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| ID | Type | Description | Link |
|---|---|---|---|
| B1821004 |
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This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Benefix | Experimental | Subjects received on-demand treatments with BeneFIX over a 6-month (calendar day) period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Benefix | Biological | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | Baseline up to 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline up to 6 months |
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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 |
|---|---|---|---|---|---|---|
| Pfizer Investigational Site | Guangzhou | Guangzhou | 510515 | China | ||
| Pfizer Investigational Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22776196 | Derived | 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. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Participants were enrolled in six centers in China.
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| ID | Title | Description |
|---|---|---|
| FG000 | BeneFactor IX (BeneFIX) | Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital). |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | BeneFactor IX (BeneFIX) | Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital). |
| 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 | 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. | FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. | Posted | Mean | Standard Deviation | Units on a scale | 8 hours post infusion |
|
From screening until 30 days after the last visit (Month 6/Final/Early Termination visit
The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
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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 | BeneFactor IX (BeneFIX) | Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital). |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hemorrhage intracranial | Nervous system disorders | MedDRA | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | MedDRA | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | 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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|
| 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. | 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. | 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. | 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. | 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. | Baseline up to 6 months |
| Suzhou |
| Jiangsu |
| 215006 |
| China |
| Pfizer Investigational Site | Tianjin | Tianjin Municipality | 300020 | China |
| Pfizer Investigational Site | Hangzhou | Zhejiang | 310003 | China |
| Pfizer Investigational Site | Beijing | 100730 | China |
| Pfizer Investigational Site | Shanghai | 200025 | China |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
|
|
| Primary | 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. | FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. | Posted | Mean | Standard Deviation | Units on a scale | 24 hours post infusion |
|
|
|
| Primary | 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. | 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. | Posted | Number | Percentage of Participants | Baseline up to 6 months |
|
|
|
| Secondary | 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. | FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. | Posted | Mean | Standard Deviation | Infusions | Baseline up to 6 months |
|
|
|
| Secondary | 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. | FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. The 'n' is signifying those participants who received study drug and were evaluated for this measure at the timepoint for each visit respectively. | Posted | Mean | Standard Deviation | IU/dL per IU/kg | Baseline (Visit 2) up to 6 months (Visit 4) |
|
|
|
| Secondary | 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. | FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. | Posted | Number | Percentage of Participants | Baseline up to 6 months |
|
|
|
| Secondary | 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. | SS population included all enrolled participants who had taken at least 1 dose of drug. | Posted | Number | Percentage of Participants | Baseline up to 6 months |
|
|
|
| Secondary | 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. | SS population included all enrolled participants who had taken at least 1 dose of drug. | Posted | Number | Percentage of Participants | Baseline up to 6 months |
|
|
|
| Secondary | 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. | SS population included all enrolled participants who had taken at least 1 dose of drug. | Posted | Number | Percentage of Participants | Baseline up to 6 months |
|
|
|
| 2 |
| 35 |
| 19 |
| 35 |
| Hemophilia B with anti factor IX | Congenital, familial and genetic disorders | MedDRA | Non-systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
|
| Fatigue | General disorders | MedDRA | Non-systematic Assessment |
|
| Inflammation | General disorders | MedDRA | Non-systematic Assessment |
|
| Edema peripheral | General disorders | MedDRA | Non-systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA | Non-systematic Assessment |
|
| Injury | Injury, poisoning and procedural complications | MedDRA | Non-systematic Assessment |
|
| Joint Sprain | Injury, poisoning and procedural complications | MedDRA | Non-systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA | Non-systematic Assessment |
|
| Hematocrit abnormal | Investigations | MedDRA | Non-systematic Assessment |
|
| Hemoglobin abnormal | Investigations | MedDRA | Non-systematic Assessment |
|
| Red blood cell count abnormal | Investigations | MedDRA | Non-systematic Assessment |
|
| Vertigo | Nervous system disorders | MedDRA | Non-systematic Assessment |
|
| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA | Non-systematic Assessment |
|
| Hemorrhage | Vascular disorders | MedDRA | Non-systematic Assessment |
|
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 |
| Title | Measurements |
|---|---|
|