Hemophilia APhase 1NCT02461992

Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A

A Phase 1 study testing Intravenous infusions of Xyntha in people with Hemophilia A, 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

An open-label, single dose pharmacokinetic study of Xyntha (Moroctocog Alfa (AF-CC), Recombinant Factor VIII) in male Chinese subjects with hemophilia A Read more about this study

Who is it for?: Ages 6 years and older, men only
Conditions the study is for: Hemophilia A
What is being tested?: Intravenous infusions of Xyntha
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 13 participants
Where: 2 sitesChina
Study dates: Jul 2015to Aug 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

Subjects must meet all of the following inclusion criteria to be eligible for enrollment in the study:
Male Chinese subjects 6 years or older (weight >20 kg) with severe hemophilia A (factor VIII activity <1%) previously treated with > 150 exposure days to any FVIII-containing products.
Subjects should not have received an infusion of any FVIII products for at least 3 days (at least 72 hours) before the administration of Xyntha on Day 1.
Subjects must be in a non bleeding state before the administration of Xyntha on Day 1.
Evidence of a personally or legally acceptable representative (legally acceptable representative is only applicable to pediatric subjects) signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.

Who cannot join

Subjects with any of the following characteristics/conditions will not be included in the study:
Current FVIII inhibitor or history of FVIII inhibitor (defined as > upper limit of normal (ULN) of the local reporting laboratory).
Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing) or clinical findings at Screening.
Diagnosed with any other bleeding disorder in addition to hemophilia A.
Documented Human Immunodeficiency Virus (HIV).
Subjects anticipating elective surgery or other invasive procedure within 1 month following study entry.

Show all 24Show fewer

Treatment with immunomodulatory therapy within 30 days or 5 half lives whichever is longer, prior to study entry or planned use for the duration of study participation.
Subjects with known hypersensitivity to the active substance or to any of the excipients of Xyntha.
Subjects with a known hypersensitivity to Chinese Hamster Ovary cell (CHO cell) proteins.
Subjects with ANY of the following abnormalities in clinical laboratory tests at screening, as assessed by the study specific laboratory and confirmed by a single repeat, if deemed necessary: significant hepatic or renal impairment (alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 x ULN, or total bilirubin >2 x ULN or serum creatinine >2 x ULN), prothrombin time >1.5 x ULN, platelet count <80,000 L. Subjects with Gilbert's disease may be enrolled.
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.
A positive urine drug screen.
History of regular alcohol consumption exceeding 14 drinks/week (1 drink = 5 ounces (150 mL) of wine, 12 ounces (360 mL) of beer, or 1.5 ounces (45 mL) of hard liquor) within 6 months of screening.
Treatment with an investigational drug within 30 days or 5 half lives preceding Day 1, whichever is longer.
Screening supine blood pressure 140 mm Hg (systolic) or 90 mm Hg (diastolic), following at least 5 minutes of supine rest. If blood pressure (BP) is 140 mm Hg (systolic) or 90 mm Hg (diastolic), the BP should be repeated two more times and the average of the three BP values should be used to determine the subject's eligibility.
Screening supine 12 lead ECG demonstrating QTcF >450 or a QRS interval >120 msec msec. If QTcF exceeds 450 msec, or QRS exceeds 120 msec, the ECG should be repeated two more times and the average of the three QTcF values should be used to determine the subject's eligibility.
Blood donation (excluding plasma donations) of approximately 500 mL or more within 56 days prior to dosing.
History of sensitivity to heparin or heparin induced thrombocytopenia.
Unwilling or unable to comply with the Lifestyle Guidelines described in this protocol.
Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct of the study.
Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
Subjects with history of infection within 1 week prior to study entry.
Male subjects with partners currently pregnant and male subjects able to father children who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 28 days after the last dose of investigational product.

Trial locations and contacts

2 sites in 1 country.

China2 sites

Phase I Unit, Clinical Pharmacology Research Center, 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 Xyntha (Moroctocog Alfa (Af-cc), Recombinant Factor Viii) In Male Chinese Subjects With Hemophilia A

An open-label, single dose pharmacokinetic study of Xyntha (Moroctocog Alfa (AF-CC), Recombinant Factor VIII) in male Chinese subjects with hemophilia A

Read the detailed description

The purpose of this study is to obtain pharmacokinetic profiles of FVIII:C after Xyntha administration in Chinese patients with severe hemophilia A, which is in support of the continued registration of Xyntha in China

Conditions: Hemophilia A
Being tested: Intravenous infusions of Xyntha
Keywords: FVIII activity
Medical subject headings: Hemophilia A

