Hemophilia BPhase 1NCT02213250

An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B

A Phase 1 study testing BENEFIX in people with Hemophilia B, run by Pfizer at 2 sites in China.

Completed

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

Trial summary

The sample size of 12 male Chinese subjects are based on the CFDA requirement for a China PK study and to support the registration in China. Read more about this study

Who is it for?: Ages 6 years and older, men only
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: 12 participants
Where: 2 sitesChina
Study dates: Mar 2015to Apr 2015
Phase: Phase 1
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
Men only
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Male Chinese subjects 6 years or older (weight ≥20kg) with moderate to severe hemophilia B (Factor IX activity ≤2%).
Subjects should not have received an infusion of any Factor IX products for at least 4 days before the administration of BeneFIX on Day 1.
Subjects must be in a non-bleeding state before the administration of BeneFIX on Day 1.

Who cannot join

Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing) disease or clinical findings at Screening.
Diagnosed with any other bleeding disorder in addition to hemophilia B.
Current FIX inhibitor or history of FIX inhibitor (defined as > Upper Limit of Normal (ULN) of the reporting lab).

Trial locations and contacts

2 sites in 1 country.

China2 sites

Peking Union Medical College Hospital
Beijing, 100032
Hematology Department,Beijing Children's Hospital, Capital Medical University
Beijing, 100045

Other trials in China

About this study

Official title: An Open-label, Single Dose Pharmacokinetic Study Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Male Chinese Subjects With Hemophilia B

The sample size of 12 male Chinese subjects are based on the CFDA requirement for a China PK study and to support the registration in China.

Conditions: Hemophilia B
Being tested: BENEFIX
Medical subject headings: Hemophilia B

What participants receive

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

BENEFIXDrug
Single dose of 50 IU/kg of BeneFIX by intravenous infusion within 10 minutes.

Study groups in NCT02213250
Study group	Role	What they receive
BeneFIX	Experimental	Not described Receives: Drug: BENEFIX

What is being measured

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

Maximum Observed Plasma Concentration (Cmax)
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast)
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf)
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Time to Reach Cmax (Tmax)
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Volume of Distribution at Steady State (Vss)
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steady-state.
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Terminal Phase Rate Constant (Kel)
Linear regression of the log linear concentration time curve. Only those data points judged to describe the terminal log linear decline were used in the regression.
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Mean Residence Time (MRT)
AUMCinf/AUCinf, where AUMCinf is the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method.
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Plasma Decay Half-Life (t½)
Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Systemic Clearance (CL)
CL is a quantitative measure of the rate at which a drug substance is removed from the body.
Measured: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Incremental Recovery
Incremental recovery: Increase in circulating increase in FIX activity for every IU of BeneFIX administered per kg of body weight.
Measured: Pre-dose, 0.25, 0.5 and 1 hour post-dose

Secondary outcomes (6)

Number of Participants With Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE), and Withdrawals Due to Adverse Events (AE)
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; lifethreatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. TEAE is defined as newly occurring or worsening after first dose.
Measured: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
Number of Participants With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality)
Clinical laboratory analysis tests included hematology, serium chemistry, prothrombin time and urianalysis. Numbers of subjects with laboratory test abnormalities without regard to baseline abnormality were reported.
Measured: Baseline up to 96 hours post-dose (Day 5 or early termination)
Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)
Measured: Baseline up to 96 hours post-dose (Day 5 or early termination)
Number of Participants With Inhibitor Development
Measured: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
Number of Participants With Allergic Reactions
Measured: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
Number of Subjects With Thrombogenicity
Measured: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.

Timeline

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

Mar 2015Apr 2015

First posted11 Aug 2014 (estimated)
Study startMar 2015
Primary completionApr 2015
Study completionApr 2015

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 BStudies 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 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 12 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02213250
Sponsor's protocol ID: B1821048
Other IDs: 2015-003027-61, 2015
Status last verified: Jun 2016
First submitted: 7 Aug 2014
Last updated: 25 Jul 2016
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

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

All Hemophilia B trials

Study at a glance

From the registry record, last updated by the sponsor on 25 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label, Single Dose Pharmacokinetic Study Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Male Chinese Subjects With Hemophilia B
ClinicalTrials.gov ID: NCT02213250
Acronym / sponsor ID: B1821048
Conditions: Hemophilia B
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 12 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Mar 2015
Primary completion: Apr 2015 (actual)
Study completion: Apr 2015 (actual)
First posted: 11 Aug 2014 (estimated)
Results: Posted 25 Jul 2016

Clinical summary

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

The sample size of 12 male Chinese subjects are based on the CFDA requirement for a China PK study and to support the registration in China.

