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| Name | Class |
|---|---|
| LFB USA, Inc. | INDUSTRY |
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The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).
A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients from birth to <12 years old with Inhibitors to Factor VIII or IX: PerSept 2
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Coagulation Factor VIIa (Recombinant): 75 µg/kg | Active Comparator | 75 µg/kg treatment regimen for 3 months |
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| Coagulation Factor VIIa (Recombinant): 225 µg/kg | Active Comparator | 225 µg/kg treatment regimen for 3 months |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Coagulation FVIIa (Recombinant) | Biological | A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement. | For the primary efficacy endpoint, successful treatment of mild/moderate bleeding episode was defined as meeting all of the following:
| 12 hours after first administration of study drug |
| Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition |
| 12 hours after first administration of study drug |
| Measure | Description | Time Frame |
|---|---|---|
| Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes | Based on Patient-Reported "Good" or "Excellent" responses as per the below descriptions: Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug. Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage). No additional infusion of study drug was required. |
| Measure | Description | Time Frame |
|---|---|---|
| Mild/Moderate Bleeding Episodes With Successful Pain Relief | Successful pain relief was defined as a Visual Analogue Scale (VAS: 0-100; 0: no pain at all; 100: the worst pain ever possible) pain score at 12 hours after initial study drug administration that was less than the pain score at the start of treatment with study drug. | 12 hour after first administration of study drug |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Michael Wang, MD | University of Colorado, Denver | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Colorado Denver Hemophilia & Thrombosis Center | Aurora | Colorado | 80045 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40765904 | Derived | Young G, Mahlangu J, Boggio LN, Carcao M, Dargaud Y, Escobar M, Giermasz A, Hermans C, Kuriakose P, Miesbach W, Nance D, Rafique A, Sidonio RF Jr, Vilchevska KV, Wang M, Pipe SW. Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials. Blood Vessel Thromb Hemost. 2025 Mar 27;2(3):100069. doi: 10.1016/j.bvth.2025.100069. eCollection 2025 Aug. | |
| 40674256 |
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| ID | Title | Description |
|---|---|---|
| FG000 | FVIIa: 75 µg/kg First, Then 225 µg/kg | Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study. |
| FG001 | FVIIa: 225 µg/kg First, Then 75 µg/kg |
| 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 | Jun 29, 2016 | Jul 30, 2020 |
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| 12 hour after first administration of study drug |
| Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes | Categories of Response to Treatment are Described as Follows: None: No noticeable effect of the treatment on the bleed or worsening of patient's condition. Continuation of treatment with the study drug was needed. Moderate: Some effect of the treatment on the bleed was noticed, e.g., pain decreased or bleeding signs improved, but bleed continued and required continued treatment with the study drug. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug. Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required. | Within 24 hours of Bleeding Episode |
| Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode | The number of study drug administrations with non-missing dose information in order to treat one mild/moderate bleeding episode. | Within 24 hours of Bleeding Episode |
| Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode | The total amount of study drug administered in order to treat one mild/moderate bleeding episode. | Within 24 hours of Bleeding Episode |
| Jimmy Everest Center for Cancer and Bleeding Disorders |
| Oklahoma City |
| Oklahoma |
| 73117 |
| United States |
| UT Southwestern Medical Center at Dallas / Children's Medical Center | Dallas | Texas | 75390 | United States |
| University Multiprofile Hospital for Active Treatment "Sveti Georgi" | Plovdiv | Bulgaria |
| University Hospital Motol | Prague | Czechia |
| Hematology of Department Hemophilia and Thromboses center | Tbilisi | Georgia |
| Worthwhile Clinical Trials | Benoni | South Africa |
| National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine) | Kyiv | Ukraine |
| Institute of Blood Pathology and Transfusion Medicine | Lviv | Ukraine |
| Derived |
| Carcao M, Hermans C, Giermasz A, Kessler C, Miesbach W, Quon D, Windyga J, Mahlangu J. Safety and Use of Eptacog Beta 225 microg/kg in Patients With Haemophilia A or B With Inhibitors. Haemophilia. 2025 Sep;31(5):957-965. doi: 10.1111/hae.70083. Epub 2025 Jul 17. |
Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study.
