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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-003629-27 | EudraCT Number |
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| Name | Class |
|---|---|
| Swedish Orphan Biovitrum | INDUSTRY |
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The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Experimental | Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX (FIX) levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rFIXFc | Biological | Adjustments to the dose and interval of rFIXFc was made in this study based on investigator discretion using available pharmacokinetic (PK) data, subsequent FIX trough and peak levels, level of physical activity, and bleeding pattern, in accordance with local standards of care for a prophylactic regimen. There was an option to start study dosing as episodic treatment (on-demand). |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay | Development of an inhibitor was defined as an inhibitor test result of >= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of >=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay. | Up to 3 years |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) | ABR was annualized number of bleeding episodes during efficacy period (EP) per participant normalized to a 1-year interval of time. Bleeding episodes were classified as: spontaneous if parent/caregiver/participant records bleeding event when there is no known contributing factor such as definite trauma or antecedent strenuous activity; and traumatic when there is known reason for bleed. ABR=(Number of bleeding episodes during EP/total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimens excluding surgical/rehabilitation periods and large injection intervals (greater than [>]28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol-defined inclusion/exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Sacramento | California | 95817 | United States | ||
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38922990 | Derived | Nolan B, Recht M, Rendo P, Falk A, Foster M, Casiano S, Rauch A, Shapiro A. Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B-LONG study. Eur J Haematol. 2024 Oct;113(4):485-492. doi: 10.1111/ejh.14252. Epub 2024 Jun 25. | |
| 34242387 |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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A total of 38 participants were screened. A total of 33 participants were enrolled, 22 participants began study participation on episodic treatment regimen and 11 participants began study participation on prophylactic treatment regimen.
The study was conducted at 28 active centers in 11 countries. Participants were screened between 13-Nov-2014 to 23-Jul-2018.
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| ID | Title | Description |
|---|---|---|
| FG000 | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Aug 6, 2018 | Jul 15, 2020 |
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|
|
| Up to 3 years |
| Annualized Number of Spontaneous Joint Bleeding Episodes | Bleeding episodes were classified as spontaneous if parent/caregiver/participant records a bleeding event when there is no known contributing factor such as a definite trauma or antecedent "strenuous" activity. Annualized spontaneous joint bleeding episodes=(Total number of spontaneous joint bleeding episodes during efficacy period (EP) divided by total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals (>28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Up to 3 years |
| Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Using e-diary, each participant's parent/caregiver rated treatment response to any bleeding episode (BE) at approximately 8 to 12 hours from time of injection and prior to additional doses of rFIXFc given for same BE using 4-point scale:- 1=Excellent: abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hours after initial injection; 2=Good: definite pain relief and/or improvement in signs of bleeding within approx. 8 hours after injection, but possibly requiring more than 1 injection after 24-48 hours for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8 hours after initial injection and requires more than 1 injection and 4=None: No improvement or condition worsens within approx. 8 hours after initial injection. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Up to 3 years |
| Total Number of Exposure Days (EDs) | An ED was defined as a 24-hour period in which a participant received one or more doses of rFIXFc injections, with the time of the first injection of rFIXFc defined as the start of the ED. Participant who did not have a particular injection type were counted as having zero injections for that type. | Up to 3 years |
| Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes | Total annualized rFIXFc consumption (in IU/kg) was calculated for each participant as: Annualized consumption = (Total IU/kg of rFIXFc during efficacy period (EP) divided by total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (> 28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Up to 3 years |
| Number of Injections of rFIXFc Required to Resolve a Bleeding Episode | Number of injections of rFIXFc required to resolve a bleeding episode during efficacy period (EP) were reported. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (>28 days). All injections given from the initial sign of a bleed, until the last date/time within the bleed window were counted. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Up to 3 years |
| Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode | The average dose of rFIXFc per injection per bleeding episode was calculated as the average of all doses (IU/kg) administered to treat the bleeding episode during efficacy period (EP). EP begins with the first treatment regimen dose of rFIXFc and ends with the last dose (regardless of the reason for dosing). Surgery/rehabilitation periods are not included in the EP. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Up to 3 years |
| Change From Baseline in rFIXFc Incremental Recovery (IR) | Blood samples were taken at trough and Cmax (maximum concentration) for assessment of incremental recovery, measured by the one-stage clotting assay. IR (International Units per deciliter [IU/dL] per IU/kg) = (Cmax for FIX activity - Pre-dose FIX activity) (IU/dL)/ Actual dose (IU/kg), where Cmax is 30 minute FIX activity post-dose and FIX activity less than (<)0.5 IU/dL was set to 0 IU/dL for calculation of IR. | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144 |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Research Site | Atlanta | Georgia | 30322 | United States |
