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| ID | Type | Description | Link |
|---|---|---|---|
| 3090A1-3306 | Other Identifier | Alias Study Number | |
| 2011-000520-15 | EudraCT Number |
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The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BeneFIX | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nonacog alfa | Biological | Period 1: During on-demand period, dosing at the discretion of investigator. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Number of Bleeding Episodes. | The annualized bleed rate (ABR) or the annualized number of bleeding episodes per year, will be derived for each participant for each treatment period by using the following formula: ABR = number of bleeds / (Days on treatment period / 365.25) The number of bleeds for the ABR calculation includes all bleeds requiring treatment with factor IX product during the time on treatment. | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Response to On-Demand Treatment for All Bleeding Episodes. | Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by participant/caregiver or investigator/qualified staff. The 4-point scale assessments are Excellent, Good, Moderate or No response. Responses to number of observations were noted. | 2 years |
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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 |
|---|---|---|---|---|---|---|
| UMBAL Sveti Georgi, Klinika po hematologia | Plovdiv | 4002 | Bulgaria | |||
| The Ottawa Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40750723 | Derived | Wojciechowski J, Gaitonde P, Hughes JH, Ravva P. Population Modeling of Factor IX Activity Following Administration of Fidanacogene Elaparvovec Gene Therapy in Participants with Hemophilia B. Clin Pharmacokinet. 2025 Oct;64(10):1531-1548. doi: 10.1007/s40262-025-01535-y. Epub 2025 Aug 1. | |
| 33631774 | Derived |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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The duration of participation was approximately 86 weeks, consisting of a Screening period (1 day to 4 weeks), Period 1 (on-demand treatment for 26 weeks), Period 2 (prophylaxis therapy for 52 weeks), and a follow-up safety period (4 weeks).
Participation included 25 enrolled participants from 15 study centers and 9 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | All Participants | The data for all the participants is presented. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Nonacog alfa | Biological | Period 2: During the prophylaxis period, 100 IU/kg once weekly |
|
|
| Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode. | The number of study drug infusions administered to treat a bleed will be calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). The number of infusions needed to treat a bleed will be classified into the following categories: 1, 2, 3, 4 and >4 infusions. If there were more than one bleed location (e.g., ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence. | 2 years |
| Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX. | The number of spontaneous, non-traumatic breakthrough bleeds within 48 hours following a prophylaxis dose of BeneFIX were summarized. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence. | 2 years |
| Average Infusion Dose. | The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions | 2 years |
| Total Factor Consumption. | The total amount (IU) infused for each infusion recorded were summed to calculate the total factor consumption for each participant. For each infusion, IU/kg was calculated, using the most recently recorded weight measurement and the total factor consumption, divided by number of infusions, and was summarized similarly to average infusion dose (IU). Annualized TFC by weight was reported. Annualized TFC by weight = (Total IU/kg / treatment interval duration)*365.25. | 2 years |
| Incidence of Less Than Expected Therapeutic Effect (LETE) | The following criteria are the definitions for LETE in this study: 1. LETE in the On-Demand Setting: LETE occurs in the on-demand setting if 2 successive "No Response" ratings are recorded after 2 successive BeneFIX drug infusions in the absence of confounding factors. 2. LETE in the Prophylaxis Setting: LETE occurs in the prophylaxis setting if there is a spontaneous bleed within 48 hours (≤ 48 hours) after a regularly scheduled prophylactic dose of BeneFIX in the absence of confounding factors. 3. LETE (Low Recovery): LETE can also be lower than expected recovery of FIX in the opinion of the investigator following infusion of BeneFIX in the absence of confounding factors. Each reported occurrence of low recovery LETE was listed. | 2 years |
| Ottawa |
| Ontario |
| K1H 8L6 |
| Canada |
| University Hospital Center Zagreb | Zagreb | 10000 | Croatia |
| National Blood Centre | Kuala Lumpur | Kuala Lumpur | 50400 | Malaysia |
| Hospital Tengku Ampuan Afzan | Kuantan | Pahang | 25200 | Malaysia |
| Instituto Biomedico de Investigacion A.C. | Aguascalientes | Aguascalientes | 20127 | Mexico |
| Hospital y Clinica OCA | Monterrey | Nuevo León | 64000 | Mexico |
| Nzoz Triclinium | Warsaw | 02-797 | Poland |
| Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku | Wroclaw | 50-367 | Poland |
| Singapore General Hospital | Singapore | 169608 | Singapore |
| Eulji University Hospital | Daejeon | 302-799 | South Korea |
| Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali | Fatih | Istanbul | 34098 | Turkey (Türkiye) |
| Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bolumu | Ankara | 06100 | Turkey (Türkiye) |
| Ege Universitesi Tip Fakultesi | Bornova/Izmir | 35100 | Turkey (Türkiye) |
| Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi | Gaziantep | 27300 | Turkey (Türkiye) |
| Erciyes Universitesi Tip Fakultesi | Kayseri | 38039 | Turkey (Türkiye) |
| Erciyes Universitesi Tip Fakultesi Hastaneleri M. Kemal Dedeman Onkoloji Hastanesi | Kayseri̇, Erciyes | 38039 | Turkey (Türkiye) |
| Tortella BJ, Carr ME, Rendo P, Korth-Bradley J, Smith LM, Kavakli K. Once-weekly prophylaxis regimen of nonacog alfa in patients with hemophilia B: an analysis of timing of bleeding event onset. Blood Coagul Fibrinolysis. 2021 Apr 1;32(3):180-185. doi: 10.1097/MBC.0000000000001012. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | All Participants | The data for all the participants is presented. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| 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 | Annualized Number of Bleeding Episodes. | The annualized bleed rate (ABR) or the annualized number of bleeding episodes per year, will be derived for each participant for each treatment period by using the following formula: ABR = number of bleeds / (Days on treatment period / 365.25) The number of bleeds for the ABR calculation includes all bleeds requiring treatment with factor IX product during the time on treatment. | The efficacy analysis set (EAS) was used for the primary efficacy analyses with respect to ABR. It includes all participants who participated in at least one day of the routine prophylaxis period (ie, in the study through at least Visit 4). | Posted | Mean | Standard Deviation | Number of bleeds per year | 2 years |
