemorrhagic events.
Measured: Baseline up to 6 months
FIX Incremental Recovery
FIX recovery was assessed by evaluating FIX:C after initial exposure and following 6 months of repeated exposures to BeneFIX. A modified FIX recovery study was performed at Day 1 (Visit 2) and Month 6/Final/Early Termination visits (Visit 4) and when clinically indicated at the applicable on-demand visits. Blood samples for determination of FIX:C were collected immediately before BeneFIX infusion and at 30 minutes (±5 minutes) after the start of infusion. Post-infusion blood samples were collected via venipuncture in arm contralateral to arm used for infusion.
Measured: Baseline (Visit 2) up to 6 months (Visit 4)
Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)
The incidence of LETE for on-demand treatment was defined as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.
Measured: Baseline up to 6 months
Percentage of Participants With Allergic-Type Allergic Reactions
Hypersensitivity to undesirable (damaging, discomfort-producing and sometimes fatal) reactions produced by the normal immune system. Hypersensitivity reactions require a pre-sensitized (immune) state of the host.
Measured: Baseline up to 6 months
Percentage of Participants With Thrombosis
Thrombosis is the formation of a blood clot (thrombus) inside a blood vessel, obstructing the flow of blood through the circulatory system. When a blood vessel is injured, the body uses platelets and fibrin to form a blood clot to prevent blood loss.
Measured: Baseline up to 6 months
Percentage of Participants With Red Blood Cell (RBC) Agglutination
RBC Agglutination is the clumping of red blood cells in the presence of an antibody. The antibody or other molecule bonded multiple particles and joined them, creating a large complex.
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.

Feb 2009Dec 2009

First posted20 Mar 2009 (estimated)
Study startFeb 2009
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 BStudies of the same condition, open and under way.
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: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 35 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00866606
Sponsor's protocol ID: 3090A1-3305
Other IDs: B1821004
Status last verified: Apr 2011
First submitted: 19 Mar 2009
Last updated: 5 Apr 2011
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Yang R, Zhao Y, Wang X, Sun J, Jin J, Wu D, Charnigo R, O'Brien A, Zhong Z, Rendo P. Evaluation of the safety and efficacy of recombinant factor IX (nonacog alfa) in minimally treated and previously treated Chinese patients with haemophilia B. Haemophilia. 2012 Sep;18(5):e374-8. doi: 10.1111/j.1365-2516.2012.02907.x. Epub 2012 Jul 9. No abstract available.
PubMed 22776196

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

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
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
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Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
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RecruitingPhase 3
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 B trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Apr 2011. 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 BeneFIX (Nonacog Alfa, Recombinant Factor IX) In Chinese Subjects With Hemophilia B
ClinicalTrials.gov ID: NCT00866606
Acronym / sponsor ID: 3090A1-3305
Conditions: Hemophilia B
Keywords: On-demand treatment, Hemostatic efficacy, Factor IX activity, Factor IX recovery, Factor IX inhibitor, Chinese
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 35 participants (actual)
Lead sponsor: Wyeth is now a wholly owned subsidiary of PfizerIndustry (pharma, biotech, devices)
Study start: Feb 2009
Primary completion: Dec 2009 (actual)
Study completion: Dec 2009 (actual)
First posted: 20 Mar 2009 (estimated)
Results: Posted 4 Jan 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 BeneFIX in Chinese hemophilia B subjects.

Study design

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

Arm	Type	Receives
Benefix	Experimental	Biological: BenefixSubjects received on-demand treatments with BeneFIX 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 B (FIX activity: more than 5%, 1-5%, or less than 1%, respectively)
Subjects with previous exposure to FIX replacement therapy
If HIV positive, documented 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 B
Current FIX inhibitor or history of FIX inhibitor (defined as >ULN of the reporting laboratory)
Subject has no history of exposure to FIX products (previously untreated patient [PUP])
Subject is currently utilizing primary FIX 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
BenefixBeneFIX for on-demand treatment of bleeding episodes were according to investigator prescription. FIX recovery was assessed by determining the FIX activity (FIX:C) levels in individual subjects. BeneFIX dosage for recovery assessments: single 75 IU/kg (±5 IU/kg) IV bolus infusion.	Biological	Benefix

Primary outcome

Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion
Time frame: 8 hours post infusion
Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion
Time frame: 24 hours post infusion
Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
Percentage of Participants With FIX Inhibitor Development
Time frame: Baseline up to 6 months
Incidence of FIX inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory with Nijmegen assay result >=0.6 Bethesda Unit (BU). Incidence was stratified by participant exposure history - Minimally Treated Patients (MTPs): those who had received at least one prior FIX 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.

6 secondary outcomes

Number of Infusions Required to Treat Each Bleed
Time frame: Baseline up to 6 months
FIX Incremental Recovery
Time frame: Baseline (Visit 2) up to 6 months (Visit 4)
Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)
Time frame: Baseline up to 6 months
Percentage of Participants With Allergic-Type Allergic Reactions
Time frame: Baseline up to 6 months
Percentage of Participants With Thrombosis
Time frame: Baseline up to 6 months
Percentage of Participants With Red Blood Cell (RBC) Agglutination
Time frame: Baseline up to 6 months

Results and safety

Results posted to the registry on 4 Jan 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
BeneFactor IX (BeneFIX)	35	31

Primary outcome: Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion

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

Measure	BeneFactor IX (BeneFIX) n = 34
Value	1.70 (0.72)

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

Primary outcome: Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion

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

Measure	BeneFactor IX (BeneFIX) n = 34
Value	1.58 (0.71)

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

Primary outcome: Percentage of Participants With FIX Inhibitor Development

Time frame: Baseline up to 6 months. Number. Unit: Percentage of Participants

Measure	BeneFactor IX (BeneFIX) n = 35
Total (n=35)	2.86
MTP (n=24)	4.17
PTP (n=11)	0

Population analysed: Safety Set (SS) population included all enrolled participants who had taken at least 1 dose of drug.The 'n' is signifying those participants who received study drug and were evaluated for this measure at the timepoint for each visit respectively.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: From screening until 30 days after the last visit (Month 6/Final/Early Termination visit. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BeneFactor IX (BeneFIX)	2 of 35 (5.7%)	19 of 35 (54.3%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2011.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 22776196Yang R, Zhao Y, Wang X, Sun J, Jin J, Wu D, Charnigo R, O'Brien A, Zhong Z, Rendo P. Evaluation of the safety and efficacy of recombinant factor IX (nonacog alfa) in minimally treated and previously treated Chinese patients with haemophilia B. Haemophilia. 2012 Sep;18(5):e374-8. doi: 10.1111/j.1365-2516.2012.02907.x. Epub 2012 Jul 9. No abstract available.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.

Related trials for Hemophilia B

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
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
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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

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 Benefix in people with Hemophilia B, run by Wyeth is now a wholly owned subsidiary of 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 5 Apr 2011. 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 4 Jan 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 of Participants After 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 NCT00866606, 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 5 Apr 2011.