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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-004430-38 | EudraCT Number |
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Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Arm | Experimental | At least twenty five eligible male subjects will be enrolled in the treatment arm to receive Nonacog alfa until 16 exposure days (EDs) or a period of up to 8 weeks on treatment had occurred (whichever occurs first). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nonacog alfa | Biological | Nonacog alfa is indicated in India for the treatment and prophylaxis of bleeding in patients with hemophilia B (congenital factor IX deficiency). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Who Developed Factor IX (FIX) Inhibitors | FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study. | At Visit 4 (any 1 day between Day 52 to Day 60) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs) | An adverse events (AE) is any untoward medical occurrence in clinical investigation participant administered a product or medical device; event need not necessarily to have a causal relationship with treatment or usage. SAEs: an AE resulting in any of following outcomes/deemed significant for any other reason: death; initial/prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. MIEs: any confirmed FIX inhibitor development (titer greater than or equal to 0.6 BU/mL confirmed by central laboratory testing), thrombotic events (any event associated with the formation of a blood clot including catheter-associated thrombi and thrombotic complications) or hypersensitivity reactions (hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgment). |
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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 |
|---|---|---|---|---|---|---|
| Nirmal Hospital Pvt Ltd. | Surat | Gujarat | 395002 | India | ||
| K.J. Somaiya Hospital and Research Centre |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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In this study, participants aged greater than or equal to 12 years to less than or equal to 65 years with congenital moderately-severe to severe hemophilia B (factor IX activity less than or equal to 2 percent) who had at least 50 exposure days (EDs) (ED was defined as a 24 hours period in which the participant was administered any FIX-containing product) to FIX-containing products were enrolled.
The study was conducted in India from 10 Feb 2020 to 24 Sep 2020. A total of 25 participants were enrolled.
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| ID | Title | Description |
|---|---|---|
| FG000 | Nonacog Alfa | Participants received nonacog alfa intravenous (IV) injection as follows: Prophylactic treatment regimen: at a dose of 40 international unit per kilogram (IU/kg) (range 13 to 78 IU/kg) at intervals of 3 to 4 days in accordance with the local product document (LPD) guidelines until at least 16 exposure days or a period of up to 8 weeks on nonacog alfa treatment had occurred (whichever occurred first). For on demand (OD) treatment (infusions used to treat bleeding episodes) regimen nonacog alfa was administered at individual doses and frequency depending on the clinical effectiveness (clinical effectiveness refers to the efficacy of nonacog alfa) in individual participants as recommended in approved LPD. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Safety analysis set included all participants who received at least 1 dose of nonacog alfa.
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| ID | Title | Description |
|---|---|---|
| BG000 | Nonacog Alfa | Participants received nonacog alfa IV injection as follows: Prophylactic treatment regimen: at a dose of 40 IU/kg (range 13 to 78 IU/kg) at intervals of 3 to 4 days in accordance with the LPD guidelines until at least 16 exposure days or a period of up to 8 weeks on nonacog alfa treatment had occurred (whichever occurred first). For OD treatment (infusions used to treat bleeding episodes) regimen nonacog alfa was administered at individual doses and frequency depending on the clinical effectiveness (clinical effectiveness refers to the efficacy of nonacog alfa) in individual participants as recommended in approved LPD. |
| 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 | Percentage of Participants Who Developed Factor IX (FIX) Inhibitors | FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study. | Safety analysis set included all participants who received at least 1 dose of nonacog alfa. | Posted | Number | 90% Confidence Interval | Percentage of participants | At Visit 4 (any 1 day between Day 52 to Day 60) |
|
Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)
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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 | Nonacog Alfa | Participants received nonacog alfa IV injection as follows: Prophylactic treatment regimen: at a dose of 40 IU/kg (range 13 to 78 IU/kg) at intervals of 3 to 4 days in accordance with the LPD guidelines until at least 16 exposure days or a period of up to 8 weeks on nonacog alfa treatment had occurred (whichever occurred first). For OD treatment (infusions used to treat bleeding episodes) regimen nonacog alfa was administered at individual doses and frequency depending on the clinical effectiveness (clinical effectiveness refers to the efficacy of nonacog alfa) in individual participants as recommended in approved LPD. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dental caries | Gastrointestinal disorders | MedDRA v23.0 | 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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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 16, 2019 | Jun 28, 2021 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 2, 2019 | Jun 28, 2021 | SAP_001.pdf |
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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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|
| Baseline up to 28 days after last dose of study drug (i.e, up to 116 days) |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs are events between first dose of study drug and up to 116 days that were absent before treatment or that worsened relative to pretreatment state. | Baseline up to 28 days after last dose of study drug (i.e, up to 116 days) |
| Mean Annualized Bleeding Rate (ABR) | ABR: number of bleeding episodes per year. ABR for each participant = number of bleeds during treatment interval duration (TID)/(TID/365.25). Number of bleeds for each participant for ABR included all new bleeds (with unique start date and time) requiring treatment with nonacog alfa during TID. TID=date of Visit 4 (3 to 10 days after last dose) - date of Visit 2 (Day 1) +1. For participants who had Visit 4 beyond 3 (+7) days after final dose due to COVID-19 pandemic, date of final dose was used in place of date of Visit 4. For discontinued participants, date of final dose/last study visit date (whichever occurred later) was used to replace Visit 4. | Up to 88 Days |
| Mean Annualized Total Factor Consumption (TFC) Per Participant | The TFC per participant was the sum of the total amount (in international units [IU]) infused for each infusion for each participant. Annualized TFC of nonacog alfa was derived for each participant by using the following formula; Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as the number of days beginning on Visit 2 ("Day 1", provided an infusion was given) up to Visit 4 (any 1 day between Day 52 to Day 60). | Up to 88 Days |
| Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant | The total amount in international units (IU) per kilogram infused for each infusion recorded were summed to calculate the total factor consumption for each participant. Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as Date of Visit 4 - Date of Visit 2 +1. If Date of Visit 4 - Last dosing date was > 10 days then Date of Visit 4 was replaced with Last dosing date. If Date of Visit 4 was missing (due to discontinuation or any other reason), then Date of Visit 4 was replaced with last dosing date or last subject visit date, whichever was greater. International unit per kilogram= IU/kg. | Up to 88 days |
| Mean Number of Nonacog Alfa Infusions Used to Treat Each Bleed | Number of nonacog alfa infusions used to treat each bleed in participants was calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). On demand treatment was defined as treatment used to treat a bleeding episode. | Up to 88 days |
| Mumbai |
| Maharashtra |
| 400022 |
| India |
| Sahyadri Clinical Research & Development Centre | Pune | Maharashtra | 411004 | India |
| Christian Medical College and Hospital | Ludhiana | Punjab | 141008 | India |
| Sahyadri Super Specialty Hospital | Pune | 411004 | India |
| Years |
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| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| Secondary | Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs) | An adverse events (AE) is any untoward medical occurrence in clinical investigation participant administered a product or medical device; event need not necessarily to have a causal relationship with treatment or usage. SAEs: an AE resulting in any of following outcomes/deemed significant for any other reason: death; initial/prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. MIEs: any confirmed FIX inhibitor development (titer greater than or equal to 0.6 BU/mL confirmed by central laboratory testing), thrombotic events (any event associated with the formation of a blood clot including catheter-associated thrombi and thrombotic complications) or hypersensitivity reactions (hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgment). | Safety analysis set included all participants who received at least 1 dose of nonacog alfa. | Posted | Count of Participants | Participants | Baseline up to 28 days after last dose of study drug (i.e, up to 116 days) |
|
|
|
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs are events between first dose of study drug and up to 116 days that were absent before treatment or that worsened relative to pretreatment state. | Safety analysis set included all participants who received at least 1 dose of nonacog alfa. | Posted | Count of Participants | Participants | Baseline up to 28 days after last dose of study drug (i.e, up to 116 days) |
|
|
|
| Secondary | Mean Annualized Bleeding Rate (ABR) | ABR: number of bleeding episodes per year. ABR for each participant = number of bleeds during treatment interval duration (TID)/(TID/365.25). Number of bleeds for each participant for ABR included all new bleeds (with unique start date and time) requiring treatment with nonacog alfa during TID. TID=date of Visit 4 (3 to 10 days after last dose) - date of Visit 2 (Day 1) +1. For participants who had Visit 4 beyond 3 (+7) days after final dose due to COVID-19 pandemic, date of final dose was used in place of date of Visit 4. For discontinued participants, date of final dose/last study visit date (whichever occurred later) was used to replace Visit 4. | The overall mean ABR was calculated on participants who received an on-demand infusion of Nonacog-Alfa. Since, there were no participants with bleeds, data for this outcome measure was not collected and reported. | Posted | Up to 88 Days |
|
|
| Secondary | Mean Annualized Total Factor Consumption (TFC) Per Participant | The TFC per participant was the sum of the total amount (in international units [IU]) infused for each infusion for each participant. Annualized TFC of nonacog alfa was derived for each participant by using the following formula; Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as the number of days beginning on Visit 2 ("Day 1", provided an infusion was given) up to Visit 4 (any 1 day between Day 52 to Day 60). | Safety analysis set included all participants who received at least 1 dose of nonacog alfa. | Posted | Mean | Standard Deviation | International units per participant | Up to 88 Days |
|
|
|
| Secondary | Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant | The total amount in international units (IU) per kilogram infused for each infusion recorded were summed to calculate the total factor consumption for each participant. Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as Date of Visit 4 - Date of Visit 2 +1. If Date of Visit 4 - Last dosing date was > 10 days then Date of Visit 4 was replaced with Last dosing date. If Date of Visit 4 was missing (due to discontinuation or any other reason), then Date of Visit 4 was replaced with last dosing date or last subject visit date, whichever was greater. International unit per kilogram= IU/kg. | Safety analysis set included all participants who received at least 1 dose of nonacog alfa. | Posted | Mean | Standard Deviation | IU/kg per participant | Up to 88 days |
|
|
|
| Secondary | Mean Number of Nonacog Alfa Infusions Used to Treat Each Bleed | Number of nonacog alfa infusions used to treat each bleed in participants was calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). On demand treatment was defined as treatment used to treat a bleeding episode. | Since, there were no participants with bleed, therefore data for this outcome measure was not collected and analyzed. | Posted | Up to 88 days |
|
|
| 0 |
| 25 |
| 0 |
| 25 |
| 3 |
| 25 |
| Pyrexia | General disorders | MedDRA v23.0 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | 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.
| 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 |