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| Secondary | Response to On-Demand Treatment for All Bleeding Episodes. | Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by participant/caregiver or investigator/qualified staff. The 4-point scale assessments are Excellent, Good, Moderate or No response. Responses to number of observations were noted. | The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. Follow-up infusion was only required for 18 participants. | Posted | Number | Number of observations with response | 2 years | bleeds | Participants |
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| Secondary | Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode. | The number of study drug infusions administered to treat a bleed will be calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). The number of infusions needed to treat a bleed will be classified into the following categories: 1, 2, 3, 4 and >4 infusions. If there were more than one bleed location (e.g., ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence. | The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. | Posted | Number | Number of bleeds requiring infusion | 2 years | bleeds | Participants |
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| Secondary | Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX. | The number of spontaneous, non-traumatic breakthrough bleeds within 48 hours following a prophylaxis dose of BeneFIX were summarized. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence. | The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. Three participants experienced 1 spontaneous bleeding episode each within 48 hours of a previous prophylaxis infusion. | Posted | Mean | Standard Deviation | Number of breakthrough bleeds | 2 years | breakthrough bleeds | Participants |
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| Secondary | Average Infusion Dose. | The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions | The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. | Posted | Mean | Standard Deviation | IU/Kg | 2 years |
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| Secondary | Total Factor Consumption. | The total amount (IU) infused for each infusion recorded were summed to calculate the total factor consumption for each participant. For each infusion, IU/kg was calculated, using the most recently recorded weight measurement and the total factor consumption, divided by number of infusions, and was summarized similarly to average infusion dose (IU). Annualized TFC by weight was reported. Annualized TFC by weight = (Total IU/kg / treatment interval duration)*365.25. | The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. | Posted | Mean | Standard Deviation | IU/Kg | 2 years |
|
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| Secondary | Incidence of Less Than Expected Therapeutic Effect (LETE) | The following criteria are the definitions for LETE in this study: 1. LETE in the On-Demand Setting: LETE occurs in the on-demand setting if 2 successive "No Response" ratings are recorded after 2 successive BeneFIX drug infusions in the absence of confounding factors. 2. LETE in the Prophylaxis Setting: LETE occurs in the prophylaxis setting if there is a spontaneous bleed within 48 hours (≤ 48 hours) after a regularly scheduled prophylactic dose of BeneFIX in the absence of confounding factors. 3. LETE (Low Recovery): LETE can also be lower than expected recovery of FIX in the opinion of the investigator following infusion of BeneFIX in the absence of confounding factors. Each reported occurrence of low recovery LETE was listed. | The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. | Posted | Number | Percentage of occurence | 2 years |
|
|
Until follow-up 82 weeks +/- 4 days.
Five participants experienced six SAEs since one participant experienced the same event twice (nephrolithiasis).
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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 | On-Demand Therapy | Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label. | 1 | 25 | 16 | 25 | ||
| EG001 | Prophylaxis Therapy | The prophylaxis regimen of approximately 100IU/kg once weekly was initiated at Visit 4. | 4 | 25 | 20 | 25 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pain | General disorders | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Blood pressure decreased | Investigations | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Lipoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA version 17.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA version 17.0 | Non-systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA version 17.0 | Non-systematic Assessment |
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| Influenza | Infections and infestations | MedDRA version 17.0 | Non-systematic Assessment |
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| Inappropriate schedule of drug administration | Injury, poisoning and procedural complications | MedDRA version 17.0 | Non-systematic Assessment |
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| Drug dose omission | Injury, poisoning and procedural complications | MedDRA version 17.0 | Non-systematic Assessment |
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| Medication error | Injury, poisoning and procedural complications | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Underdose | Injury, poisoning and procedural complications | MedDRA version 17.0 | Non-systematic Assessment |
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| Wrong dose administration | Injury, poisoning and procedural complications | MedDRA version 17.0 | Non-systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | MedDRA version 17.0 | Non-systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA version 17.0 | Non-systematic Assessment |
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| Pyrexia | General disorders | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Local swelling | General disorders | MedDRA version 17.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 17.0 | 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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
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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 |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
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| bleeds |
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| bleeds |
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| breakthrough bleeds |
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