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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-003027-61 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BeneFIX | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BENEFIX | Drug | Single dose of 50 IU/kg of BeneFIX by intravenous infusion within 10 minutes. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Observed Plasma Concentration (Cmax) | 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) | 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) | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose | |
| Time to Reach Cmax (Tmax) | 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. | 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. | 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. | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Beijing | 100032 | China | |||
| Hematology Department,Beijing Children's Hospital, Capital Medical University |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Participants did not receive an infusion of any FIX products for at least 4 days and were required to be in a non-bleeding state before the administration of BeneFIX on Day 1.
Twelve (12) participants that were age 6 years or older (weight ≥20 kg) with moderate severe to severe hemophilia B (FIX activity ≤2%) were enrolled. Four (4) participants were at the younger age range of ≥6 and <12 years; remaining participants were 12 years or older.
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| ID | Title | Description |
|---|---|---|
| FG000 | BeneFIX 50 IU/kg; Age Group: >=6 and <12 Years | Participants received a single dose of 50 IU/kg BeneFIX administered by intravenous (IV) infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1. |
| FG001 | BeneFIX 50 IU/kg; Age Group: >=12 Years | Participants received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
All participants were included in the baseline population.
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| ID | Title | Description |
|---|---|---|
| BG000 | All Participants | Participants received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Maximum Observed Plasma Concentration (Cmax) | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | IU/milliliter (IU/mL) | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | BeneFIX 50 IU/kg | Participants received a single dose of 50 IU/kg BeneFIX administered by IV infusion within 10 minutes at approximately 0800 hours (±2 hours) on Day 1. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc | (001)8007181021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D005164 | Factor IX |
| ID | Term |
|---|---|
| D004792 | Enzyme Precursors |
| D045762 | Enzymes and Coenzymes |
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
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| Plasma Decay Half-Life (t½) | Plasma decay half-life is the time measured for the plasma concentration to decrease by one half. | 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. | 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. | Pre-dose, 0.25, 0.5 and 1 hour post-dose |
| 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. | 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) | Baseline up to 96 hours post-dose (Day 5 or early termination) |
| Number of Participants With Inhibitor Development | 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 | 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 | From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug. |
| Beijing |
| 100045 |
| China |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
|
|
| Primary | Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast) | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | IU*hour/milliliter (IU*hr/mL) | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf) | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | IU*hr/ml | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | Time to Reach Cmax (Tmax) | 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. | Posted | Median | Full Range | hours | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | 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. | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | milliliter/kilogram (mL/kg) | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | 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. | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | 1/hr | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | 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. | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | Plasma Decay Half-Life (t½) | Plasma decay half-life is the time measured for the plasma concentration to decrease by one half. | 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. | Posted | Mean | Standard Deviation | hours | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | Systemic Clearance (CL) | CL is a quantitative measure of the rate at which a drug substance is removed from the body. | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | mL/hr/kg | Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose |
|
|
|
| Primary | Incremental Recovery | Incremental recovery: Increase in circulating increase in FIX activity for every IU of BeneFIX administered per kg of body weight. | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | (IU/dL)/(IU/Kg) | Pre-dose, 0.25, 0.5 and 1 hour post-dose |
|
|
|
| Secondary | 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. | The safety analysis set was defined as all participants who received at least 1 dose of BeneFIX. | Posted | Number | Particpants | From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug. |
|
|
|
| Secondary | 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. | The safety analysis population included All participants who received at least 1 dose of BeneFIX. | Posted | Number | Participants | Baseline up to 96 hours post-dose (Day 5 or early termination) |
|
|
|
| Secondary | Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality) | The safety analysis population included All participants who received at least 1 dose of BeneFIX. | Posted | Number | Participants | Baseline up to 96 hours post-dose (Day 5 or early termination) |
|
|
|
| Secondary | Number of Participants With Inhibitor Development | The safety analysis population included All participants who received at least 1 dose of BeneFIX. | Posted | Number | Participants | From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug. |
|
|
|
| Secondary | Number of Participants With Allergic Reactions | The safety analysis population included All participants who received at least 1 dose of BeneFIX. | Posted | Number | Participants | From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug. |
|
|
|
| Secondary | Number of Subjects With Thrombogenicity | The safety analysis population included All participants who received at least 1 dose of BeneFIX. | Posted | Number | Participants | From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug. |
|
|
|
| 0 |
| 12 |
| 0 |
| 12 |
Restriction Description: Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D011506 |
| Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |