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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1258-0779 | Other Identifier | WHO | |
| jRCT2051200066 | Registry Identifier | jRCT |
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The main aims of the study are to learn if TAK-672 can control bleeds in participants with acquired hemophilia A and if the participants have side effects from TAK-672. Acquired hemophilia A is when people's immune system attacks specific proteins, known as clotting factors, in their bodies. This is different from hemophilia A, which is a condition people are born with.
At the first visit, the study doctor will check who can take part. For those who can take part, participants will visit the clinic or hospital when they get their next bleed. They will receive TAK-672 slowly through a vein. This is called an infusion. They might need extra infusions of TAK-672 to control the bleed. After their bleed is controlled, participants will regularly visit the clinic for a check-up and to treat any further bleeds. This will happen until all participants have received their last dose of TAK-672 to control their 1st bleed. After this, all participants will visit the clinic 90 days later for a final check-up.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TAK-672 | Experimental | TAK-672 was administered at an initial dose of 200 U/kg with IV infusion at a rate of 1-2 mL/min at Day 1. Subsequent doses were determined based on the post-infusion FVIII:C achieved after the most recent dose given, the target FVIII:C, and pFVIII inhibitor titer. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TAK-672 | Biological | B-Domain Deleted Recombinant Porcine Factor VIII |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Severe Bleeding Episodes Who Demonstrated Response to TAK-672 Therapy at 24 Hours After the Initiation of Treatment | Percentage of severe bleeding episodes with demonstrated response to TAK-672 therapy at 24 hours after the initiation of treatment was assessed by using a well-defined 4-point ordinal scale - A 'positive response' was defined as 'effective' (bleeding stopped with clinical control and FVIII:C levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII:C levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' was defined as 'bleeding slightly reduced or unchanged and FVIII:C levels of less than 50%'. 'Not effective' was defined as 'bleeding worsening and FVIII:C levels of less than 20%'. | 24 hours after the initial dose of TAK-672 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Severe Bleeding Episodes Successfully Controlled With TAK-672 Therapy, as Assessed by the Investigator | If the status assessed by the investigator at the end of study treatment of initial treatment period was "successfully controlled", the participant's initial severe bleeding episode was considered as successfully controlled. | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
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Inclusion Criteria:
Male or female Japanese participants of >=18 years of age.
Participants who (or their legally authorized representatives) have provided his/her written informed consent form prior to any study-related procedures and study product administration.
Participants with a diagnosis of AHA based on clinical evaluation and supportive local laboratory testing as shown below:
Participants with a severe bleeding episode which the investigator finds necessary to treat and whose severe bleeding episode meets at least 1 of the following criteria:
Participants who are taking anti-thrombotics (including anti-platelet agents and anticoagulantswith confirmatory laboratory testing documenting specific FVIII inhibitor titer and with 3 half-lives of the agent have elapsed since the last dose.
Participants with expected life expectancies of at least 90 days prior to the onset of the hemorrhagic episode.
Participants of reproductive age who have agreed to use acceptable methods of contraception and if female, undergo pregnancy testing as part of the screening process.
Participant who are able to willing and able to comply with the requirements of the protocol.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Gunma University Hospital | Maebashi | Gunma | Japan | |||
| Nagoya University Hospital |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites.
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A total of 5 participants with Acquired Hemophilia A (AHA) were enrolled in this study to receive TAK-672 at an initial dose of 200 units per kilogram (U/kg).
Participants took part in the study at 9 investigative sites in Japan from 09 April 2021 to 29 November 2022.
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| ID | Title | Description |
|---|---|---|
| FG000 | TAK-672 | TAK-672 was administered at an initial dose of 200 U/kg with intravenous (IV) infusion at a rate of 1-2 milliliters per minute (mL/min) at Day 1. Subsequent doses were determined based on the post-infusion factor VIII activity (FVIII:C) achieved after the most recent dose given, the target FVIII:C, and pFVIII inhibitor titer. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Full analysis Set (FAS) included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses.