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | FVIIa: 75 µg/kg First, Then 225 µg/kg | Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study. |
| BG001 | FVIIa: 225 µg/kg First, Then 75 µg/kg | Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants | No |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Weight | Mean | Standard Deviation | kg |
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| Height | Mean | Standard Deviation | cm |
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| Body Mass Index (BMI) | Mean | Standard Deviation | kg/m^2 |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement. | For the primary efficacy endpoint, successful treatment of mild/moderate bleeding episode was defined as meeting all of the following:
| Treated Population | Posted | Number | 95% Confidence Interval | Proportion of successfully treated BEs | 12 hours after first administration of study drug | Bleeding Episodes (BEs) | Bleeding Episodes (BEs) |
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| Primary | Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition |
| Treated Population | Posted | Number | 95% Confidence Interval | Proportion of successfully treated BEs | 12 hours after first administration of study drug | Bleeding Episodes (BEs) | Bleeding Episodes (BEs) |
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| Secondary | Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes | Based on Patient-Reported "Good" or "Excellent" responses as per the below descriptions: Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug. Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage). No additional infusion of study drug was required. | Treated Population | Posted | Number | 95% Confidence Interval | Proportion of successfully treated BEs | 12 hour after first administration of study drug | Bleeding Episodes (BEs) | Bleeding Episodes (BEs) |
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| Secondary | Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes | Categories of Response to Treatment are Described as Follows: None: No noticeable effect of the treatment on the bleed or worsening of patient's condition. Continuation of treatment with the study drug was needed. Moderate: Some effect of the treatment on the bleed was noticed, e.g., pain decreased or bleeding signs improved, but bleed continued and required continued treatment with the study drug. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug. Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required. | Treated Population with non-missing measurements | Posted | Median | 95% Confidence Interval | Hours | Within 24 hours of Bleeding Episode | Bleeding Episodes with Event | Bleeding Episodes with Event |
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| Secondary | Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode | The number of study drug administrations with non-missing dose information in order to treat one mild/moderate bleeding episode. | Treated Population | Posted | Mean | Standard Deviation | Administrations of Study Drug | Within 24 hours of Bleeding Episode | Bleeding Episodes (BEs) | Bleeding Episodes (BEs) |
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| Secondary | Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode | The total amount of study drug administered in order to treat one mild/moderate bleeding episode. | Treated Population with non-missing measurements | Posted | Mean | Standard Deviation | mL per bleeding episode | Within 24 hours of Bleeding Episode | Bleeding Episodes (BEs) | Bleeding Episodes (BEs) |
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| Other Pre-specified | Mild/Moderate Bleeding Episodes With Successful Pain Relief | Successful pain relief was defined as a Visual Analogue Scale (VAS: 0-100; 0: no pain at all; 100: the worst pain ever possible) pain score at 12 hours after initial study drug administration that was less than the pain score at the start of treatment with study drug. | Treated Population | Posted | Count of Units | Bleeding Episodes (BEs) | 12 hour after first administration of study drug | Bleeding Episodes (BEs) | Bleeding Episodes (BEs) |
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From signing of the informed consent/assent until resolution or 30 days after the last dose of LR769 or early termination, whichever came first. The average duration for monitoring adverse event is about 11.2 months per patient.
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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 | Coagulation Factor VIIa (Recombinant): 75 µg/kg | 75 µg/kg treatment regimen for 3 months Coagulation FVIIa (Recombinant): A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX | 0 | 23 | 0 | 23 | 12 | 23 |
| EG001 | Coagulation Factor VIIa (Recombinant): 225 µg/kg | 225 µg/kg treatment regimen for 3 months Coagulation FVIIa (Recombinant): A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX | 0 | 25 | 2 | 25 | 15 | 25 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dysentery | Infections and infestations | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Paresis | Nervous system disorders | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Diarrhoea | Gastrointestinal disorders | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Vomiting | Gastrointestinal disorders | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Bronchitis | Infections and infestations | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Nasopharyngitis | Infections and infestations | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Respiratory tract infection viral | Infections and infestations | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Rhinitis | Infections and infestations | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | Term from vocabulary, MedDRA 15.1 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Kerry Biron, Director US Clinical Operations | LFB USA, Inc. | 508-370-5166 | kerry.biron@lfb-usa.com |
| Prot_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Final Version 2.0 | Sep 12, 2017 | Jun 23, 2020 | SAP_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Statistical Analysis Plan (v2.0) Addendum | Sep 25, 2017 | Jul 30, 2020 | SAP_002.pdf |
| ID | Term |
|---|---|
| C103587 | recombinant FVIIa |
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| >= 6 years to < 12 years old |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Bleeding Episodes (BEs) |
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| Bleeding Episodes (BEs) |
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| Bleeding Episodes (BEs) |
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| Bleeding Episodes (BEs) |
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| Bleeding Episodes (BEs) |
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