Study design

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

Arm	Type	Receives
BeneFIX	Experimental	Drug: BENEFIX

Who may qualify

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

Key inclusion criteria (3 listed)

Male Chinese subjects 6 years or older (weight ≥20kg) with moderate to severe hemophilia B (Factor IX activity ≤2%).
Subjects should not have received an infusion of any Factor IX products for at least 4 days before the administration of BeneFIX on Day 1.
Subjects must be in a non-bleeding state before the administration of BeneFIX on Day 1.

Key exclusion criteria (3 listed)

Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing) disease or clinical findings at Screening.
Diagnosed with any other bleeding disorder in addition to hemophilia B.
Current FIX inhibitor or history of FIX inhibitor (defined as > Upper Limit of Normal (ULN) of the reporting lab).

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
BENEFIXSingle dose of 50 IU/kg of BeneFIX by intravenous infusion within 10 minutes.	Drug	BeneFIX

Primary outcome

Maximum Observed Plasma Concentration (Cmax)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Time to Reach Cmax (Tmax)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Volume of Distribution at Steady State (Vss)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steady-state.
Terminal Phase Rate Constant (Kel)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Linear regression of the log linear concentration time curve. Only those data points judged to describe the terminal log linear decline were used in the regression.
Mean Residence Time (MRT)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
AUMCinf/AUCinf, where AUMCinf is the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method.
Plasma Decay Half-Life (t½)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.
Systemic Clearance (CL)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
CL is a quantitative measure of the rate at which a drug substance is removed from the body.
Incremental Recovery
Time frame: Pre-dose, 0.25, 0.5 and 1 hour post-dose
Incremental recovery: Increase in circulating increase in FIX activity for every IU of BeneFIX administered per kg of body weight.

6 secondary outcomes

Number of Participants With Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE), and Withdrawals Due to Adverse Events (AE)
Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
Number of Participants With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality)
Time frame: Baseline up to 96 hours post-dose (Day 5 or early termination)
Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)
Time frame: Baseline up to 96 hours post-dose (Day 5 or early termination)
Number of Participants With Inhibitor Development
Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
Number of Participants With Allergic Reactions
Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
Number of Subjects With Thrombogenicity
Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.

Results and safety

Results posted to the registry on 25 Jul 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BeneFIX 50 IU/kg; Age Group: >=6 and <12 Years	4	4
BeneFIX 50 IU/kg; Age Group: >=12 Years	8	8

Primary outcome: Maximum Observed Plasma Concentration (Cmax)

Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose. Geometric mean (Geometric Coefficient of Variation). Unit: IU/milliliter (IU/mL)

Measure	BeneFIX 50 IU/kg; >=6 and <12 Years n = 4	BeneFIX 50 IU/kg; Age Group:>=12 Years n = 8
Value	0.3880 (26)	0.4226 (16)

Population analysed: The pharmacokinetics (PK) parameter analysis population was defined as all participants enrolled and treated who had at least 1 of the PK parameters of primary interest. All participants were included in the PK analysis population.

Primary outcome: Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast)

Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose. Geometric mean (Geometric Coefficient of Variation). Unit: IU*hour/milliliter (IU*hr/mL)

Measure	BeneFIX 50 IU/kg; >=6 and <12 Years n = 4	BeneFIX 50 IU/kg; Age Group:>=12 Years n = 8
Value	6.930 (18)	9.707 (15)

Population analysed: The PK parameter analysis population was defined as all participants enrolled and treated who had at least 1 of the PK parameters of primary interest. All participants were included in the PK analysis population.

Primary outcome: Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf)

Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose. Geometric mean (Geometric Coefficient of Variation). Unit: IU*hr/ml

Measure	BeneFIX 50 IU/kg; Age Group>=6 and <12 Years n = 4	BeneFIX 50 IU/kg; Age Group:>=12 Years n = 8
Value	7.841 (16)	11.66 (15)

Primary outcome: Time to Reach Cmax (Tmax)

Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose. Median (Full Range). Unit: hours

Measure	BeneFIX 50 IU/kg; >=6 and <12 Years n = 4	BeneFIX 50 IU/kg; Age Group:>=12 Years n = 8
Value	0.500 (0.250 to 3.00)	0.375 (0.250 to 3.00)

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.. Other adverse events reported at 0.05% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BeneFIX 50 IU/kg	0 of 12 (0%)	0 of 12 (0%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2016.
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 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
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
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
Hemophilia

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

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

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BENEFIX in people with Hemophilia B, run by Pfizer at 2 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 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 25 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 25 Jul 2016). 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?

Maximum Observed Plasma Concentration (Cmax) (time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose). The study lists 10 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 NCT02213250.

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 25 Jul 2016.