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| ID | Title | Description |
|---|---|---|
| BG000 | TAK-672 | TAK-672 was administered at an initial dose of 200 U/kg with IV infusion at a rate of 1-2 mL/min at Day 1. Subsequent doses were determined based on the post-infusion FVIII:C achieved after the most recent dose given, the target FVIII:C, and pFVIII inhibitor titer. |
| 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 With Severe Bleeding Episodes Who Demonstrated Response to TAK-672 Therapy at 24 Hours After the Initiation of Treatment | Percentage of severe bleeding episodes with demonstrated response to TAK-672 therapy at 24 hours after the initiation of treatment was assessed by using a well-defined 4-point ordinal scale - A 'positive response' was defined as 'effective' (bleeding stopped with clinical control and FVIII:C levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII:C levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' was defined as 'bleeding slightly reduced or unchanged and FVIII:C levels of less than 50%'. 'Not effective' was defined as 'bleeding worsening and FVIII:C levels of less than 20%'. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Number | 95% Confidence Interval | percentage of participants | 24 hours after the initial dose of TAK-672 |
|
From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
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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 | TAK-672 | TAK-672 was administered at an initial dose of 200 U/kg with IV infusion at a rate of 1-2 mL/min at Day 1. Subsequent doses were determined based on the post-infusion FVIII:C achieved after the most recent dose given, the target FVIII:C, and pFVIII inhibitor titer. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Central hypothyroidism | Endocrine disorders | MedDRA 25 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anal fissure | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study | Director | +1-877-825-3327 | TrialDisclosures@takeda.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 23, 2020 | Nov 12, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 14, 2022 | Nov 12, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| C536392 | Factor 8 deficiency, acquired |
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| ID | Term |
|---|---|
| D005169 | Factor VIII |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
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| Percentage of Participants With Bleeding Episodes Responsive to TAK-672 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator | A 'positive response' was defined as 'effective' (bleeding stopped with clinical control and FVIII:C levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII:C levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' was defined as 'bleeding slightly reduced or unchanged and FVIII:C levels of less than 50%'. 'Not effective' was defined as 'bleeding worsening and FVIII:C levels of less than 20%'. Data is reported only for the timepoints having at least one participant available for analysis. | At 0.5, 8, 16, 24, 48, 60, 96, and 120 hours post dose and at 1, 14, 28, 42, 56, 70, and 90 day follow-up |
| Frequency of Infusions Per Participant of TAK-672 Required to Successfully Control Qualifying Bleeding Episodes | 'Frequency of infusions ' was calculated as the 'average number of infusions per day'. 'Qualifying bleeding episode' was defined as the 'initial, severe bleeding episode'. | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Total Dose of Infusions Per Participant of TAK-672 Required to Successfully Control Qualifying Bleeding Episodes | 'Qualifying bleeding episode' was defined as the 'initial, severe bleeding episode'. Total dose of infusions is sum of doses from start of treatment with TAK-672 for qualifying bleeding episodes until completion of the management of the bleeding. | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Total Number of Infusions Per Participant of TAK-672 Required to Successfully Control Qualifying Bleeding Episodes | 'Qualifying bleeding episode' was defined as the 'initial, severe bleeding episode'. If the status assessed by the investigator at the end of study treatment of initial treatment period was "successfully controlled", the participant's initial severe bleeding episode was considered as successfully controlled. | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Percentage of Participants With Response to TAK-672 Therapy at Specified Time Points and Eventual Control of Severe Bleeding Episodes | Percentage of participants with response to TAK-672 therapy at specified time points and eventual control of severe bleeding episodes was assessed to determine the correlation between response to TAK-672 therapy and eventual control of severe bleeding episodes. A 'positive response' was defined as 'effective' (bleeding stopped with clinical control and FVIII:C levels of 50% or higher) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII:C levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' was defined as 'bleeding slightly reduced or unchanged and FVIII:C levels of less than 50%'. 'Not effective' was defined as 'bleeding worsening and FVIII:C levels of less than 20%'. Data is reported only for the timepoints having at least one participant available for analysis. | At 0.5, 8, 16, 24, 48, 60, 96,120, and 144 hours post dose and at 1, 14, 28, 42, 56, 70, and 90 day follow-up |
| Mean Value of Pre-infusion Anti-TAK-672 Antibody Titers in Participants With Successful Control of the Bleeding Episode | Mean value of pre-infusion anti-TAK-672 antibody titers (unit: Bethesda unit per milliliters [BU/mL]) in participants with successful control of the bleeding episode was reported as a result of correlation among the pre-infusion pFVIII inhibitor titers and the eventual control of the bleeding episode in this outcome measure. | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Mean Value of Total Dose Per Participant in Participants With Successful Control of the Bleeding Episode | Mean value of total dose per participant for initial treatment period (unit: units/kg per participant) in participants with successful control of the bleeding episode was reported as a results of correlation among the total dose per participant of TAK-672 and the eventual control of the bleeding episode in this outcome measure. | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Number of Participants With Positive Response at 24 Hours After the Initiation of Treatment in Participants With Successful Control of the Bleeding Episode | Number of participants with positive response at 24 hours after the initiation of treatment in participants with successful control of the bleeding episode was reported as a results of correlation among the response at 24 hours and the eventual control of the bleeding episode. A 'positive response' was defined as 'effective'(bleeding stopped with clinical control and FVIII:C levels of 50%or higher) or 'partially effective'(bleeding reduced with clinical stabilization and FVIII:C levels of 20%or higher) control of bleeding, as determined by investigator using 4-point rating scale (effective-partially effective-poorly effective-not effective). | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Anti-hFVIII Inhibitor Titers | Data is reported only for the timepoints having at least one participant available for analysis. | Baseline, 72 hours postdose, and at 14, 28, 42, 56, 70, and 90 days follow-up |
| Anti-pFVIII Inhibitor Titer | Data is reported only for the timepoints having at least one participant available for analysis. | Baseline, 72 hours postdose, and at 14, 28, 42, 56, 70, and 90 days follow-up |
| Terminal Half-life (t1/2) for TAK-672 | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
| Clearance (CL) for TAK-672 | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
| Volume of Distribution (Vd) for TAK-672 | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
| Area Under the Concentration-Time Curve (AUC) for TAK-672 | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with the initial dose of TAK-672 |
| Maximum Drug Concentration (Cmax) Per Dose (Cmax/Dose) for TAK-672 | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
| Duration Period From Initial Dose of TAK-672 Until Completion of Hemostasis Control | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Total Dose Per Participant From Initial Dose of TAK-672 Until Completion of Hemostasis Control | Total dose is sum of doses from start of treatment with TAK-672 until completion of hemostasis control. | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Number of Participants With New Qualified Severe Bleeding Episodes | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
| Aichi |
| Nagoya |
| Japan |
| Nara Medical University Hospital | Kashihara-shi | Nara | Japan |
| Uonuma Kikan Hospital | Minamiuonuma | Niigata | Japan |
| Jichi Medical University Hospital | Shimotsuke | Tochigi | Japan |
| Tokyo Saiseikai Central Hospital | Mita | Tokyo | Japan |
| Chiba University Hospital | Chiba | Japan |
| Fukushima Medical University Hospital | Fukushima | Japan |
| Yamagata University Hospital | Yamagata | Japan |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Height | Mean | Standard Deviation | centimeters (cm) |
|
| Weight | Mean | Standard Deviation | kilograms (kg) |
|
| Title |
|---|
| Description |
|---|
| OG000 | TAK-672 | TAK-672 was administered at an initial dose of 200 U/kg with IV infusion at a rate of 1-2 mL/min at Day 1. Subsequent doses were determined based on the post-infusion FVIII:C achieved after the most recent dose given, the target FVIII:C, and pFVIII inhibitor titer. |
|
|
| Secondary | Percentage of Participants With Severe Bleeding Episodes Successfully Controlled With TAK-672 Therapy, as Assessed by the Investigator | If the status assessed by the investigator at the end of study treatment of initial treatment period was "successfully controlled", the participant's initial severe bleeding episode was considered as successfully controlled. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Number | 95% Confidence Interval | percentage of participants | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Percentage of Participants With Bleeding Episodes Responsive to TAK-672 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator | A 'positive response' was defined as 'effective' (bleeding stopped with clinical control and FVIII:C levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII:C levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' was defined as 'bleeding slightly reduced or unchanged and FVIII:C levels of less than 50%'. 'Not effective' was defined as 'bleeding worsening and FVIII:C levels of less than 20%'. Data is reported only for the timepoints having at least one participant available for analysis. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. Number of participants analyzed are the number of participants available for analysis at the given timepoint. | Posted | Number | 95% Confidence Interval | percentage of participants | At 0.5, 8, 16, 24, 48, 60, 96, and 120 hours post dose and at 1, 14, 28, 42, 56, 70, and 90 day follow-up |
|
|
|
| Secondary | Frequency of Infusions Per Participant of TAK-672 Required to Successfully Control Qualifying Bleeding Episodes | 'Frequency of infusions ' was calculated as the 'average number of infusions per day'. 'Qualifying bleeding episode' was defined as the 'initial, severe bleeding episode'. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Mean | Standard Deviation | infusions per participant | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Total Dose of Infusions Per Participant of TAK-672 Required to Successfully Control Qualifying Bleeding Episodes | 'Qualifying bleeding episode' was defined as the 'initial, severe bleeding episode'. Total dose of infusions is sum of doses from start of treatment with TAK-672 for qualifying bleeding episodes until completion of the management of the bleeding. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Mean | Standard Deviation | dose per participant | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Total Number of Infusions Per Participant of TAK-672 Required to Successfully Control Qualifying Bleeding Episodes | 'Qualifying bleeding episode' was defined as the 'initial, severe bleeding episode'. If the status assessed by the investigator at the end of study treatment of initial treatment period was "successfully controlled", the participant's initial severe bleeding episode was considered as successfully controlled. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Mean | Standard Deviation | infusions per participant | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Percentage of Participants With Response to TAK-672 Therapy at Specified Time Points and Eventual Control of Severe Bleeding Episodes | Percentage of participants with response to TAK-672 therapy at specified time points and eventual control of severe bleeding episodes was assessed to determine the correlation between response to TAK-672 therapy and eventual control of severe bleeding episodes. A 'positive response' was defined as 'effective' (bleeding stopped with clinical control and FVIII:C levels of 50% or higher) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII:C levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' was defined as 'bleeding slightly reduced or unchanged and FVIII:C levels of less than 50%'. 'Not effective' was defined as 'bleeding worsening and FVIII:C levels of less than 20%'. Data is reported only for the timepoints having at least one participant available for analysis. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. Number of participants analyzed are the number of participants available for analysis at the given timepoint. | Posted | Number | percentage of participants | At 0.5, 8, 16, 24, 48, 60, 96,120, and 144 hours post dose and at 1, 14, 28, 42, 56, 70, and 90 day follow-up |
|
|
|
| Secondary | Mean Value of Pre-infusion Anti-TAK-672 Antibody Titers in Participants With Successful Control of the Bleeding Episode | Mean value of pre-infusion anti-TAK-672 antibody titers (unit: Bethesda unit per milliliters [BU/mL]) in participants with successful control of the bleeding episode was reported as a result of correlation among the pre-infusion pFVIII inhibitor titers and the eventual control of the bleeding episode in this outcome measure. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Mean | Standard Deviation | Bethesda unit per milliliters (BU/mL) | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Mean Value of Total Dose Per Participant in Participants With Successful Control of the Bleeding Episode | Mean value of total dose per participant for initial treatment period (unit: units/kg per participant) in participants with successful control of the bleeding episode was reported as a results of correlation among the total dose per participant of TAK-672 and the eventual control of the bleeding episode in this outcome measure. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Mean | Standard Deviation | units/kg per participant | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Number of Participants With Positive Response at 24 Hours After the Initiation of Treatment in Participants With Successful Control of the Bleeding Episode | Number of participants with positive response at 24 hours after the initiation of treatment in participants with successful control of the bleeding episode was reported as a results of correlation among the response at 24 hours and the eventual control of the bleeding episode. A 'positive response' was defined as 'effective'(bleeding stopped with clinical control and FVIII:C levels of 50%or higher) or 'partially effective'(bleeding reduced with clinical stabilization and FVIII:C levels of 20%or higher) control of bleeding, as determined by investigator using 4-point rating scale (effective-partially effective-poorly effective-not effective). | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Count of Participants | Participants | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Anti-hFVIII Inhibitor Titers | Data is reported only for the timepoints having at least one participant available for analysis. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. Number analyzed is the number of participants available for analysis at the given timepoint. | Posted | Mean | Standard Deviation | BU/mL | Baseline, 72 hours postdose, and at 14, 28, 42, 56, 70, and 90 days follow-up |
|
|
|
| Secondary | Anti-pFVIII Inhibitor Titer | Data is reported only for the timepoints having at least one participant available for analysis. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. Number analyzed is the number of participants available for analysis at the given timepoint. | Posted | Mean | Standard Deviation | BU/mL | Baseline, 72 hours postdose, and at 14, 28, 42, 56, 70, and 90 days follow-up |
|
|
|
| Secondary | Terminal Half-life (t1/2) for TAK-672 | PK evaluation was planned however, no PK sample was collected in this study due to none of the participants gave consent for the same. | Posted | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
|
|
| Secondary | Clearance (CL) for TAK-672 | PK evaluation was planned however, no PK sample was collected in this study due to none of the participants gave consent for the same. | Posted | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
|
|
| Secondary | Volume of Distribution (Vd) for TAK-672 | PK evaluation was planned however, no PK sample was collected in this study due to none of the participants gave consent for the same. | Posted | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
|
|
| Secondary | Area Under the Concentration-Time Curve (AUC) for TAK-672 | PK evaluation was planned however, no PK sample was collected in this study due to none of the participants gave consent for the same. | Posted | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with the initial dose of TAK-672 |
|
|
| Secondary | Maximum Drug Concentration (Cmax) Per Dose (Cmax/Dose) for TAK-672 | PK evaluation was planned however, no PK sample was collected in this study due to none of the participants gave consent for the same. | Posted | Pre-dose and at multiple time points post-dose (up to 24 hours) after over 48 hours from the last treatment with TAK-672 |
|
|
| Secondary | Duration Period From Initial Dose of TAK-672 Until Completion of Hemostasis Control | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Median | Full Range | days | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Total Dose Per Participant From Initial Dose of TAK-672 Until Completion of Hemostasis Control | Total dose is sum of doses from start of treatment with TAK-672 until completion of hemostasis control. | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Mean | Standard Deviation | dose per participant | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| Secondary | Number of Participants With New Qualified Severe Bleeding Episodes | FAS included all participants who had received at least 1 dose of the TAK-672 and was used for the efficacy analyses. | Posted | Count of Participants | Participants | From first dose of study drug up to 90 days after the last dose of study drug or until discontinued (Up to 95 days as a maximum) |
|
|
|
| 0 |
| 5 |
| 2 |
| 5 |
| 5 |
| 5 |
| Cryoglobulinaemia | Vascular disorders | MedDRA 25 | Systematic Assessment |
|
| Haemorrhage subcutaneous | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
|
| Haemorrhagic diathesis | Blood and lymphatic system disorders | MedDRA 25 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 25 | Systematic Assessment |
|
| Apnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
|
| Blood urine present | Investigations | MedDRA 25 | Systematic Assessment |
|
| Conjunctival haemorrhage | Eye disorders | MedDRA 25 | Systematic Assessment |
|
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
|
| Delirium | Psychiatric disorders | MedDRA 25 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 25 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 25 | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
|
| Haematochezia | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
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| Haemorrhage subcutaneous | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
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| Hyperplastic cholecystopathy | Hepatobiliary disorders | MedDRA 25 | Systematic Assessment |
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| Hypoaesthesia | Nervous system disorders | MedDRA 25 | Systematic Assessment |
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| Mouth haemorrhage | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
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| Nephrolithiasis | Renal and urinary disorders | MedDRA 25 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
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| Periodontal disease | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
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| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
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| Pulmonary alveolar haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 25 | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
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| Skin haemorrhage | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 25 | Systematic Assessment |
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| Vessel puncture site haemorrhage | General disorders | MedDRA 25 | Systematic Assessment |
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The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
| D011498 |
| Protein Precursors |
| D001685 | Biological Factors |
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| Initial Treatment Period at 16 Hours |
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| Initial Treatment Period at 24 Hours |
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| Initial Treatment Period at 48 Hours |
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| Initial Treatment Period at 60 Hours |
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| Initial Treatment Period at 96 Hours |
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| Initial Treatment Period at 120 Hours |
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| Initial Treatment Period Follow-up at 24 Hours on Day 1 |
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| Initial Treatment Period Follow-up at 14 Days |
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| Initial Treatment Period Follow-up at 28 Days |
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| Initial Treatment Period Follow-up at 42 Days |
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| Initial Treatment Period Follow-up at 56 Days |
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| Initial Treatment Period Follow-up at 70 Days |
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| Initial Treatment Period Follow-up at 90 Days (End of Study) |
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| Initial Treatment Period at 16 Hours |
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| Initial Treatment Period at 24 Hours |
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| Initial Treatment Period at 48 Hours |
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| Initial Treatment Period at 60 Hours |
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| Initial Treatment Period at 96 Hours |
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| Initial Treatment Period at 120 Hours |
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| Initial Treatment Period at 144 Hours |
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| Initial Treatment Period Follow-up at 24 Hours on Day 1 |
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| Initial Treatment Period Follow-up at 14 Days |
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| Initial Treatment Period Follow-up at 28 Days |
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| Initial Treatment Period Follow-up at 42 Days |
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| Initial Treatment Period Follow-up at 56 Days |
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| Initial Treatment Period Follow-up at 70 Days |
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| Initial Treatment Period Follow-up at 90 days (End of Study) |
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| Initial Treatment Period Follow-up at 14 Days |
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| Initial Treatment Period Follow-up at 28 Days |
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| Initial Treatment Period Follow-up at 42 Days |
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| Initial Treatment Period Follow-up at 56 Days |
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| Initial Treatment Period Follow-up at 70 Days |
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| Initial Treatment Period Follow-up at 90 Days (End of Study) |
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| Initial Treatment Period Follow-up at 14 Days |
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| Initial Treatment Period Follow-up at 28 Days |
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| Initial Treatment Period Follow-up at 42 Days |
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| Initial Treatment Period Follow-up at 56 Days |
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| Initial Treatment Period Follow-up at 70 Days |
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| Initial Treatment Period Follow-up at 90 Days (End of Study) |
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