| Research Site | Indianapolis | Indiana | 46260 | United States |
| Research Site | Louisville | Kentucky | 40202 | United States |
| Research Site | New Orleans | Louisiana | 70112 | United States |
| Research Site | East Lansing | Michigan | 48823 | United States |
| Research Site | Traverse City | Michigan | 49684 | United States |
| Research Site | Columbus | Ohio | 43205 | United States |
| Research Site | Portland | Oregon | 97239 | United States |
| Research Site | Pittsburgh | Pennsylvania | 15213 | United States |
| Research Site | Westmead | New South Wales | 2145 | Australia |
| Research Site | Aarhus | 8200 | Denmark |
| Hopital Cardiologique - CHU Lille | Lille | Nord | 59037 | France |
| Research Site | Lyon | Rhone | 69437 | France |
| Research Site | Dublin | D12 N512 | Ireland |
| Research Site | Milan | 20122 | Italy |
| Research Site | Naples | 80122 | Italy |
| Research Site | Parma | 43126 | Italy |
| Research Site | Roma | 00165 | Italy |
| Research Site | Utrecht | 3584 CX | Netherlands |
| Research Site | Auckland | 1023 | New Zealand |
| Research Site | Warsaw | 02-091 | Poland |
| Research Site | Malmö | 205 02 | Sweden |
| Research Site | Cambridge | Cambridgeshire | CB2 0QQ | United Kingdom |
| Research Site | Whitechapel | London | E1 1BB | United Kingdom |
| Research Site | London | WC1N3JH | United Kingdom |
| Derived |
| Nolan B, Klukowska A, Shapiro A, Rauch A, Recht M, Ragni M, Curtin J, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Fischer K, Liesner R. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Adv. 2021 Jul 13;5(13):2732-2739. doi: 10.1182/bloodadvances.2020004085. |
| Episodic Treatment Regimen |
|
| Prophylactic Treatment Regimen |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Analysis was performed on Safety Analysis Set which included all participants who received at least 1 dose of study rFIXFc.
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| ID | Title | Description |
|---|---|---|
| BG000 | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which >=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| 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 With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay | Development of an inhibitor was defined as an inhibitor test result of >= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of >=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay. | Analysis performed on Safety Analysis set which included all participants who had received at least 1 dose of study treatment. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to 3 years |
|
|
| |||||||||||||||||||||||||
| Secondary | Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) | ABR was annualized number of bleeding episodes during efficacy period (EP) per participant normalized to a 1-year interval of time. Bleeding episodes were classified as: spontaneous if parent/caregiver/participant records bleeding event when there is no known contributing factor such as definite trauma or antecedent strenuous activity; and traumatic when there is known reason for bleed. ABR=(Number of bleeding episodes during EP/total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimens excluding surgical/rehabilitation periods and large injection intervals (greater than [>]28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Analysis performed on Full Analysis Set (FAS) which included all enrolled participants with at least 1 dose of study treatment. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen. | Posted | Median | Full Range | episodes per participant per year | Up to 3 years |
| |||||||||||||||||||||||||||
| Secondary | Annualized Number of Spontaneous Joint Bleeding Episodes | Bleeding episodes were classified as spontaneous if parent/caregiver/participant records a bleeding event when there is no known contributing factor such as a definite trauma or antecedent "strenuous" activity. Annualized spontaneous joint bleeding episodes=(Total number of spontaneous joint bleeding episodes during efficacy period (EP) divided by total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals (>28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Analysis performed on FAS. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen. | Posted | Median | Full Range | episodes per participant per year | Up to 3 years |
| |||||||||||||||||||||||||||
| Secondary | Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Using e-diary, each participant's parent/caregiver rated treatment response to any bleeding episode (BE) at approximately 8 to 12 hours from time of injection and prior to additional doses of rFIXFc given for same BE using 4-point scale:- 1=Excellent: abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hours after initial injection; 2=Good: definite pain relief and/or improvement in signs of bleeding within approx. 8 hours after injection, but possibly requiring more than 1 injection after 24-48 hours for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8 hours after initial injection and requires more than 1 injection and 4=None: No improvement or condition worsens within approx. 8 hours after initial injection. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Analysis performed on FAS. Analysis was based on all injections. Here, number analyzed signifies number of responses to injections reported for each treatment regimen. | Posted | Count of Units | responses to injections | Up to 3 years | responses to injections | responses to injections |
| ||||||||||||||||||||||||||
| Secondary | Total Number of Exposure Days (EDs) | An ED was defined as a 24-hour period in which a participant received one or more doses of rFIXFc injections, with the time of the first injection of rFIXFc defined as the start of the ED. Participant who did not have a particular injection type were counted as having zero injections for that type. | Analysis performed on safety analysis set. | Posted | Median | Full Range | days | Up to 3 years |
|
| ||||||||||||||||||||||||||
| Secondary | Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes | Total annualized rFIXFc consumption (in IU/kg) was calculated for each participant as: Annualized consumption = (Total IU/kg of rFIXFc during efficacy period (EP) divided by total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (> 28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Analysis performed on FAS. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen. | Posted | Median | Full Range | IU per kilogram per participant per year | Up to 3 years |