What participants receive

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

Intravenous infusions of XynthaDrug
A single dose 50IU/kg dose of Xyntha administered by intravenous infusion within 10 minutes on Day 1

Study groups in NCT02461992
Study group	Role	What they receive
Intravenous infusion of Xyntha	Experimental	observation arm Receives: Drug: Intravenous infusions of Xyntha

What is being measured

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

Maximum Plasma FVIII Activity (Cmax)
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Area Under the Plasma FVIII Activity-Time Profile From Time 0 to Time of the Last Quantifiable Concentration (AUClast)
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Area Under the Plasma FVIII Activity-Time Profile From Time 0 Extrapolated to Infinite Time (AUCinf)
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Time to Reach Maximum Observed Plasma Concentration (Tmax)
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Clearance (CL)
Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Volume of Distribution at Steady-State (Vss)
Volume of distribution is 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 volume of distribution at steady-state.
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Terminal Phase Rate Constant (Kel)
Terminal phase rate constant is the absolute value of the slope of a linear regression during the terminal phase of the natural--logarithm transformed concentration--time profile.
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Terminal Elimination Half-Life (t1/2)
Terminal half-life is the time measured for the plasma concentration to decrease by one half.
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Mean Residence Time (MRT)
MRT = AUMCinf / AUCinf, where AUMCinf is the area under the first moment curve from zero time to infinity calculated as AUMCinf = AUMCt + ((t x Ct) / kel) + (Ct / kel^2). AUMCt is the area under the first moment curve from zero time to time t calculated using the trapezoidal method.
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Incremental Recovery (INCREC)
Incremental recovery is the increase in circulating FVIII activity for every IU of Xyntha administered per kilogram of body weight.
Measured: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose

Timeline

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

Jul 2015Aug 2015

First posted3 Jun 2015 (estimated)
Study startJul 2015
Primary completionAug 2015
Study completionAug 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.
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 1
Primary purpose: Basic science
Masking: None (open label)
Enrolment: 13 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02461992
Sponsor's protocol ID: B1831082
Other IDs: 2015-003685-88, 2015
Status last verified: Jul 2016
First submitted: 1 Jun 2015
Last updated: 7 Sep 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Liu H, Wu R, Hu P, Sun F, Xu L, Liang Y, Nepal S, Qu PR, Huard F, Korth-Bradley JM. An Open-label, Single-dose, Pharmacokinetic Study of Factor VIII Activity After Administration of Moroctocog Alfa (AF-CC) in Male Chinese Patients With Hemophilia A. Clin Ther. 2017 Jul;39(7):1313-1319. doi: 10.1016/j.clinthera.2017.05.344. Epub 2017 Jun 7.
PubMed 28601434

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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All Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 7 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label, Single Dose Pharmacokinetic Study Of Xyntha (Moroctocog Alfa (Af-cc), Recombinant Factor Viii) In Male Chinese Subjects With Hemophilia A
ClinicalTrials.gov ID: NCT02461992
Acronym / sponsor ID: B1831082
Conditions: Hemophilia A
Keywords: FVIII activity
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 13 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Jul 2015
Primary completion: Aug 2015 (actual)
Study completion: Aug 2015 (actual)
First posted: 3 Jun 2015 (estimated)
Results: Posted 15 Jun 2016

Clinical summary

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

An open-label, single dose pharmacokinetic study of Xyntha (Moroctocog Alfa (AF-CC), Recombinant Factor VIII) in male Chinese subjects with hemophilia A

Detailed description

Study design

Primary purpose: Basic science
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Intravenous infusion of Xyntha	Experimental	Drug: Intravenous infusions of Xynthaobservation arm

Who may qualify

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

Key inclusion criteria (6 listed)

Subjects must meet all of the following inclusion criteria to be eligible for enrollment in the study:
Male Chinese subjects 6 years or older (weight >20 kg) with severe hemophilia A (factor VIII activity <1%) previously treated with > 150 exposure days to any FVIII-containing products.
Subjects should not have received an infusion of any FVIII products for at least 3 days (at least 72 hours) before the administration of Xyntha on Day 1.
Subjects must be in a non bleeding state before the administration of Xyntha on Day 1.
Evidence of a personally or legally acceptable representative (legally acceptable representative is only applicable to pediatric subjects) signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.

Key exclusion criteria (24 listed)

Subjects with any of the following characteristics/conditions will not be included in the study:
Current FVIII inhibitor or history of FVIII inhibitor (defined as > upper limit of normal (ULN) of the local reporting laboratory).
Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing) or clinical findings at Screening.
Diagnosed with any other bleeding disorder in addition to hemophilia A.
Documented Human Immunodeficiency Virus (HIV).

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
Intravenous infusions of XynthaA single dose 50IU/kg dose of Xyntha administered by intravenous infusion within 10 minutes on Day 1	Drug	Intravenous infusion of Xyntha

Primary outcome

Maximum Plasma FVIII Activity (Cmax)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Area Under the Plasma FVIII Activity-Time Profile From Time 0 to Time of the Last Quantifiable Concentration (AUClast)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Area Under the Plasma FVIII Activity-Time Profile From Time 0 Extrapolated to Infinite Time (AUCinf)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Time to Reach Maximum Observed Plasma Concentration (Tmax)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Clearance (CL)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.
Volume of Distribution at Steady-State (Vss)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Volume of distribution is 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 volume of distribution at steady-state.
Terminal Phase Rate Constant (Kel)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Terminal phase rate constant is the absolute value of the slope of a linear regression during the terminal phase of the natural--logarithm transformed concentration--time profile.
Terminal Elimination Half-Life (t1/2)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Terminal half-life is the time measured for the plasma concentration to decrease by one half.
Mean Residence Time (MRT)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
MRT = AUMCinf / AUCinf, where AUMCinf is the area under the first moment curve from zero time to infinity calculated as AUMCinf = AUMCt + ((t x Ct) / kel) + (Ct / kel^2). AUMCt is the area under the first moment curve from zero time to time t calculated using the trapezoidal method.
Incremental Recovery (INCREC)
Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose
Incremental recovery is the increase in circulating FVIII activity for every IU of Xyntha administered per kilogram of body weight.

Results and safety

Results posted to the registry on 15 Jun 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
Xyntha 50 IU/kg	13	13

Primary outcome: Maximum Plasma FVIII Activity (Cmax)

Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose. Geometric mean (Geometric Coefficient of Variation). Unit: IU/milliliter (mL)

Measure	Xyntha 50 IU/kg n = 13
Value	1.147 (44)

Population analysed: The pharmacokinetic (PK) parameter analysis population is defined as all participants enrolled and treated who have at least 1 of the PK parameters of primary interest reported.

Primary outcome: Area Under the Plasma FVIII Activity-Time Profile From Time 0 to Time of the Last Quantifiable Concentration (AUClast)

Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose. Geometric mean (Geometric Coefficient of Variation). Unit: IU*hour/mL

Measure	Xyntha 50 IU/kg n = 13
Value	14.49 (57)

Population analysed: The PK parameter analysis population is defined as all participants enrolled and treated who have at least 1 of the PK parameters of primary interest reported.

Primary outcome: Area Under the Plasma FVIII Activity-Time Profile From Time 0 Extrapolated to Infinite Time (AUCinf)

Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose. Geometric mean (Geometric Coefficient of Variation). Unit: IU*hour/mL

Measure	Xyntha 50 IU/kg n = 13
Value	15.21 (58)

Population analysed: The PK parameter analysis population is defined as all participants enrolled and treated who have at least 1 of the PK parameters of primary interest reported.

Primary outcome: Time to Reach Maximum Observed Plasma Concentration (Tmax)

Time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose. Median (Full Range). Unit: hour

Measure	Xyntha 50 IU/kg n = 13
Value	0.500 (0.250 to 3.00)

Population analysed: The PK parameter analysis population is defined as all participants enrolled and treated who have at least 1 of the PK parameters of primary interest reported.

Adverse events

Time frame: Baseline up to Day 28. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Xyntha 50 IU/kg	2 of 13 (15.4%)	2 of 13 (15.4%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Sep 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 28601434Liu H, Wu R, Hu P, Sun F, Xu L, Liang Y, Nepal S, Qu PR, Huard F, Korth-Bradley JM. An Open-label, Single-dose, Pharmacokinetic Study of Factor VIII Activity After Administration of Moroctocog Alfa (AF-CC) in Male Chinese Patients With Hemophilia A. Clin Ther. 2017 Jul;39(7):1313-1319. doi: 10.1016/j.clinthera.2017.05.344. Epub 2017 Jun 7.
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
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Not yet recruiting
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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

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 Intravenous infusions of Xyntha in people with Hemophilia A, 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 6 inclusion and 24 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 7 Sep 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 15 Jun 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 Plasma FVIII Activity (Cmax) (time frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 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 NCT02461992, 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 7 Sep 2016.