| |||||||||||||||||||||||||||
| Secondary | Number of Injections of rFIXFc Required to Resolve a Bleeding Episode | Number of injections of rFIXFc required to resolve a bleeding episode during efficacy period (EP) were reported. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (>28 days). All injections given from the initial sign of a bleed, until the last date/time within the bleed window were counted. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Analysis performed on FAS. Here, number analyzed signifies number of bleeding episodes reported for each treatment regimen. | Posted | Median | Full Range | injections | Up to 3 years | Bleeding episodes | Bleeding episodes |
| |||||||||||||||||||||||||
| Secondary | Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode | The average dose of rFIXFc per injection per bleeding episode was calculated as the average of all doses (IU/kg) administered to treat the bleeding episode during efficacy period (EP). EP begins with the first treatment regimen dose of rFIXFc and ends with the last dose (regardless of the reason for dosing). Surgery/rehabilitation periods are not included in the EP. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. | Analysis performed on FAS. Here, number analyzed signifies number of bleeding episodes reported for each treatment regimen. | Posted | Median | Full Range | IU/kg | Up to 3 years | Bleeding episodes | Bleeding episodes |
| |||||||||||||||||||||||||
| Secondary | Change From Baseline in rFIXFc Incremental Recovery (IR) | Blood samples were taken at trough and Cmax (maximum concentration) for assessment of incremental recovery, measured by the one-stage clotting assay. IR (International Units per deciliter [IU/dL] per IU/kg) = (Cmax for FIX activity - Pre-dose FIX activity) (IU/dL)/ Actual dose (IU/kg), where Cmax is 30 minute FIX activity post-dose and FIX activity less than (<)0.5 IU/dL was set to 0 IU/dL for calculation of IR. | Analysis performed on FAS. Here number analyzed signifies number of FAS participants with available data for each visit. | Posted | Median | Inter-Quartile Range | IU/dL per IU/kg | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144 |
|
All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
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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 | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which >=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR. | 0 | 33 | 23 | 33 | 27 | 33 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Factor ix inhibition | Blood and lymphatic system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Immune thrombocytopenic purpura | Blood and lymphatic system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Phimosis | Congenital, familial and genetic disorders | MedDra 22.0 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Tongue haemorrhage | Gastrointestinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Vessel puncture site haematoma | General disorders | MedDra 22.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Infusion site pustule | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Viral rash | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Accidental exposure to product | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Face injury | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Skull fracture | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Compartment syndrome | Musculoskeletal and connective tissue disorders | MedDra 22.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDra 22.0 | Systematic Assessment |
| |
| Coma | Nervous system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Febrile convulsion | Nervous system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Spinal cord haematoma | Nervous system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Tongue biting | Nervous system disorders | MedDra 22.0 | Systematic Assessment |
| |
| Prepuce redundant | Reproductive system and breast disorders | MedDra 22.0 | Systematic Assessment |
| |
| Central venous catheterisation | Surgical and medical procedures | MedDra 22.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDra 22.0 | Systematic Assessment |
| |
| Poor venous access | Vascular disorders | MedDra 22.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Middle ear effusion | Ear and labyrinth disorders | MedDra 22.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDra 22.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Viral rash | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDra 22.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDra 22.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDra 22.0 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDra 22.0 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDra 22.0 | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDra 22.0 | Systematic Assessment |
| |
| Balanoposthitis | Reproductive system and breast disorders | MedDra 22.0 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDra 22.0 | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDra 22.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDra 22.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDra 22.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDra 22.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDra 22.0 | Systematic Assessment |
|
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Bioverativ, a Sanofi company | 800-633-1610 | 6 | Contact-US@sanofi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 1, 2019 | Jul 15, 2020 | SAP_001.pdf |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C000599709 | factor IX Fc fusion protein |
Not provided
Not provided
Not provided
| Asian |
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| American Indian or Alaska Native |
|
| Native Hawaiian or other Pacific Islander |
|
| Unknown or Not reported |
|
| Other |
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| Units | Counts |
|---|---|
| Participants |
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| Bleeding episodes |
|
|
| Units | Counts |
|---|---|
| Participants |
|
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| None |
|
| Response not provided |
|